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Fall 2016 Spring 2017 I I Volume Volume8 9

A CHES Publication

Clinical Trials 101: Understanding the Process and the Terminology

Making Life-Long-Distance Friendships: a Review of Inhibitor Family Camp The Timeline of Education: A New and Uncertain Turn l Living with Uncertainty Isn’t Easy. Here’s Help l Clinical Trials: Optimistic Caution Infusion Strife? Productive Life. l Engaging Uncertainty l AllCare: Proving that personalized care is not lost... One patient at a time.


Did YOU Know? In 2017, CHES will be presenting three inhibitor programs!

(1) Leverage is an adventure-based camp for individuals with inhibitors ages 18 and up. It’s scheduled for Wednesday, May 17th-21st in Portland, OR.

(2) Momentum debuted in the early spring of 2015 for men with inhibitors ages 18 and up. CHES is bringing back this popular, 3-day session scheduled on July 14th-16th at the Nikko Hotel in San Francisco, CA.

(3) For its 8th consecutive year, Inhibitor Family Camp (IFC) is returning for families with children living with inhibitors ages 6-18. IFC is set to run on Friday, Oct. 6th-9th at The Painted Turtle. More info on all of our programs is available on comphealthed.com.

Letter From the Editors Spring is in the air! The time when the world around us emerges from its long winter’s nap, bringing with it a sense of rejuvenation. The world becomes brighter, the days become longer, the trees and flowers reveal their splendor and there is the anticipation of rebirth. Change is in the air! In 2017, we are excited to offer three (3) programs for the inhibitor community and making plans to expand Inhibitor Family Camp in 2018. We are also anticipating an expansion of Ladybugs, a program for women ages 16+ that live with or care for an individual(s) with any bleeding disorder. Comprehensive Health Education Services’ (CHES) mission is to inspire awareness and self-reliance for patients with chronic health conditions, their families, and their communities. Here at CHES, we continue to learn from YOU, our program participants and families about what you want and need to learn to become informed, educated and empowered advocates for your child’s or your own healthcare needs. We are an independent, educational, privately owned company run by two members of the hemophilia within the inhibitor community who care passionately about our families. As we often say, “we don’t just know inhibitors, with live them.” All our programs are supported by competitive, educational grants from manufacturers that provide the opportunity to offer educational programs free of charge to families. It is an exhilarating time for the bleeding disorders community. The promise of longeracting products, some delivered subcutaneously as opposed to intravenously, are now a reality. With multiple products in trial there is renewed hope for less frequent infusions, and for some, maybe a potential cure! I remember when my brother was born in 1970 and we anticipated “a cure in our lifetime.” This dream is now knocking at the door. The question is... do you open it? We hope you enjoy our feature article detailing the clinical trial process and helping you to decide what is best for you or your child. As we live through this time of expanding options, change is at the forefront. Some view change as a thrilling adventure with endless possibilities while others are reluctant to embrace change and feel as though, “If it ain’t broke, don’t fix it!” We like things just the way they are. In our Family Matters feature, we take a closer look at our own personal attitudes and acknowledge that uncertainty is a normal reaction to change and that is okay. In What’s the Plan, we take a closer look at how the face of public education may look and how it will affect individuals with special needs. The proposed changes could be devastating for many. Community Chatter brings news of events and programs specific to the inhibitor community written by community members. We love to hear our participant’s voice as they share why these programs are special to them.

LifeLines for HealthSM Disclaimers The views and opinions of our writers are not a reflection of Comprehensive Health Education ServicesTM, Inc. (CHES) or its sponsors. This newsletter is designed to provide a forum for community members to express their views from an open and honest platform. It is meant to provide a sharing of knowledge and experience to help one another. Nothing in this newsletter is meant to replace the advice of your HTC, medical professional team or insurance provider. You are always urged to seek the opinion of a healthcare professional for treatment and your specific insurance provider for information. We take your privacy very seriously. We would never disclose your personal health information without your express written consent. We would never sell nor make available our secure database to anyone. Articles and pictures may not be reproduced, published, and/or placed on websites without the express written permission of CHES. In every publication of LifeLines for HealthSM, we will provide links to other websites that are not owned or controlled by CHES or its sponsors. We cannot be responsible for privacy practices of other website owners, nor can we be responsible for the accuracy of the information provided.

Self-Infusion is a rite of passage for those managing a bleeding disorder. This rite of passage becomes a challenge for those managing an inhibitor given the sheer volume and regular need for factor. Throw in an immune tolerance regimen, and self-infusing seems impossible. In Fun & Inspiration, we share some ways to make this process a little easier. In Bloodlines, we share a glossary of terms related to clinical trials developed by NEHA’s very first Consumer Medical Update Program Committee. In What’s New, we offer a personal perspective of the pros and cons to the avalanche of new products and treatments. We introduce a new segment to LifeLines for Health this year! Mind-Body Connection shares simple exercises to implement daily to manage everyday stressors. In a growing body of research, the paradigm of mind over matter has overwhelming possibilities. As we move through 2017 in a sea of change, uncertainty and in some cases fear, lift your voices. I am reminded of Dr. Suess’ book, “Horton Hears a Who”. Shout it loud, “we are here, we are here, we are here”! Do you have an idea, comment or suggestion? We really want to hear from you! Share your thoughts at info@comphealthed.com. - Janet Brewer & Eric Lowe

“You can’t depend on your judgement when your imagination is out of focus.” -Mark Twain jbrewer@comphealthed.com l elowe@comphealthed.com


Integrity, Accuracy, Empathy...

FEATURE 15 I Clinical Trials 101: Understanding the Process and the Terminology With so many new products on the horizon, these are exciting times. But they can also be confusing times. Allow research coordinator, Emily Coe and Dr. Stacy E. Croteau of Boston Children’s Hospital (Harvard) explain the process of clinical trials, as well as the different approaches of treatment and perhaps even a cure.

CONTENTS COMMUNITY CHATTER

FAMILY MATTERS

7 I Inhibitor Family Camp Updates Inhibitor Family Camp and upcoming dates/locations.

25 I Living with Uncertainty Isn’t Easy. Here’s Help.

8 I Making Life-Long-Distance Friendships: a Review of Inhibitor Family Camp at Victory Junction

Big medical decisions inevitably show their ugly face from time to time, and there’s a lot of uncertainty that goes with them. Dr. Gary McClain offers his advice and some exercises to face them head-on.

Ashley Davis (mother of a child with inhibitors) attended Inhibitor Family Camp for the first time with her family early this spring to discover different connections, but with the same experiences.

SPOTLIGHT 9 I AllCare: Proving that personalized care is not lost... One patient at a time. Meet Tom Joseph, Account Manager of Hemophilia and Bleeding Disorders at AllCare Plus Pharmacy Inc. The very personable Tom speaks on AllCare’s focus - the patient, revealing that both his and AllCare’s heart is in the right place.

FUN & INSPIRATION 14 I Infusion Strife? Productive Life. So you’ve got places to go, people to see, but “Drat!” you’ve got to infuse too. How are you supposed to fit this into your day? We have some tips to consider. (For entertainment purposes only.)

23 I Glossary of Helpful Clinical Trial Terms Still feeling a bit confused about clinical trials after reading our feature? Perhaps this handy glossary will clear things up? Thanks NEHA!

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30 I The Timeline of Education: A New and Uncertain Turn School psychologist, Lisa Cosseboom guides you through the history of our school system’s establishment of equal opportunities in education. But that could soon change with H.R. 610, which would eliminate the No Child Left Behind and Every Student Succeeds Acts among others.

WHAT’S NEW 33 I Clinical Trials: Optimistic Caution Clinical trials are on the rise for some very exciting products. But don’t let you’re excitement get the best of you. We often let our emotions speak louder than our senses. So we’re throwing a side of caution to the wind. Remember to ask yourself the questions in this article before participating in any clinical trial.

MIND BODY CONNECTION

BLOODLINES

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37 I Engaging Uncertainty This spring, we added a new section to Lifelines called Mind Body Connection. Meditation and yoga expert, Krystyn Strother explains that there is a fine balance between control and surrender of our everyday events. Read her strategies for managing uncertainty.

CONTENTS


2017 PROGRAM GUIDE

Event opportunities for members of the inhibitor community CHES has been serving the needs of those with rare bleeding conditions since 2009. As long time members of the bleeding disorder community, our mission is to inspire awareness and self-reliance for patients with chronic health conditions, their families, and their communities. Below is a brief overview of the various programs we are offering to the inhibitor community in 2017. More details on each of our programs can be found on our website: www.comphealthed.com

Lifelines for Health is the only national publication specifically created for the inhibitor community. It is distributed biannually to over 750 families living with inhibitors, HTC’s, local chapters, and industry members. Visit comphealthed.com today to start receiving this free publication. SM

LEVERAGE

SM

The Ultimate Inhibitor Adventure

LeverageSM is a pioneering, national program for young adults from ages 18 and up that have hemophilia with an inhibitor. The program consists of life changing experiences that allow participants to challenge themselves in ways they never thought possible through a series of outdoor adventure, experiential education activities, including fishing, rafting, reflection, and various ropes courses.

Inhibitor Family CampSM is specially designed to meet the needs and limitations of children with both hemophilia & inhibitors. Immediate family members are invited because we understand that chronic illness affects the entire family. We play, learn, and grow while we build a stronger community. To register, applicants must have an active inhibitor, or a history of an inhibitor within the last 24 months, and are between the ages of 6-18. If you’re interested, please act now. Space is limited, and slots are filled on a first come, first-served basis for those who qualify. New families have first priority. Friday, Oct. 6th thru Monday, the 9th, 2017 The Painted Turtle - Lake Hughes, CA Learn more at comphealthed.com/ifc

MomentumSM is a men’s only, national program for those ages 18 and up that continue to live with both - hemophilia and an inhibitor.  The program provides opportunities to not only speak with specialists familiar with inhibitors, but most of all, amongst themselves about issues and challenges important to them.

Ended: Monday, May 15th thru Friday, the 19th, 2017 YMCA Camp Collins - Gresham, OR Learn more at comphealthed.com/Leverage The CHESTM logo and program logos are registered trademarks of Comprehensive Health Education Services, LLC. The use of these marks are restricted in part or their entirety without expressed written consent.

Friday, July 14th thru Sunday, the 16th, 2017 Hotel Nikko - San Francisco, CA Learn more at comphealthed.com/Momentum


COMING SOON! The LadyBugsSM program empowers women ages 16+ who are affected by, care for someone, or carry a bleeding disorder. The program’s goal is to assist women to find their voice when it comes to decision making about the health of themselves and their loved ones. Our goal is to provide education about medical developments, advocacy skill building, stress management techniques and more to encourage women to recognize that their health is equally important.

Make plans to join us for this important three-day event which will bring together LadyBugs from across the country. You’ll enjoy networking and sharing with other LadyBugs, and learn the latest medical advances, treatment protocols, and ways to deal effectively with bleeding disorders on a daily basis. National LadyBugs Women’s Summit November 4-6, 2017 Newport News, VA Learn more at comphealthed.com/LadyBugs

A Special Message from As debate surrounding the future of the Affordable Care Act continues, 100’s

of 1,000’s of Americans are at risk of losing life-saving health care assistance and need help right now. In 41 states across the country, charitable premium and cost-sharing assistance for those living with lifethreatening and chronic conditions is under threat as a result of a misguided federal policy. Many of our nation’s most vulnerable patients need your help to ensure it can continue. Visit our Action Center to urge your lawmakers to sign on to a new bipartisan letter urging Health and Human Services (HHS) Secretary Tom Price to help secure access to critical care for patients who need it most. While policymakers take time to agree on a new health care plan, patients with expensive diseases continue to face excessive premium costs and they need help now. Write your members of Congress now to help stop insurers from discriminating against patients with expensive conditions.

Visit our Action Center today to quickly and easily send a letter to your elected officials. Tell your members of Congress to sign on to a bipartisan letter calling on Secretary Price to put an end to this harmful policy– and please help spread the word about this critical opportunity to help patients in-need among your family, friends, and work colleagues. Email your legislators now – many lives depend on it. Action Center: http://bit.ly/2nOk49c -Dana Kuhn, Ph.D. President & Founder of Patient Services, Inc.


an Innovative CHES Program

About Inhibitor

About the Program Family Camp This program is specially designed to meet the needs and limitations of children with both hemophilia & inhibitors. Immediate family members are also invited because we understand that chronic illness affects the whole family. We play, learn, and grow while we build a stronger community. To register, applicants must have an active inhibitor, or a history of an inhibitor within the last 24 months, and are between the ages of 6-18. If you’re interested, visit our site July 7th to apply. Space is limited, and slots are filled on a first come, first-served basis for those who qualify. New families have first priority. To learn more or register visit: comphealthed.com/ifc, or call 781.878.8561 Presented by

2017 FALL SESSION

Date: Friday, Oct. 6th - Monday, Oct. 9th, 2017 Location: The Painted Turtle in Lake Hughes, CA Registration Opens: Friday, July 7th, 2017

See It to Experience It!

Check out our video at comphealthed.com/ifc to understand what camp is really all about.

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Supported by an educational grant from


Making

Life-LongDistance

Friendships

A Review of Inhibitor Family Camp at Victory Junction This was our first year attending Inhibitor Family Camp at Victory Junction. We were beyond excited. We really didn’t know what to expect. We knew there would be rap sessions and fun activities, but we had no idea the dynamic relationships we would create. Upon arriving, we met our awesome crew chief who stayed with us all weekend and helped us out with anything we needed. Our six year old son warmed up to her pretty quickly and was never torn about having to leave us to go hang out with her and the other children. He normally does not want to leave our side if he’s around people he doesn’t know well, so we weren’t sure how he was going to feel when we headed off to rap sessions and he had to go a separate way with the kids. However, our crew chief was so good with him, and he had no hesitation about going off to have some fun without mom and dad! This was great for us, since we hardly ever leave his side at home. We needed this time to talk and empathize with other parents dealing with the same things. The emotional and physical aspect of being a caregiver is often very overwhelming. Understandably, nobody back home – even those closest to us – really knows what it feels like on a daily basis. Being able to meet other parents, and for me other mothers, was extremely humbling. For once, we were able to be around people who really, truly get it. This was the biggest takeaway for me, personally. I needed desperately to find that connection. I was able to make lifelong friendships and find people to connect with, even if hundreds of miles away. In the same way, our son was able to finally meet other boys going through the same things he does each day. He was able to do fun activities and participate in things we wouldn’t normally do back home. All of the boys were so welcoming and made him feel like they had always been friends. During infusion time, our son was able to build up enough courage to stick his dad for the first time. Some of the older boys rallied around him and gave him their support, which was so cool to watch. These kids shouldn’t have to worry about things like this, but they embrace it so well and run with it, and then encourage the others to run with it as well. Amazing.

By Ashley Davis

We are so grateful for the opportunity that CHES provided us to attend Inhibitor Family Camp. Without their generosity and willingness to host, we would not be able to do something like this. They made it such a special weekend for us. We each had our own personal takeaways, as well as family experiences to take home. Thank you CHES!

COMMUNITY CHATTER


By Tom Joseph, Account Manager - Hemophilia and Bleeding Disorders

Dedicated to Making a Difference in the Lives of People with Bleeding Disorders

Proving that personalized care is not lost…

One Patient at a Time.

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llCare Plus Pharmacy is a specialty pharmacy leading the industry by offering top level service. We have a dedicated team of professionals whose sole focus is the wellness and happiness of our patients. We offer extensive patient benefits research, expert clinical knowledge, assistance with copays & funding, delivery notifications, monthly wellness checks, and more. Each of our patients receives a custom plan of care to make their treatment as simple and as effective as possible. One of our providers shares, “AllCare Plus has always provided the most consistent and outstanding service to our practice and our patients. We are pleased to have worked with them and look forward to working together in the future.” We are dedicated to helping individuals living with Hemophilia, Bleeding Disorders, and other chronic medical conditions – empowering them with independence, knowledge, and a high quality of life. As a specialty pharmacy, we are able to make good on our belief that service should be tailored to each person’s

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needs. We really care about the wellbeing of our patients; they are the reason AllCare Plus Pharmacy exists. Whether you receive your medications delivered by our trained drivers, or mailed via UPS, AllCare Plus guarantees that you will be welcomed and cared for. AllCare Plus Pharmacy specializes in medication management of complex diseases. Before we start treating a patient, we review the severity of the condition, medications prescribed, and their health insurance plan. Our pharmacists are trained to help patients simplify drug treatments. Upon review, we develop a unique plan of care for the patient. This plan is shared with the patient and their provider to ensure proper communication. AllCare Plus Pharmacy works with all major insurance companies. We have strong, long-standing relationships with many of the leading charitable organizations as well as drug manufacturer support programs. We will work on your behalf to make sure financial barriers are not impeding your access to care.


One Mom shares, “Working with Tom and the team at AllCare Plus has been a phenomenal experience. Before AllCare Plus Pharmacy I did not believe myself and family were considered a priority. My family has been taken care of with the upmost professionalism and the team has answered any insurance questions that I have encountered. I have been surprised with the increased one-to-one hemophilia service we have been receiving from AllCare Plus and would recommend anyone with hemophilia to give them a try and see for yourself firsthand. Thank you for your service, we wouldn’t know what to do without you!” Our Pharmacists are clinically trained in your specialty medications. We are here to support your questions at every step of treatment. We are dedicated to helping our patients succeed in therapy. We will call you on a regular basis to offer support and remind you of medication refills before you run out. Our vision is to continue redefining the limits of specialty pharmacy services. Just when people think that there is no service left in pharmacy, AllCare Plus will gladly show them how great we treat our patients and clients. We will continue to innovate new services and service combinations to better help our community and increase efficiency. Here at AllCare Plus, we all share the same core values; Passion, Integrity, Professionalism, Assertiveness, Empathy and Teamwork. No core value is more important than the other. All core values are equally essential for providing the best care possible. This is what enables us to keep growing as a company and what allows us to serve more and more patients across the nation. We look forward to what the future holds and will continue to fight the good fight alongside our patients, providers, payors and manufacturers.

AllCare Plus Pharmacy Phone: 1-855-880-1091 Fax: 1-844-265-0265 www.AllCarePlusPharmacy.com Tom Joseph Account Manager Hemophilia and Bleeding Disorders AllCare Plus Pharmacy Inc. Phone: 309-645-8215 Fax: 508-459-3534

SPOTLIGHT


Infusion Strife? Productive Life.

T

by Eric Lowe

he demands of managing a chronic condition are absorbent on your time. I get it. As patients or caregivers, we have jobs and/or school (sometimes multiple jobs just to make it), meals to fix, dishes to wash, laundry to do, baths to give, kids to put to bed, and before you know it, it starts all over again. Who has time for anything else!?

patient, I only do so when the car rides align with that time.

I started my second attempt at ITI (immune tolerance therapy) in 2008, so I’ve been self-infusing everyday for the last nine years. We are an adaptive group to say the least; we have to be. I don’t claim to be an expert on this, and I certainly don’t believe I have it worse than anyone else. But with this experience I’ve learned to condense and even multi-task during the infusion process, so I thought I’d share with you some tips and techniques I’ve developed for myself.

After I’m finished using my tourniquet, I just loosen it rather than completely removing it. Once I finish infusing and the needle is removed, I slide my tourniquet down my arm to cover the gauze and infusion site. I tighten it up, and I have complete freedom to accomplish something else during the compression time.

Start an Assembly Line During the mixing process, I tend to mix all of my vials simultaneously. So I unpack all of the factor boxes first. Then I pop all of the tops off of the bottles, etc., etc. rather than mixing up one box/vial at a time from start to finish. Although the time difference isn’t significant, I’ve found that the assembly line approach is quicker according to my time trials. Perhaps it’s slightly faster because I spare my brain from shifting gears on what I’m doing throughout the entire process. The One-Armed Man This concept is somewhat particular on the infusion location. Hands and forearms are ideal places. Once I’ve placed my I.V., I maneuver my syringe to the same hand that the needle was placed in. This frees up an arm allowing

me to multi-task. So I can begin cleanup, fixing breakfast, etc. during the infusion process. And in most cases, I use a little tape for security purposes.

The Big Squeeze

Hands-Free Some specialty pharmacies, such as mine, offer free compact infusion pumps. I’m infusing my feet more and more these days just to preserve my veins elsewhere. Hook the pump up and I’m ready to get some work done with two free hands from a chair, or eat breakfast (or dinner, if I were to infuse in the evenings.) No-Clutter Workspace I keep a baggie nearby for constant trash dumping. My specialty pharmacy bundles my factor in small bags, so it’s pretty convenient to use the bags they provide to keep a trash-free workspace. Now it’s easier to scan the table for that alcohol swab that I need.

These are my methods of saving time and fitting the infusion process into my busy days. I’m not suggesting that our readers try them, as I am not a medical professional and I would not, could not, should not provide any medicalrelated advice. But I’m hoping at least one of these leaves you feeling more enlightened than before, and it sparks your mind to think about how you can optimize your time during any healthrelated task!

The Hitch I.V.ers’ Club This one is almost not worth mentioning for lack of originality, but I do it quite frequently and I’m sure that most of you do too. I rarely turn down the opportunity to infuse in the car. But I am a morning-infuser, so as a compliant

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For entertainment purposes only. See full disclaimer on page 3.

FUN & INSPIRATION


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FEATURE


by: Emily Coe and Stacy E. Croteau, MD, MMS

Clinical Trials 101 Understanding the Process and the Terminology

The Clinical Trial Process

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efore drugs and therapeutic devices are made available to individuals to treat a disease or medical condition, the treatments are subjected to a rigorous clinical trial process in an effort to test both safety and effectiveness. Clinical research study is an umbrella term that includes a variety of different types of patient-focused studies. Interventional trials (including clinical trials) involve making an intervention to typical patient care such as the introduction of a new drug or device. Observation research studies are also important in helping us learn more about diseases and clinical care, but in these studies researchers typically collect data on participants. This can include information about medical treatments received, disease outcomes overtime, and may include surveys of patient’s report of quality of life or disease burden.

To initiate a clinical trial, a sponsor, typically a drug manufacturer and a medical researcher associated with a hospital/academic institution, needs to decide that there is evidence (data) that a drug may be of clinical use for a specific disease or group of diseases. In order to obtain permission to proceed with studying this drug in humans, the sponsor must get approval from the Food and Drug Administration (FDA) and file an Investigational New Drug (IND) application. This application includes several components such as a Study Protocol and Investigator’s Brochure (IB). The Study Protocol details the sponsor’s plan to study the new investigational (experimental) drug or therapy while monitoring its safety and effectiveness. The IB describes the pre-clinical studies that investigate the chemical properties of the drug, as well as the results of animal toxicology studies and any in-human study data to date. The FDA then reviews this application to ensure that potential participants are not at an unreasonable risk. If granted permission to proceed, the sponsor will then identify study sites (medical centers) to carry out the clinical trial. The sponsor manages all of the primary clinical operations, ensuring proper data management and coordination across all clinical sites so the study protocol is followed correctly. The study sites identify patients who may be eligible to participate in the clinical trial, and if patients enroll on the clinical trial, the study site serves as both a treatment team as well as an intermediary between the participant and the sponsor. This protects the individual’s privacy and ensures that the highest standards of medical care are maintained during participation on the clinical trial.

FEATURE


The purpose of a clinical trial is to advance the current standard of medical care and to offer new and potentially more effective treatment options in a safe and objectively monitored setting. As a drug progresses in development, it moves through different phases of clinical trials as described in more detail next.

Key Terminology of Clinical Trials The selection of study sites is a joint decision between the sponsor and the medical center. The medical center must decide if they have any patients who might be eligible for the specific study protocol, whether they have any trials already open that might compete for the same type of patients, and whether they have enough research team personnel to open the trial. The sponsor determines the

number of participants needed to complete their study and selects study sites by the number of patients they anticipate, may participate, and the presence of research infrastructure. Before a clinical trial can open for enrollment at a study site, the study protocol and informed consent form must be reviewed and approved by the local/site Institutional Review Board (IRB). The IRB is charged with protecting the interests of human subjects. This group carefully reviews the study protocol and other documentation from the sponsor including pre-clinical data and, if available, clinical data, which describes the known safety, toxicity and effectiveness of a product in animals and healthy human volunteers. Key components of the study protocol include the purpose of the study, the scientific rationale, the specific disease group and corresponding patient

Institutional Review Board

Sponsor

Study Sites (Medical Centers)

Eligible Patients

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population, and the patient monitoring plan. To be eligible for participation, a patient must meet the inclusion and exclusion criteria provided by the sponsor. The inclusion criteria define the disease and age group of interest while the exclusion criteria focus on specific health states that may put patients at unreasonable risk for receiving the study drug (i.e. poor kidney, liver, or heart function). Once the study has been approved by the site’s research team and IRB, the research team can begin recruiting patients to participate. Upon identifying eligible patients, the research team must approach the patient in order to discuss the research and obtain consent for participation in the study. An informed consent is a document that describes the details of the study, the purpose of the study, the assessments required during the duration of the study, and any known side effects of previous participants; all of which needs to be written in common, plain language. The informed consent must provide all details about what is required by a study participant including the potential risks and benefits of the study. This allows a patient to make an informed decision before agreeing to participate. A signed informed consent is required to participate in a research study.

Furthermore, if the patient is under eighteen years of age, the parent or legal guardian must sign the consent form. However, the patient must still be part of the informed consent process and sign an assent form to participate. Despite signing an informed consent document, a participant can decide to discontinue study participation at any time or can be removed at the investigator’s discretion. This may occur if the participant develops significant side effects during the study, feels the study is too much of a commitment, or is not following the study requirements (such study visits, questionnaire completion or adherence to medication regimen). Patient safety and patient choice are paramount concepts in clinical trials. Throughout the clinical trial a participant will interact directly with the study team. This group of individuals, typically consisting of a physician, or principal investigator when leading a research team, and a study coordinator or study nurse, will facilitate and participate in study visits, ensure all required tests and surveys are completed, and help to schedule other study visits or procedures as needed.

FEATURE


Phases of a Trial Clinical trials are typically conducted in phases. Each phase builds upon the data provided by the prior phase and seeks to answer a different question. Phase I studies are focused on safety and identifying the safe dose range of a drug. This is usually conducted in a small group of individuals and is designed to see how the drug reacts in the body. A placebo is not used in a Phase I study. If the drug is determined to be reasonably safe, the study will continue to Phase II.

Phase II studies expand to include a bigger group of participants. Each phase reports side effects of the participants and weighs the adverse events against the potential benefit to determine if it’s safe to continue testing the drug.

Phase III studies compare the safety and effectiveness of the study drug to the current standard of care. Phase III participants are usually randomized to receive study drug compared to a standard of care regimen or placebo. To ensure that the desired potential effect can be detected, these studies tend to be the largest studies. The study could be blinded (participants don’t know if they are receiving study drug or placebo), double-blinded (neither participant nor investigator know) or open label (everyone knows).

Phase IV studies, also referred to as post-marketing studies, may also be conducted following the drug’s approval in order to gather more data about safety. Each phase of a clinical trial has specific eligibility criteria to meet their goals for the study. Some patients who may be interested in participating may be excluded (unable to participate) from a trial based on the eligibility criteria. The inclusion and exclusion for a research study is very strict with the intention of clearly defining the patient group to be included and protect patient safety.

Pros and Cons of Clinical Trials

When considering a clinical trial, both the advantages and disadvantages should be discussed in detail with the study team before deciding to participate. These topics can be discussed with your other medical providers, family members or friends. Table 1 outlines some of the pros (benefits) and cons (risks) of participating in a clinical study; these will differ by the clinical trial phase.

Pros

Cons

Access to a drug that has shown potential benefit before it is commercially available; often years ahead of the drug being available.

The potential benefit may not be significant or well known; the new drug may not have greater benefit than current medical treatment.

Experience benefit of the drug; assume there are risks with most drug including those commercially approved.

Extent of risk or side effects are not entirely known and are always possible.

May help improve knowledge of disease and treatment for future patients, despite potential of receiving placebo rather than study drug.

Participation in a research study may require frequent site visits and may be inconvenient/disruptive to your schedule.

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Special FDA Designations The clinical data gathered during the different phases of clinical studies are prepared as part of the submission to the FDA. This application, the New Drug Application (NDA), is submitted once the drug manufacturer or researcher believes the data is robust enough, proving safety and effectiveness of the drug, for market approval. The FDA has implemented various pathways to expedite drug development and review for serious/life threatening conditions or unfulfilled medical needs. This can be requested by the sponsor if the drug has the potential to be lifesaving or life changing, which ensures the drug is available to the public as quickly as possible.

Fast Track Designation If the drug treats a serious/life threatening disease or is believed to fulfill an unmet need for a population, the sponsor can request consideration for a Fast Track Designation. Nonclinical or clinical data from clinical trials is needed to support this request. If granted the FDA will review the application within a few months of submission. This enables a drug that has potential to be lifesaving or life changing to be available to the market for its intended population as quickly as possible.

If Product Treats: 1. Serious condition 2. Life-threatening condition 3. Fulfills unmet need

=

Breakthrough Therapy Designation Similar to fast track, a Breakthrough Therapy Designation is granted to drugs that demonstrate significant improvement in clinical outcomes in a serious disease compared to available therapies with preliminary clinical data.

Product displays significant improvement in trials

=

Accelerated Approval An Accelerated Approval can be requested if the drug or therapy is shown to have a clinical benefit or advantage over current therapies. This process allows a pharmaceutical company to market the drug to the indicated population, while continuing to conduct the research study. The FDA may grant a Priority Review when filing the NDA if the clinical data demonstrates a benefit for a serious condition.

Has clinical benefit over current therapies

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Orphan Drug Status is another incentive for drug development in rare diseases, like hemophilia. This designation provides additional support from the FDA, which includes tax incentives and grants that offset the costs and allow for market exclusivity for seven years following approval. The approval process is also expedited.

FEATURE


Current Clinical Trials in Hemophilia More than 100 clinical trials are currently open and listed on clinicaltrials.gov for patients with hemophilia. These studies, as well as dozens that have been completed in the past few years, reflect a potential transformation in our approach to hemophilia treatment. Although routine (preventative) factor replacement has dramatically improved health outcomes and reduced debilitating, chronic joint damage in this population, a number of challenges remain with the use of factor concentrates to treat hemophilia A and B. Reliable intravenous access, the frequency of infusions necessary to maintain desired factor levels, and development of neutralizing alloantibodies (“inhibitors�) are among the biggest barriers. New hemophilia therapies aim to improve on our current approach of factor replacement in a number of different ways. Some products extend the circulation time of factor protein [extended half-life (EHL) products], others attempt to introduce the genetic material (DNA) necessary to allow the body to produce the missing factor protein [gene therapy, gene editing], and still others take innovative approaches to either mimic the function of the missing factor VIII protein or disrupt the protein responsible for regulating the formation of blood clots. A reduction or elimination of the need for intravenous factor concentrate or the option of a subcutaneous injection rather than an intravenous infusion are exciting prospective therapeutic options for both hemophilia patients with and without inhibitors.

Extended half-life (EHL) factor concentrates Several EHL factor VIII and IX concentrates have completed clinical trials and have been approved in adults and older children. The efficacy, safety, and immunogenicity (inhibitor risk) of these products continues to be investigated in our youngest hemophilia patients (previously untreated patients, PUPs). Modifications have been made to the factor VIII and factor IX proteins to extend the length of time they circulate in the blood and are available to participate in blood clot formation. The precise modification made to prolong the circulation time of the factor depends on the specific product.

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Despite evidence that these products do not seem to increase the risk of inhibitor development in patients who have never had an inhibitor previously and have had more than 150 exposure days to a prior factor product, studies to investigate the risk of inhibitor development in PUPs are still ongoing, [NCT01493778, NCT02234323, NCT02615691, NCT01712438, NCT01311648, NCT02172950, NCT02137850, NCT02053792, NCT02141074]. Additionally, researchers are also exploring whether these new products can help achieve faster and overall more successful immune tolerance for patients with inhibitors [NCT02196207].

Use these identifiers to locate each study on clinicaltrials.gov


Gene therapy and gene editing Hemophilia A and B are ideal candidates for gene therapy or gene editing because the deficiency in factors FVIII and FIX arise from a defect in a single gene. A small increase in the body’s ability to make these clotting proteins can reduce a patient’s bleeding symptoms. Most of the current gene therapy programs are investigating the safety and efficacy of gene therapy in adult hemophilia patients without inhibitors; however, potential opportunities for gene therapy/editing in patients with inhibitors using cell-based gene therapy and editing techniques are being pursued, [clinicaltrials. gov: NCT02576795, NCT02484092, NCT02618915, NCT02396342, NCT00979238, NCT02695160, NCT03003533].

Nonfactor Replacement Therapy Therapeutic products in this category fall into two general types: 1. mimicking the co-factor function of factor VIII or 2. blocking or reducing the production of proteins responsible for decreasing clot generation (maintaining normal balance to prevent excessive clotting) Emicizumab (ACE910) mimics the function of factor VIII and is presently in phase 3 clinical trials for both hemophilia patients with and without inhibitors, [NCT02622321, NCT02795767, NCT02847637]. While early data has demonstrated a remarkable decrease in bleed events, particularly for patients with inhibitors, recently reported blood clot adverse events, which arose in the setting of concomitant use of bypassing agents, have increased scrutiny of this agent. There are several investigational drugs that block or reduce anti-coagulation protein, namely tissue factor pathway inhibitor (TFPI) inhibitors (concizumab and BAY 1093884) and blocking production of antithrombin (AT) (fitusiran). TFPI plays an important role in regulating the initiation of thrombin generation by inhibition of tissue factor-factor VIIa (TF-FVIIa) and prothrombinase. For patients with hemophilia, amplification of coagulation signaling and the thrombin burst is inadequate. Products designed to block the function of TFPI are in ongoing phase I clinical trials, [NCT02571569, NCT02490787]. Both IV and SQ formulations are being studied. Antithrombin plays a direct role in inhibiting thrombin production and decreases clotting activity of other coagulation proteins. A phase I dose escalation trial investigating weekly and monthly infusion schedules of fitusiran is underway, NCT02035605. Early results show a decrease in AT levels and a corresponding decrease in annualized bleed rates and no safety concerns in the small number of patients studied so far.

While it is exciting to have so many new therapeutic products under investigation, as we discussed above, large clinical trials to explore the effectiveness and safety of these products are imperative. Early phase studies can have encouraging results, but then expansion to a larger pool of patients can demonstrate that an investigational product is not as effective as initially thought or that there are important safety considerations that were not previously identified. Until a drug is reviewed and approved by the FDA, it is important to remember that the products described above are investigational in nature and may or may not become commercially available for individuals with hemophilia. In many cases, ongoing surveillance for safety is important even after a product is approved by the FDA for use. To learn more about specific clinical trials, study sites that are actively enrolling patients, and the general inclusion and exclusion criteria, please see clinicaltrials.gov. Editor’s note: On May 15, 2017, in Hemophilia News Today, Dimension Therapeutics announced they would end development of DTX101 as Gene Therapy for Hemophilia B.

FEATURE


Glossary of Helpful Clinical Trial Terms Adverse Event: Any negative change in the health of a clinical trial participant’s health that occurs during or for a specified period after the trial. Antibody: A protein secreted into the blood stream to neutralize pathogens, including bacteria, viruses, or foreign proteins. Antibodies may be designed to interact with specific proteins for therapeutic purposes. Clinical Trial: A process of testing the safety and efficacy of a new drug in people. Divided into four different “phases� (see below).

Cohort: A group of clinical trial research participant who share a characteristic of interest. Double-Blind: Both the research participants and investigators do not know the natire of the intervention being administered to specific patients. A third party reveals which group received which intervention after the outcome of the trial has been assessed. Used to prevent bias when the drug being tested is compared to placebo or another approved drug.

Exclusion Criteria: Attributes that prevent an individual from participating in a clinical trial. Determined before the trial begins and cannot be altered. These are designed to keep research participants safe. Gene Therapy or Editing: Inserting a functional copy of a gene into a patient to treat a disease. Half-life: The amount of time it takes for the concentration of a drug to decrease by half. Inclusion Criteria: Required attributes for individuals to participate in a clinical trial. Determined before the trial begins and cannot be altered. These are designed to define the type of patients being studied and to keep research participants safe. Investigational New Drug (IND): An application to the FDA for permission to begin testing a drug in humans. Contains results of toxicity studies and protocols for manufacturing the drug and carrying out clinical trials. Required before a drug can enter phase clinical trials.

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Intravenously: Administration of a drug directly into a vein. Investigators: A researcher who is involved in conduction a clinical trial.

Primary endpoint: The outcome that is measured at the end of the trial to determine if the drug was successful in treating the disease. The primary endpoint is determined before the trial begins.

Off-Label Usage: Use of an FDA approved drug for an indication, or in a group, not approved on the prescription label.

RNA Interference: The process in which the production of a specific protein is decreased by an RNA molecule. RNA is a chemical “cousin” of DNA.

Open-Label: Both research participants and investigators know the nature of the intervention being administrated.

Single Blind: The research participants do not know the nature of the interventions being administered. Used to prevent bias when the drug being tested is compared to placebo or another drug.

Peak Level: Maximum concretion of a drug in the bloodstream of a patient after administration of one dose. Phase I: A clinical trial in which a drug is tested on research participants to determine the maximum tolerated dose of a drug and to evaluate safety. Phase II: A clinical trial in which a drug is tested on a small group of patients to establish whether a drug has any efficacy and further test the safety of the drug. This phase can only begin after a phase I trial has been completed successfully. Phase III: The phase of testing for a drug can be approved. In phase III trial a drug tested on a large group of patients to further establish the efficacy of a drug in treating a disease. The data from this trial is reviewed by FDA in hopes of approval. This phase can only begin after a phase I trial has been completed successfully. Phase IV: Continued evaluation of a drug for safety and efficacy after it has been approved for marketing. It is also known as post-marketing surveillance. Does NOT evaluate the drug for new indications.

Study Arm: A group of research participants that receives a specific type of intervention. Includes the drug being investigated, a placebo control, or a previously approved treatment for the same condition. Subcutaneous: Administration of a drug under the skin. Top Line Results: The results of a statistical analysis of clinical trial data that indicate if endpoints have been met. Examples may include showing an experimental drug is statistically superior to another already approved drug, or that an experimental drug shows no statistical difference with a placebo. Trial Sponsor: The person or organization who initiates a clinical trial and holds the investigational new drug application. May be a governmental organization, a corporation, or an individual investigator. Trough Level: minimum concentration of a drug in a patient’s bloodstream.

This glossary was created for New England Hemophilia Association for the first of its kind, consumer medical update offered on March 25th 2017. It was created by the Consumer Medical Update Committee to help understand key terms used for clinical trials. www.NewEnglandHemophilia.org

BLOODLINES


Living with

by Dr. Gary McClain, PhD

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UNCERTAINTY

Isn’t Easy

“I’m having one of those days when the world feels like it’s crumbling around me. I know you’re supposed to be able to live with a certain amount of uncertainty when you have a child with a chronic condition. But we’ve got some decisions to make about her care, and the outcomes aren’t guaranteed. This kind of uncertainty really takes a toll on parents. What do I do?”

Here’s Help

My client described how a lot of patients and their loved ones feel when they are placed in the position of having to make a health-related decision. Living with a medical diagnosis – yours or a member of family – means being aware of the importance of making informed and thoughtful decisions. But what do you do when everywhere you look uncertainty seems to be staring back at you?

• “I’m so afraid that…” • “If I could just get some kind of a guarantee…” • “What if…” Ever catch yourself using one of these phrases? If you do, I suspect it’s during one of those times when you’ve got a hard decision to make, you want a clear direction forward, and all you see around you is shades of gray. Yet again that unwanted house guest in your home – the chronic condition – leaves you at a crossroads and then demands answers!

FAMILY MATTERS


Nobody likes uncertainty. We want to know. We want answers. We want to make perfect decisions. And when we’re faced with uncertainty, it’s only human nature to allow our imagination to go to town and fill in the gaps with those stories we tell ourselves to satisfy our inquiring minds. Sure, stories at least give us something to think about, and react to, in the absence of real information. But, on the other hand, those stories are usually worst case scenarios that turn lack of information into misinformation. Are we trying to do ourselves a favor by getting prepared for the worst? It sure seems that way. But wow, we cause ourselves a lot of unnecessary pain in the process. If you’re living with a chronic condition, or have a family member who is, you’re no stranger to uncertainty. Like when you’re waiting for your latest test results. Wondering why your doctor didn’t get back to you on a question right away like she normally does. Adjusting to a new regimen and anticipating how you might be affected. Or making a treatment decision! We never get used to uncertainty, do we? Yet uncertainty is part of life. So what can we do when the urge to fill in those big information gaps with your own version of the outcome?

I may not have enough information here yet. I’m working on getting it.

Here are some ideas: Get clear on what you’re dealing with. The starting place for making a decision about the way forward is to define the specifics of the situation: The decision to be made. The options. The potential outcomes. The risk factors. You’re in a better position to make a decision when you have clearly defined exactly what that decision is. Free-floating anxiety just keeps you stuck. So be clear with yourself on what you’re dealing with. Keep antidotes for negative self-talk handy. When you tell yourself how scary and bleak the future looks, you are training your mind to focus on the negative side. Chances are that your view of reality will match your expectations. But like any poison, negative self-talk will shrivel away when zapped with your most powerful antidote – positive self-talk to balance out the negative. So, how about working on your self-talk, that internal dialogue that’s going on in your mind? You can start by talking back to that internal storyteller with a few simple facts below.

We’ve got resources to help me make the best decision possible. And to help me cope with the outcome. We aren’t alone here.

I don’t like uncertainty. But making up stories is just going to make me feel worse. While things could turn out bad, they could also turn out good. Or somewhere in between.

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Gather your fan club around you. One of the lessons of uncertain times is to build a solid support system. Who are the people in your life who help you to bring out your best self, and who rely on you to do the same for them? Make sure you keep them close. Spending some time with your support network is a good way to help you keep your focus on what’s going well in your life right now. Sit down with a family member or a friend and talk about what’s going on with you. Review the options with them. You know, Plan A, Plan B. Share your concerns, your fears… your stories about potential outcomes. This helps in a couple of ways. First, you won’t feel so alone. And second, saying something out loud helps you to clarify your thinking. And even to see the direction forward more clearly. How about looking at support this way: The best way to get out of your own mind is by enlisting somebody else’s mind.

Remind yourself how you have met challenges in the past. Start your list of antidotes with your greatest successes. Don’t forget your key skills and personal qualities. Here’s one to add to the list: resilience. You’ve made decisions in the past, and you’ve done your best to make the right ones. So you know you have what it takes to face the next challenge.

While you’re at it, come up with a couple of alternate endings. For any given direction, there are any number of possible outcomes. So if you are going to create stories, then how about creating more than one? If a decision can lead to a negative outcome, can it also lead to a positive one? Make sure you balance each bad ending with a positive one.

Limit yourself to a few minutes of storytelling each day. Can’t quite let go of that need to indulge that need to create your own yarn? Okay, then. Give in. But on a schedule. Allow yourself to sit with your anxious stories for fifteen minutes, maybe twice a day. Time yourself. At the end of the fifteen minutes, put the crystal ball away and get back to the present.

Get informed. After you’ve identified exactly what it is that’s making you feel uncertain, identify resources you can tap into if needed. Some information-gathering might be in order here, on your own, or by reaching out to people who can give you advice. Sources might include the Web, your insurance company, healthcare professionals, and other families you have met through your support network. Flood the fear with facts!

FAMILY MATTERS


Speak up. When you need information, ask for it. If you can’t get the information you need, ask why and when. Talk to your healthcare professionals about what you’ve learned. Communicate to professionals the potential outcomes that concern you. Do everything you can to advocate for yourself and your loved one. Is it time to do the numbers? Your uncertainty may involve financial concerns. And money is always pretty scary to think about. Take a hard look at what you’re spending each week for medical-related expenses. Consider what expenses you might have in the near future. While all those numbers may not give you a warm and fuzzy feeling, at least you have a clear picture of what you’re dealing with. That’s another toward feeling more empowered. Ask for some help from the other people in your household who are involved in making financial decisions. After all, you’re a team.

Watch your self-care. What’s going well in your life? What are the other responsibilities in your life that need your attention? Eating healthy? Getting enough rest? Getting support? Balance out the uncertainty with what you can count on – and control – in your life. In other words, maintain your perspective! Your mind will be that much clearer. Embrace your Higher Power. Believe in something beyond the dayto-day setbacks. Your Higher Power can be found through a spiritual or religious practice, or it may be found in simply trusting in your own inner strength for strength and guidance.

struggle to be in control. You can’t go back and fix what you did or didn’t do in the past, you can’t control the future, and you certainly can’t control what anybody else is doing. The best you can do is to do your best to make the best decision possible. Life is uncertain. The answers aren’t on our schedule. Take charge of your inner storyteller by being patient and seeking real information. When it’s time, you’ll know. And then you’ll harness your resources and face the outcome head on. Like you always do!

Remember: Nothing is guaranteed, except this moment in time. You may not want to hear this, but here’s some tough love for you. Give up the

Gary McClain, PhD, LMHC, Dr. Gary McClain, PhD, is a therapist, patient advocate, and author, specializing in helping clients deal with the emotional impact of chronic and life-threatening illnesses, as well as their families and professional caregivers. He works with them to understand and cope with their emotions, to learn about their lifestyle and treatment options, to maintain compliance with medical regimens, to communicate effectively with the medical establishment, to communicate better with other family members, and to listen to their own inner voice as they make decisions about the future. He writes articles for healthcare publications and websites, facilitates discussions in social health communities, and conducts workshops on living with chronic conditions and Chronic Communicationssm. He maintains a Website, www.JustGotDiagnosed.com.

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FAMILY MATTERS


The Timeline of Education: A New and Uncertain Turn? by Lisa Cosseboom, M.Ed. & C.A.G.S School Psychologist & Special Education Evaluation Team Chairperson

Important Events in Public Education: April 11, 1965: President Lyndon B. Johnson signs the Elementary and Secondary Education Act (ESEA) which expanded the federal role in k-12 education. Title 1 was implemented that enabled the Federal Government to assist with providing funding to school districts to help disadvantaged students. 1968: Congress expands on ESEA to provide programs for immigrant children, neglected children and passed the Bilingual act. 1973: The Rehabilitation Act becomes law in which the Section 504 ensures civil rights for people with disabilities requiring school districts to accommodate for students with disabilities to access buildings, programs and activities. 1974: Equal Educational Opportunities Act passes. This Act requires school districts to take action and overcome barriers which would provide equal protection for students. 1975: The Education of All Handicapped Children Act (PL-94-142) becomes Federal Law. This law provided that handicapped children and adults ages 3-21 be educated in the “least restrictive environment” to the maximum extent appropriate, meaning that they are educated with children who are not handicapped and that special classes, separate schools or other removal of children from their regular educational environment, occurs only when the severity of the handicap is such that education in regular classes cannot be achieved. 1978: President Jimmy Carter reauthorized ESEA and changed Title 1 rules allowing schoolwide Title 1 programs when 75% or more of the students are low-income. 1979: President Ronald Regan reauthorized ESEA and changed funding into one block grant and reduced regulatory requirements by states. 1988: Student testing and accountability takes hold and regulations require districts to test annually and to create improvement plans.

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1989: President George Bush Sr. – Education Summit with Governors. Developed National Education Goals with federal/state partnerships for accountability. 1990: Individuals with Disabilities Education Act (IDEA) amended PL 94-142 to change terminology of “handicap to disability” and added mandates for transition services, and added Autism and TBI (Traumatic Brain Injuries) to the eligibility list. 1994: President Clinton reauthorized ESEA for states to develop standards and align tests for all students. This reauthorization included increased funding for bilingual and immigrant education and requirements for charter schools. 2001: (Public Law 107-110) was signed into law in 2002. The Act requires states to develop basic skills assessments to be given to all students in certain grades, if those states are to receive federal funding for schools. The Act does not assert a national achievement standard; standards are set by each individual state. 2004: A change was made to IDEA (H.R. 1350) which more closely aligned IDEA with No Child Left Behind. 2009: President Obama launched a reauthorization of ESEA that was the largest federal investment in public education. This 115 billion dollar investment was to “help local school districts avoid layoffs and program cuts, boosts funding for special education and programs for disadvantaged students, and offer the prospect of funding for school repairs and modernization, among other elements.” (Education Week, February 12, 2009) 2010: President Obama’s Blueprint for Education was released. It emphasized implementing high standards for students and educators by passing state tests and introduced Race to the Top for states to compete in grants to help reform schools. 2015: Obama’s Every Student Succeeds Act (ESSA) passed that was a reauthorization of the 50-year-old ESEA. This act allowed for states to have more control in the education process but continued to have protections for disadvantaged students, students with disabilities and ELL’s (English Language Learners, who are students that are unable to communicate or learn well in English.)

WHAT’S the PLAN?


Recent Education Bills Passed: House Joint Resolution 58: Passed House on 2/7/17, Senate on 3/8/2017: Rejected a Department of Education regulation that imposed a new federal standard for the education and preparation of teachers which linked teacher preparation to eligibility for federal grants. House Joint Resolution 57: Passed House on 2/7/2017, Senate on 3/9/2017: This resolution overturns a regulation by the Department of Education that placed federal restrictions on systems developed by states to hold schools accountable to parents and taxpayers for their performance.

New Bills introduced:

What is H.R. 610? Termed “Choices in Education Act of 2017” according to Congress.gov, this Act “Repeals the Elementary and Secondary Education Act of 1965 (ESEA) and limits the authority of the Department of Education” to nothing but the power to award block grants to qualified states.

The repeal of ESEA would essentially eliminate every education act noted in the timeline... including Every Student Succeeds Act (ESSA) under Obama and No Child Left Behind under Bush. The more recent ESSA promotes equality in education and provides federal protections for disadvantaged and disabled students. The block grants would distribute federal funding to eligible states to award in the form of vouchers for eligible students

to use in school choice. Additionally, this Act repeals the rules surrounding established nutrition standards (availability of fruits, vegetables, reduction of sodium etc.) for the national school lunch and breakfast programs. This Act was introduced to the House Committee on Education and the Workforce on 1/23/2017.

Thinking behind H.R. 610: Proponents behind the Choices in Education Act believe that this Act would provide better competition between public and private schools and therefore increase the quality of public education through the spirit of competition. They feel that disadvantaged students would have access to private or religious schools through the voucher system where they would not have had access freely because of their poverty levels. Families who tend to be financially well-off, tend to live in identified communities that are known for quality education, or can afford to send their children to private schools.

Nuts & Bolts of Choices in Education Act: Title 1 is federal funding provided to public school systems based on the number of disadvantaged students that are enrolled in their districts. The funds are distributed in public schools to assist in educating the disadvantaged. H.R. 610 would remove Title 1 funds to public schools and transform this funding into the voucher system that would follow an eligible child to whatever school they are attending (including religious, private, charter schools or students being home-schooled). Many states have laws in place separating church from state and the voucher system could allow for public funding to be funneled to religious organizations. The proposal is assuming that states would contribute approximately 110 billion dollars into the voucher system which would ultimately provide approximately $12,000 per year to each student who qualifies for the voucher system. Additionally, the vouchers are geared towards students whose family fall in the poverty level and may not cover the complete cost of private schools and would require the

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families to pay the difference. The very families that have met poverty levels. There are a lot of proposals on how this voucher system would work, but none seem very clear. States hold local control over education policy and regulation, and would need to “buy-in”, literally and figuratively, with the federal proposal. The Act appears to be the beginning of an attempt to privatize education and dismantle public education. Essentially, it reallocates federal funding leaving public school districts scrambling to make cuts and find funding. Voucher programs have existed on a smaller scale in this country for a long time. Some of the research points to inconclusive or contradictory results. Some states have reported better success of student on vouchers in testing and graduation and some have noted no increase in testing scores or graduation. The second component of H.R. 610 introduces the “No Hungry Kids Act.” While on the surface this seems like a positive Act, it is actually removing the previous “Healthy, Hunger-Free Kids Act which purpose was to improve child nutrition through the school lunch and breakfast programs. The Healthy Act was requiring the schools to increase

availability of fruits, vegetables, whole-grains, low-fat milk and reduce levels of sodium, trans-fats and saturated fats in school breakfast/lunch The prosed act would prohibit the USDA from rationing calories to children and remove the previous Healthy act requirements.

Effect of H.R. 610 Special Needs Students: The Elementary and Secondary Education Act of 1965 has been reauthorized every 5 years since its inception and has changed names several times. Whatever name it was at a given time did not matter to special education students, as it always provided protection. The ESEA provided that schools who receive federal funding must provide support for students with disabilities. Under the new proposal, federal funding in the form of grants, would not require the schools receiving the vouchered students to provide services. Private and religious schools are not required to provide special education services. Removing funding from public schools that offer a spectrum of services and place it in the hands of private or religious schools further impacts public education and decreases funding for special education services.

Summary: If H.R. 610 passes, essentially it removes federal funding to public education and repeals the ESEA which protects programs for special education students, students in poverty, gifted students, ESL programs, rural education and school safety. Providing a free and appropriate education to all and ensuring special education students access to the curriculum and accommodations will cease to exist. The goal of H.R. 610 is to privatize education and defund public education. To dismantle fifty years of progress in education and rely on inconsistent research of school vouchers is a dangerous path and may leave many disabled, under-privileged and middle-class students abandoned.

WHAT’S the PLAN?


Clinical Trials: Trials: Optimistic Optimistic Caution Caution Clinical by Janet Brewer, M. Ed

“Somewhere, something incredible is waiting to be known.” - Carl Sagan

I

t is an incredibly hopeful time in the bleeding disorders community! Longer acting products, gene therapy, and subcutaneous injections all seem to be within our reach. The plethora of new treatments truly boggle the mind. Keeping up with the changes of names for manufactures alone is bewildering! Baxter became Baxalta, which then became Shire. Biogen became Biaverativ, Emergent became Aptevo. While multiple new companies are offering emerging therapies in our community such as Spark, Dimension and uniQure. Deep hope has been ignited again for the first time since recombinant product became available. Treatment promises that may yield fewer infusions per month, subcutaneous injections could make self-infusion and ports a thing of the past. Gene therapy has significantly prolonged FIX activity levels, which substantially increases the length of time one may be infusion-free. Each advancement comes with the opportunity to participate in a clinical trial. This decision however, comes with excitement, responsibility,

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uncertainty and at times - false hope. Hemophilia has been in my family for over 7 decades and I have heard “a cure in our lifetime” since 1970. I have personally seen the excitement and uncertainty as we seek new treatment that will make our lives more predictable, better, maybe even…. normal? When carrying my first son in 1988, knowing that I was a carrier, all I knew was that I was carrying a son and he had a 50/50 chance of having hemophilia. Like every newborn, he was miraculous! A preciously beautiful son who looked normal in every way. I dared to hope that he didn’t have hemophilia. Three days postpartum, cord blood results indicated that he did in fact have severe FVIII deficiency. As a family member of one affected by hemophilia, at least I had some knowledge of what life with a chronic condition might look like. Nothing prepared me for being the mom of a child with a bleeding disorder. In 1992, I was anticipating the arrival of another beautiful child who would be blessed with hemophilia. Prenatal testing had evolved by that point, that via

Spring 2017

amniocentesis, we were prepared. Once I learned that he was a boy, I knew in my heart he had it. In truth, I was grateful to have two (2) sons that shared the same disorder. Boundaries and rules would be the same for each. There wouldn’t be one who could pummel the other, while the other one couldn’t pummel them back. My salvation was that I was the one who would infuse them! “Choices have consequences” was our family motto. In 1991, while pregnant with my second son, I was approached about participating in a clinical trial for recombinant factor. He was the last previously untreated patient (PUP) in the world to be enrolled. Looking back at that time, I realized I knew very little about what adverse events could be anticipated. As a community, we were most worried about HIV and Hepatitis. The scramble to be sure that factor products held no human element of plasma was the goal. My first son was tested for HIV every year until his 6th birthday. The anxiety waiting for those results would reach a crescendo.


With a family member impacted by HIV/Hep C, my first concern was that my sons would also be affected. Signing up for a clinical trial seemed like the best possible choice I could make. Decreased risk of HIV/Hep C was paramount. The trial lasted for five years. It required multiple blood tests at a HTC nearly 2 hours from our home. I look back on that time making those long trips, multiple pokes, the fabulous nurse who bribed him with trucks-one for each hand with a sense of pride that we were doing our part to contribute to science and ground breaking research that would benefit others. Fast forward to 2002, my second son has developed an inhibitor at the age of 10. THIS was a totally different diagnosis than “plain old hemophilia”. An inhibitor was life changing for our family. Our family was now coping with the challenges of an adverse event. In under 50 years we have seen the development of factor in 1970, (a huge improvement over fresh frozen plasma or cryoprecipitate). Factor then evolved from being plasma derived to recombinant, and then multigenerational factors that eliminated any element of human blood. In that time, our community lost thousands of lives to AIDS and Hepatitis C. Those who survived, now manage as many as three chronic illnesses-hemophilia, HIV and Hep C. Recombinant factor saved my sons from HIV/Hep C, which I will be forever grateful for. Yet, in the

last 25+ years, we have seen a 25% or higher rise in inhibitors affecting those with hemophilia A and B from mild to severe. Since 2012, inhibitors are the biggest threat to our community today; finally, surpassing HIV/Hep C. Now in 2017, there are multiple new treatments in clinical trials that heightens our feelings of hope that finally, THIS might be the one to change our lives. It is both an exciting and perplexing time in the bleeding disorders community. On average it takes 14 years and billions of dollars for a new product to be developed. Choosing to participate in a clinical trial can be a challenging decision. At the 2016 Inhibitor

Summits, Dr. Tarantino’s and Dr. KruseJaress’ presented on the “Knowns and Unknowns” of current therapies in clinical trials. There is still so much we do not know when it comes to choosing to participate in a clinical trial. Choices have consequences, with positive and negative effects. If you read the adverse effects of aspirin or acetaminophen, the list is lengthy. When participating in a clinical trial,

every symptom is reported. On a trial and have a headache? It will be reported. The headache may have nothing to do with being on a trial, but it could be, so it must be documented. It is probably fair to say that every medication has side effects. This is a big decision, one that may not affect just you. As a parent, you are

deciding for your child something that will affect them for the rest of their lives. As an individual, you may have loved ones who could be affected by your decision. Only you can decide what is best for yourself or your family. It is all about making the most informed choice you can possibly make, and your ability to trust in it. So, what can you do as an informed potential participant? (next page)

WHAT’S NEW?


Clinical Trials: Optimistic Caution

(continued)

Answer: Arm yourself with as much knowledge as you possibly can.

1. What phase trial am I participating in?

12. Avail yourself of resources such as clinicaltrials.gov, PubMed https://www.ncbi.nlm.nih.gov/pubmed/, or Wiley Online Library wiley.com. Trial abstracts are available on those sites and if you would like to read the entire journal articles, ask your HTC provider for them or purchase if available.

2. What is the inclusive/exclusion criteria?

13. Read.

3. By participating in this trial, does it preclude me from ever participating in another?

14. Talk to friends, family, clergy.

Clinical trial thoughts to examine:

4. Carefully read the Informed Consent Form that contains: a. Purpose

15. Write down your questions before visiting your health care professional. They want you to make a wellinformed decision as well.

b. Details

16. Take your time. There are plenty of opportunities in the pipeline.

c. Duration

17. Trust your gut.

d. Required procedures such as lab work and how often needed

18. Read some more.

e. Key contacts

19. Be prepared that this may or may not produce the desired affects you were anticipating.

5. Is there a patient advisory committee included with this trial and may I participate? 6. What are the risk/benefits? 7. Is my current HTC an Investigational Site? If not, is there still a way I could enroll? 8. Will I be reimbursed for travel if participating in an Investigational Site far from my home? 9. Will study drugs be included free of charge? For how long? Will my insurance company pick up remaining costs of lab draws, office visits, etc.? 10. What is the purpose behind wanting to participate in this trial? a. Quality of Life?

b. A feeling of duty/determination to further research for others?

11. What are your feelings on taking risks?

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WHAT’S NEW?


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Engaging

UNCERTAINTY

by Krystyn Strother

“The quest for certainty blocks the search for meaning. Uncertainty is the very condition to impel man to unfold his powers.” - Erich Fromm

A

s uncomfortable as it may feel, uncertainty is at the foundation of our lives. We want to know, always, especially when it comes to our loved ones and our future. These unknown prospects can leave us feeling unsettled. Yet, the reality that every living being on this earth faces is one of uncertainty. Our jobs are not guaranteed, our cars will inevitably need fixing, and the sun shines even when rain is in the forecast. When we cling to an expectation of a certain outcome, we set ourselves up to suffer even more if it doesn’t happen. Uncertainty is so upsetting, causing stress and anxiety, that many of us try to avoid or control it altogether. Often, people will say that we need to cope with these feelings, but what does that really mean? How do we cope? Certainly, not by putting your head down and hoping for something different. Moving through moments of uncertainty requires engaging with it and this is where a mindfulness practice can be helpful, if not essential.

Be Confident Engaging with your uncertainty will only give you more insight and a better understanding of what you are really feeling. A better understanding of what we are feeling and why, gives us more access to our experience, and while we can’t control everything, we can learn to ground ourselves in the moment. This helps us to feel better prepared to tackle whatever comes our way. The only constant in life is that it will involve change, and try as we may to control the future, sometimes all we can do is trust that whatever happens, we can adapt and make the best of it. Uncertainty is inevitable. And no matter how hard we try, controlling it simply doesn’t work. Instead, practice acceptance, control what you can and relinquish the rest. Mindfulness, at its best, teaches us how to be open to both. Practicing mindfulness cultivates comfort with discomfort.

Mindfulness teaches us to disassemble reactionary thoughts into manageable parts and pieces. It gives us a buffer between event and reaction so that we can form a productive response. Rather than focusing on the negativity, we learn to pay attention to our experience with curiosity and without judgement. This is engagement. A sitting back in your seat when those uncomfortable feelings come up and extending a chair and invitation for them to sit down right across from you. Mindfulness The subtle art of positive, internal manipulation. There are things that you can and cannot control. For example, you cannot know what the outcome will be regarding our nation’s current healthcare debate. You can, however, take note of what you are able to control when you experience any negative or stressful thinking around this topic. You can, for the most part, control your environment. Are there ways, in these moments of stress and anxiety, that you can alter your environment to find a sense of grounding? Open a window, take a deep breath, turn on a light, or adjust your posture. When you’re thinking about a moment, you can control your response, which influences everyone around you. The problem with dwelling upon a moment that is out of your control is that you are too overwhelmed by your expectations or fears of the future that you lose sight of what is taking place in the present.

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Push to Engage Uncertainty


B R E A K

Remember to take a BREAK

REATH Bring attention to the breath and the breath at the nostrils. Follow the breath into the chest and belly. Focus on the exhalation, notice the body release the breath. ECOGNIZE Notice how you are in this moment. What sensations are here? What feelings are here? What thoughts are here? How do I want to react to this information? Clearly seeing what is already here. NGAGE Gently investigate and approach sensations or emotions in the body with a quality of curiosity and kindness. Soften to the edges of intense sensations if needed. CCEPTANCE Be willing to let whatever is already here to be present just as it is. If you have resistance to this, see if you can make room for that too.

INDNESS Bring a quality of compassion and kindness to your experience. Remind yourself that everyone needs a break now and then. Let the intention of kindness nurture you.

Krystyn Strother is the former program director at HUSH Meditation, strategic designer/author of the HUSH meditation curriculum, is a certified meditation instructor, co-founder of NOMAD, “Adventures in Wellness”, and yoga instructor.

Push to Engage Certainty

Krystyn’s yoga classes range from Vinyasa to Yin. In addition to her regularly scheduled classes, Krystyn guest teaches at several yoga teacher training programs throughout the country, speaks at conferences on mindfulness and stress reduction practices, teaches specialized workshops, facilitates yoga + adventure retreats, and conducts continuing education classes for currently registered RYTs. Krystyn holds a certificate of completion in the Yoga of Awareness For Chronic Pain, an evidencebased program sponsored by the Department of Anesthesiology at OHSU. Read more about Krystyn at krystynstrother.com

MIND BODY CONNECTION


89 E. Washington Street Hanson, MA 02341-1125

CHES Mission To Inspire awareness and selfreliance for patients with chronic health conditions, their families, and their communities.

Editors in Chief Janet Brewer, M.Ed Eric Lowe

Editor Lisa Cosseboom, M.Ed., C.A.G.S

Publication Designer Eric Lowe

Contributing Writers Janet Brewer, M.Ed Emily Coe Lisa Cosseboom, M.Ed., C.A.G.S Stacy E. Croteau, MD, MMS Ashley Davis Tom Joseph Eric Lowe Dr. Gary McClain, PhD Krystyn Strother

Contributing Materials NEHA (New England Hemophilia Association) Patient Services, Inc (PSI) Giving a contribution to support the newsletter:

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Lifelines for Health (vol 9): Clinical Trials 101: Understanding the Process and the Terminology  

With so many new products on the horizon, these are exciting times. But they can also be confusing times. Allow research coordinator, Emily...

Lifelines for Health (vol 9): Clinical Trials 101: Understanding the Process and the Terminology  

With so many new products on the horizon, these are exciting times. But they can also be confusing times. Allow research coordinator, Emily...

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