2025 Boca PFS Friday Slides by International Myeloma Foundation

2025

IMF PATIENT AND FAMILY SEMINAR

BOCA RATON, FL

MARCH 14 & 15, 2025

Thank you to our sponsors!

IMF PATIENT AND FAMILY SEMINAR

BOCA RATON

FRIDAY AGENDA

Program Evaluations

Please be sure to complete your program evaluation today. If you are only attending the Friday Program, you can return your evaluations at the end of today. If you are returning for Saturday Program, please hold onto your survey, bring it back tomorrow and turn it in at the end of the program.

We greatly appreciate your time and feedback!

The IMF Support Group Team is Here

 Melbourne Multiple Myeloma Support Group

 Meets in-person on the 4th Monday of each month at 10:30AM

 Maitland Multiple Myeloma Support Group

 Meets in-person on the 2nd Monday of each month at 6:30pm

 Ocala Multiple Myeloma Support Group

 Meets in-person on the 3rd Saturday of each month at 11AM

 Palm Beach County Multiple Myeloma Support Group

 Meets in a hybrid format on the 1st nonholiday Monday of each month at 6:30PM

 Fort Myers Multiple Myeloma Support Group

 Meets in-person on the 3rd Tuesday of each month at 6pm

 Hollywood Multiple Myeloma Support Group

 Sarasota Multiple Myeloma Network & Education Group

 Meets in-person on the 4th Friday of each month at 11AM

 Meets virtually on the 2nd Tuesday of each month at 6PM

 Jacksonville Multiple Myeloma Support Group

 Meets in a hybrid format on the 2nd Wednesday of each month at 6PM

 Palm Coast Multiple Myeloma Support Group

 Meets in-person on the 2nd Thursday of each month at 3:30PM

 Brooksville / Nature Coast

Multiple Myeloma Support Group

 Meets virtually on the 3rd Wednesday of each month at 6PM

 Tampa Bay/St Petersburg Multiple Myeloma Educational Group

 Meets virtually on the 1st Saturday of each month at 10:30AM

 Naples Multiple Myeloma Support Group

 Meets hybrid on the 3rd Thursday of each month at 12pm

 The Villages Multiple Myeloma Support Group

 Meets in-person on the 1st Tuesday of each month at 1PM

 North Tampa Multiple Myeloma Support Group

 Meets hybrid on the 3rd Saturday of each month at 10:30AM

 Tampa Central-Multiple Reasons Support Group

 Meets virtually on the 2nd Thursday of each month at 11AM

 Panama City Multiple Myeloma Support Group

 Meets in-person on the 2nd Saturday of each month at 10AM

 Sebring Multiple Myeloma Support Group

 Meets virtually on the 3rd Thursday of each month at 6pm

 Tallahassee Multiple Myeloma Support Group

 Meets in-person on the 4th Monday of each month at 5:30PM

Myeloma Voices at ASH

In Person / 5 Virtual

Myeloma Voices

IMF InfoLine

Connecting Patients to Resources…

Shortening “Time to Hope” for Over 1,000 First-Time Callers Each Year

 Assistance with understanding lab results, terminology and disease state

 Preparing for medical visits

 Access to medical providers

 Access to medications

 Financial resources

“Thank you so much for the informative conversation and all the time you spent listening and helping me decipher the MM lingo. What an amazing service!”

“Thank you for your response and excellent question suggestions for my hematology team.”

Written Education

Understanding Booklets

Tip Cards

Myeloma Minute Weekly Updates

Myeloma Today Quarterly News

Live Patient Education

4 PATIENT & FAMILY SEMINARS including world-renowned experts

10 MYELOMA COMMUNITY WORKSHOPS including local myeloma experts

Locations

2025 Live Patient Education

Scan for Upcoming Events!

Hot Topics in Myeloma

Joseph Mikhael, MD, MEd, FRCPC, FACP, FASCO Chief Medical Officer, International Myeloma Foundation

Patient Empowerment: Shared Decision Making

Teresa Miceli, RN, BSN, OCN, InfoLine Advisor,

Nurse Leadership Board, Mayo Clinic - Rochester

Patient Empowerment: Participating in Shared Decision Making

Teresa Miceli, RN BSN OCN

International Myeloma Foundation - InfoLine Advisor, NLB Member, Support Group Leader (MMSS, Smolder Bolder)

Mayo Clinic – Myeloma Nurse Navigator

National Cancer Institute - Myeloma Patient Advocate

OUR VISION:

A world where every myeloma patient can live life to the fullest, unburdened by the disease.

OUR MISSION:

Improving the quality of life of myeloma patients while working toward prevention and a cure.

Goals

• Review Shared Decision Making (SDM) Concepts

• Identify Influencing Factors To Treatment Decision Making

• Discuss Strategies To Enhance Patient Empowerment & Promote Shared Decision Making

Patient-Centric Care

“The aim of shared decisionmaking is to ensure that:

• Patients understand their options and the pros and cons of those options.

• Patient's goals and treatment preferences are used to guide decisions.”

Image Credit: https://www.ahrq.gov/health-literacy/professional-training/shared-decision/index.html

Agency

for Healthcare Research and Quality (AHRQ)

https://www.ahrq.gov/cahps/quality-improvement/improvement-guide/6-strategies-for-improving/communication/strategy6i-shared-decisionmaking.html#6i1

Steps in the Shared Decision-Making Process

 Identify that a decision is needed: The HCP informs the patient that a decision is to be made and that the patient's opinion is important (Choice talk).

 Understand the options:

The HCP explains the evidence-based options and their pros and cons. The patient expresses their preferences, and the HCP supports the patient in decision-making (Option talk).

 Come to a decision:

The HCP and patient discuss the patient's wish to take part in the decision making and incorporate the patient's values and preferences into the decision (Decision talk).

 Follow-up:

Review and evaluate the decision, adjust as needed

Benefits of Shared-Decision Making

Patients, regardless of age, want to be a part of treatment decisionmaking

Reduces uncertainty and alleviates concerns

Decisions reflect personal and family values and preferences

Requires staying informed

Promotes patient and care partner engagement and sense of empowerment

Positive impact on QOL and continuation on therapy

“The 'efficacy' of treatment means different things to different patients, and treatment decision-making in the context of personalized medicine must be guided by an individual's composite definition of what constitutes the best treatment choice.” Terpos, et al.

Factors That Influence Shared-Decision Making

Disease-Derived

Biology: Risk stratification, Urgent intervention needed vs time to consider options

Patient-Derived

Treatment: Availability/access, effectiveness, toxicity, current research

Understanding complex treatment options

Physical and emotional wellness

Comfort in speaking up “Doctor knows best”

Financial, Cultural and Religious factors

Care partner & social network, transportation

Provider-Derived

Time limitations

Support for patient involvement

Provider bias and preference

https://www.ahrq.gov/cahps/quality-improvement/improvement-guide/6-strategies-for-improving/communication/strategy6i-shared-decisionmaking.html#6i1. Choon-Quinones, M. et al. 2022; Terpos, et al. 2021 https://www.valueinhealthjournal.com/action/showFullTableHTML?isHtml=true&tableId=tbl4&pii=S1098-3015%2822%2900198-X

STRATEGIES FOR PATIENT EMPOWERMENT & SHARED DECISION MAKING

Strategies: Stay Informed

myeloma.org

Seek Information, Understand your options

 Use caution considering stories of personal experiences

 Your healthcare team members are resources

 Use reliable and current sources of information

IMF Website: http://myeloma.org

• Publications

• Videos and Replays

• Future Events, both in-person & virtual

Strategies: Be Involved In Your Care

 Consider your priorities

 Consider your goals/values/preferences

 Include your care partner/network in the discussion

 Be a part of the conversation, create a dialog

 Ask questions & express your goals/values/preferences

 Ask for time to consider options, if needed

 Arrive at a treatment decision together

 Arrange follow up to review and adjust the plan, if needed

Strategies: Know The Members Of Your Team

Understand their different roles

 Myeloma specialist and General Heme/Onc

 Primary care: for health screening, general check ups, vaccinations

 Sub-specialists: specialty needs

Stay connected

 Keep a contact list of your providers

 Know who to contact for more information

Allied

Strategies: Prepare for Medical Visits

Prepare

 Medications: Bring a current list of prescribed and over-the-counter

 Questions: Prioritize questions & concerns including financial issues

 Paperwork needing medical signature (ex FMLA, prior authorizations)

Inform

 Updates: Medical or life changes since your last visit

 Symptoms: How have they changed (improved, worsened, stable)? Keep a symptom diary. Bring it along

 Communicate effectively so your health care team can help

Follow Up

 “Next Steps”: Future appointments, medication changes, plan of care. Ask for the information in writing or on your patient portal

Include a care partner, especially for pivotal appointments

Strategies: Prepare for Tele-Health Visits

Check with your healthcare team –

 Is telemedicine an option?

 What is the process and what technology is needed?

 Are labs needed in advance? Do you need an order?

Preparation is similar for “in-person” appointment PLUS:

 Location: quiet, well-lit location with strong Wi-Fi is best

 Yourself: Do you need to show a body part - wear accessible clothing

 Vital signs (blood pressure, temp, heart rate, weight) self-serve blood pressure cuff is available at many pharmacies and for purchase

Include a care partner, especially for pivotal appointments

Create a Care Network

 Care partners assist in many ways

Myeloma causes the highest burden of symptoms, most commonly effecting people of older age with other medical issues. Care partner support is valuable in SDM

 Attending medical appointments, being present to learn and discuss possible treatment options and alert the medical team of side effects to treatment

 Some treatment options available only if care partner support exists

 Care partners can be one person or a rotation of many people

 Building a partnership is based in good communication

 Finding the balance:

- helping the patient with needed activities while maintaining a sense of independence

- allowing the care partner to have time for good self-care

Key Take-Aways and Things to Think About

Over the next two days:

 Evaluate where you are at in the process (What decisions need to be made?)

 Absorb the information being presented (What are the options?)

 Consider how the information impacts you and your family (What are your preferences?)

 Create questions that will lead to better understanding (What more do I need to know before making a decision?)

 Be an active member of your health care team

Shared Decision Making

Myeloma 101: The Big Picture Perspective with Q&A

Joseph Mikhael, MD, MEd, FRCPC, FACP, FASCO, Chief Medical Officer, International Myeloma Foundation

Teresa Miceli, RN, BSN, OCN, InfoLine Advisor,

Nurse Leadership Board, Mayo Clinic – Rochester, MN

MM101: The Big Picture Perspective

Joseph Mikhael, MD, MEd, FRCPC, FACP

• Professor, Applied Cancer Research and Drug Discovery, Translational Genomics Research Institute (TGen), City of Hope Cancer Center

• Chief Medical Officer, International Myeloma Foundation

• Consultant Hematologist and Director, Myeloma Research,

Phase 1 Program, HonorHealth Research Institute

• Adjunct Professor, College of Health Solutions, Arizona State University

Teresa S. Miceli RN BSN OCN

Mayo Associate, Assistant Professor of Nursing

Myeloma Research RN Navigator, Mayo Clinic, Rochester, MN

International Myeloma Foundation

InfoLine Advisor, Nurse Leadership Board, Support Group Leader

NCI Myeloma Steering Committee

How common is Myeloma in the US?

What

Causes Myeloma? How/Why Did I Get This?

Environmental Factors:

• Exposure to some chemicals

• Radiation exposure

Examples:

 Agent Orange

 Burn pits

 Pesticides, Herbicides

 Firefighter/First Responder exposures

Individual Factors:

• Age

• Family History of related disorders

• Personal History of MGUS or SMM

• Obesity

VA Study Documents Health Risks for Burn Pit Exposures

Leukemia and Multiple Myeloma Set to Be Added to List of Conditions Linked to Burn Pits

In most cases, the honest truth

WE DON’T KNOW

What is the Connection Between Bone Marrow & Myeloma ?

Photo Credit

Understanding (Mono)clonal Plasma Cells

Heavy Chain: G, A, M, D, E

Heavy Chain = M-Spike

 65% IgG – most common

 20% IgA – associated with AL Amyloid

 5% to 10% light chain-only (kappa, lambda)

 Less common: IgD, IgE, IgM

Is Myeloma the Only Protein Disorder?

• AL-Amyloid

• POEMS

• Light or Heavy Chain Deposition Disease

• MGCS = Clinical

• MGRS = Renal

• MGNS = Neuro

Condition

Clonal plasma cells in bone marrow

MGUS1-4 (Monoclonal Gammopathy of Undetermined Significance)

SMM1-5,8 (Smoldering Multiple Myeloma) Active Multiple Myeloma6-8

Presence of Myeloma

Defining Events

Likelihood

* In clinical trial

Testing For Myeloma: Blood & Urine

Test Name

CBC + differential

Complete metabolic panel

Beta-2 Microglobulin (B2M)

Lactate Dehydrogenase (LDH)

Serum Immunofixation and Protein

electrophoresis (SPEP+IFE)

Immunoglobulins (G, A, M, D, E)

Free light chain assay with kappa/lambda ratio

Urine immunofixation & protein electrophoresis (UPEP+IFE)

What it means

Hemoglobin, WBC, Platelets

Creatinine, Calcium, Albumin, Liver function

Part of staging and risk stratification

Measures the level of normal and clonal protein

Identifies the type of clonal protein

Measures the level of normal and clonal protein

Identifies the type of clonal protein

This Photo by Unknown Author is licensed under CC BY-SA-NC

Testing For Myeloma: Imaging

Imaging:

– Skeletal survey: Series of X-rays; less sensitive than other techniques

– Whole body low dose (CTWB-LD CT )

– Positron Emission Tomography (PET/CT)

– Magnetic Resonance Imaging (MRI)

Healthy bone versus myeloma bone disease

This Photo by Unknown Author is licensed under CC BY-NC-ND

Testing For Myeloma: Bone Marrow

Bone marrow biopsy & aspirate • Bone marrow plasma cells (%) • Congo Red staining if concern

Bone marrow genetics

• Cytogenetics

• Fluorescence in situ hybridization (FISH)

• Next generation sequencing (NGS)

This Photo by Unknown Author is licensed under CC BY-SA

What is the Myeloma Treatment Landscape?

Initial Therapy (a.k.a. Frontline, Induction)

Quad Therapy (ex. CD38+ MoAb + VRd)

HD-Melphalan + Stem Cell

Transplant (ASCT)

Maintenance

Treatment for Relapse

Supportive Care and Living Well

Relapse

Drug Class Overview

(thalidomide)

(lenalidomide)

(pomalidomide)

Rev, Len

or Pom

(daratumumab)

(isatuximab)

Drug Class Overview

Peptide Drug Conjugate* Pepaxto (Melphalan Flufenamide)

BCMA Targeted Antibody Drug

Conjugate (ADC)*

Blenrep (belantamab mafodotinblmf )

Abecma (idecabtagene vicleucel)

Belamaf, or B

Bispecific Antibodies

Carvykti (ciltacabtagene vicleucel)

Tecvayli (teclistimab)

Talvey (Talquetamab)

Elrexfio (Elranatamab)

Cevostamab, Iberdomide, Mezigdomide, Venetoclax Linvoseltamab, LCAR-B38M, ABBV-383

Measuring Disease Response: IMWG Response Criteria

Negative by next generation flow (NGF) (minimum sensitivity 1 in 10-5 nucleated cells or higher)*

mCR AND normal Free Light Chain ratio, Bone Marrow negative by flow, 2 measures

CR AND negative PCR

Complete Response: Negative immunofixation (IFE); no more than 5% plasma cells in BM; 2 measures

Very Good Partial Response: 90% reduction in myeloma protein

Partial Response: at least 50% reduction in myeloma protein

Minimal Response

Stable Disease: Not meeting above criteria

Progressive Disease: At least 25% increase in identified myeloma protein from lowest level

MRD = Minimal Residual Disease

sCR = Stringent Complete Response; BM = Bone Marrow

Kumar, S., Paiva, B., Anderson, K. C., Durie, B., Landgren, O., Moreau, P., ... & Dimopoulos, M. (2016). International Myeloma Working Group consensus criteria for response and minimal residual disease assessment in multiple myeloma. The lancet oncology, 17(8), e328-e346.

When Do I Need A New Treatment?

• Not every relapse requires immediate therapy

• Each case is different

Symptomatic or extramedullary disease

Asymptomatic biochemical relapse on 2 consecutive assessments

Asymptomatic high-risk disease or rapid doubling time or extensive marrow involvement Consider Observation Monitor Carefully Consider Treatment

Patient-/Disease-Specific Monitor Carefully

Initiate Treatment

Targets on the Myeloma Cell Surface and Therapeutic Antibodies

Bi-Specific Antibodies

Talvey (Talquetamab) CAR-T

Antibody Drug

Empliciti (Elotuzumab)

Bi-Specific Antibodies

CAR-T

Monoclonal Antibodies

Daratumumab and Darzalex Faspro

Sarclisa (Isatuximab)

TAK-079 MOR202

Immune Therapies

Abecma (Ide-cel CAR-T)

Carvykti (Cilta-cel CAR-T)

Tecvayli (Teclistamab)

Elrexfio (Elranatamab)

Other CAR Ts

Other Bi Specific Antibodies

Antibody Drug Conjugates

How it works:

An antibody directed at a target (BCMA) combined with a cytotoxic agent (chemotherapy)

ADC = Antibody-Drug Conjugate

BCMA = B-Cell Maturation Antigen

ADCP/ADCC = Antibody-Dependent Cellular Cytotoxicity & Phagocytosis

Image Credit: https://creativecommons.org/licenses/by-nc/3.0/

Bispecific Antibodies: Mechanism of Action

• Incorporates 2 antibody fragments to target and bind both tumor cells and T cells

• Brings target-expressing MM cells and T cells into close proximity, enabling T cells to induce tumor-cell death

Targets of Bispecific Molecule Vary

FcRH5

“Off the Shelf” Advantage

• No manufacturing process, unlike CAR T-cell therapy (but like ADC/belantamab therapy)

• Thus, no delay between decision to treat and administration of drug ADC = Antibody-Drug Conjugate; BCMA = B-Cell Maturation Antigen; CD3 = Cluster of Differentiation 3; FcRH5 = Fc receptor-homolog 5; GPRC5D = G-protein coupled receptor family C group 5 member D

The Process of CAR T Cell Therapy

CAR T therapy recommended. Insurance approved and ready to move forward.

What about Disease Control and Cure in Myeloma?

Requiring Treatment Stable or Unmeasurable Disease, Receiving Treatment  Control is the immediate priority with active disease  Cure remains the overall goal

Defining “Cure” has many considerations:

Minimal Residual Disease Negative (MRD-)

Time Off Therapy

Functional Cure

Unmeasurable Disease, Receiving No Treatment Active Disease

https://seer.cancer.gov/statfacts/html/mulmy.html; dated 6.15.2024

The Evolution of Myeloma Therapy

Rev/Dex

CyBorD

VTD

VRD

KRD

D-VMP

DRD

ASCT

Tandem ASCT (?)

Nothing

Thalidomide?

Bortezomib

Ixazomib

Lenalidomide

Combinations

Bortezomib

Lenalidomide

Carfilzomib

Pomalidomide

Selinexor

Panobinostat

Daratumumab

Ixazomib

Elotuzumab

Isatuximab

Belantamab mafodotin*

Melphalan flufenamide*

Idecabtagene autoleucel

Ciltacabtagene autoleucel

Teclistamab, Talquetamab

Elranatamab

D-VRD

Isa-VRD

D-KRD

Isa-VRD “More” induction?

Daratumumab?

Carfilzomib?

Lenalidomide + PI

ASCT, autologous stem cell transplant; CAR, chimeric antigen receptor; Cy, cyclophosphamide; d- daratumumab; D/dex, dexamethasone; isa, isatuximab; K, carfilzomib; M, melphalan; PD-L1, programmed death ligand-1; PI, proteasome inhibitor; Rev, lenalidomide; V, bortezomib.

Speaker’s own opinions.

CAR T Cell Therapy

Bispecific/Tri-specific

Antibodies

Cell Modifying Agents

Venetoclax

PD/PDL-1 Inhibition?

Small Molecules

* These agents are currently off the market but available through special programs

Anito-cel

Cevostomab

Linvoseltamab

Iberdomide, Mezigdomide

Sonrotoclax

Advocacy Update: Myeloma Advocacy Priorities & How to Help

Danielle Doheny International Myeloma Foundation, Director of Public Policy & Advocacy

Advocacy Overview and Medicare Changes in 2025 & Beyond

Danielle Doheny IMF, Director of U.S. Policy & Advocacy

Introduction | Advocacy at the IMF

The Global Advocacy Team collaborates with multiple stakeholders to inform and influence decision-making on the critical healthcare issues that directly impact myeloma patients.

The U.S. Advocacy Team advocates for equitable access to timely diagnosis, innovative treatments and research funding on Capitol Hill and with key regulatory agencies.

The team advocates both alongside of and on behalf of the patient community that we serve.

Advocacy plays a critical role to educate policymakers about the issues important to our community and motivate them to act.

What Do We Advocate For?

The following policy principles are the foundation on which we prioritize our advocacy work.

1. Ensure Access to Care: We advocate for policies that ensure all myeloma patients have equitable, comprehensive, patient-centered care without insurance barriers that limit options or delay treatment initiation.

2. Eliminate Financial Barriers: We advocate for policies that allow myeloma patients access to treatments and supportive care interventions without facing financial hardships.

3. Advance Myeloma Research: We advocate for annual appropriations funding for myeloma research and the advancement of clinical trial eligibility and research protocols that ensure representation from diverse populations.

2025 U.S. Advocacy Priorities Snapshot

1. ENSURE ACCESS TO CARE

INSURANCE REFORM: DRUG ACCESS

Step Therapy Protocols Safe Step Act

INSURANCE REFORM: DRUG ACCESS PBM Reform PBM Reform Act

2. ELIMINATE FINANCIAL BARRIERS

3. ADVANCE MYELOMA RESEARCH

MEDICARE REFORM:

PHYSICIAN ACCESS

Tele-Health/Medicine

Telehealth Modern. Act

INSURANCE REFORM: COINSURANCE

Oral Parity Cancer Drug Parity Act

INSURANCE REFORM: COPAYS Copay Accumulators HELP Copays Act

FEDERAL FUNDING

Annual Appropriations

NIH: National Cancer Institute, National Institute on Minority Health, ARPAH

MEDICARE REFORM: ANNUAL COST LIMITS

ANNUAL APPROPS Inflation Reduction Act implementation Cap & Smoothing (MPPP), Drug Pricing, Drug Formularies

CDC: Comprehensive Cancer Control Initiative

DoD: Congressionally Directed Medical Research Program (CDMRP) for Myeloma.

CLINICAL TRIAL DIVERSITY

Primary care education, Focus on underserved, POC, rural settings and socioeconomically disadvantaged groups

Medicare Changes Detailed | A Phased Approach

No copays for vaccines under Part D

Insulin copays limited to $35/month

Expanded eligibility for the Extra Help Program (Federal Low-Income Subsidy) to help pay premiums, deductibles, coinsurance & other costs

$3,250 annual cap (approx.) on out-of-pocket spending for prescription drugs under Part D (eliminating 5% coinsurance in catastrophic phase)

$2,000 annual cap on out-of-pocket spending for prescriptions under Part D

Option for a monthly payment program to “smooth out” total out-of-pocket spending throughout the year, with an overall monthly maximum

• Patients will need to enroll in the program (opt-in)

• The earlier in the year you join the program, the more you can benefit

• Your monthly bill may fluctuate somewhat

• No one will pay more than $2000 for the year

Inflation Reduction Act (IRA) & Highlight of Changes to Medicare

What is Happening With Research Funding?

Addressing healthcare barriers for multiple myeloma patients depends on winning over the hearts and minds of policymakers. It is not enough to just identify an issue and have data-driven evidence/research to back it up. It is not even enough to work with coalition partners that agree with our point of view. We must convince policymakers to prioritize our issues, draft legislation and vote it into law. Join Us! Join the Advocacy Team and share Your Story.

Thank You

Understanding Clinical Trials

Joseph Mikhael, MD, MEd, FRCPC, FACP, FASCO, Chief Medical Officer, International Myeloma Foundation

Clinical Trials

Joseph Mikhael MD, MEd, FRCPC

Chief Medical Officer, International Myeloma Foundation

Professor, Translational Genomics Research Institute, City of Hope Cancer Center

Yelak Biru, MSc

President and CEO , International Myeloma Foundation

Objectives

Clinical Trials - Overview

Some Of The Important Principles Of Clinical Trials:

 The drive of research has brought us to where we are

 No one is expected to be a “guinea pig” with no potential benefit to them

 Research is under very tight supervision and standards

 Open, clear communication between the physician and the patient is fundamental Driving research forward!

MYTH: If I participate in a clinical trial, I might get a placebo, not active treatment

MYTH: If I participate in a clinical trial, I can’t change my mind

Clinical Trials: Myths

• Phase 1 and 2, everyone gets active treatment

• Phase 3 standard of care vs new regimen: often standard regimen with/without additional agent in MM trials

• Patients can withdraw their consent for clinical trial participation at any time

MYTH: Clinical trials are dangerous because they have new medicines and practices

• Some risk is involved with every treatment, but medicines are used in clinical trials with people only after they have gone through testing to indicate that the drug is likely to be safe and effective for human use

MYTH: Clinical trials are expensive and not covered by insurance

• Research costs are typically covered by the sponsoring company

• Standard patient care costs are typically covered by insurance

• Check with clinical trial team/insurers; costs such as transportation, hotel, etc may not be reimbursed and are paid by patient

website. Accessed March 25, 2024. https://phrma.org/-/media/Project/PhRMA/PhRMA-Org/PhRMA-Org/PDF/A-C/CLINICAL-TRIALS-MYTH-FACTPRINT.pdf?hsCtaTracking=f6689b95-1626-40d9-8c87-c6b8d31600a4%7C35221aa8-d487-4db3-9416-b9c3c35e3bac

Clinical Trials – Why Me??

 Every patient is unique and must be viewed that way

 Benefits of trials are numerous and include:

 Early access to “new” therapy

 Delay use of standard therapy

 Contribution to myeloma world – present and future

 Financial access to certain agents

 Must be balanced with potential risks

 “Toxicity” of side effects

 Possibility of lack of efficacy

Overview of New Drug Development

Identify a target for therapy in the laboratory

Confirm the anticancer activity in laboratory and animal studies

Clinical trials (human studies) to determine safety, dosing and effectiveness

The whole process costs millions of dollars and years of effort!

Even Before Phase I

 Most agents are tested in lab models

 Various “myeloma cell lines”, also known as “in vitro”

 Next step is animal model

 We are more like mice than you think!!

 Earliest study in Phase I is called “First in Human”

 Often uses extremely low dose of drug to ensure safety

Phase 1 Clinical Trials

 All patients receive the experimental therapy

 Phase 1 trials find the optimal dose of a new drug or drug combination

 Patients get higher doses as the study continues

 Determine side effects of new drugs or combinations

 Explore how the drug is metabolized by the body

 Important for all stages of myeloma

Phase 2 Clinical Trials

 Determine if a new drug or combination is effective against the cancer

 May be added to a Phase 1 study once the ideal dose is found

 Patients usually receive the experimental therapy

 In some cases, the study may include two “arms” comparing either two different doses or a different treatment (another combination of drugs)

Phase 3 Clinical Trials

 Highest form of clinical evidence. Typically, a large number of patients are required…usually required for full FDA approval

 Patients receive either an experimental therapy (one or more drugs) or the current standard treatment

o The patient is randomly assigned to a treatment—a process called “randomization”

o Neither the physician or the patient can determine which treatment is given

 May be placebo controlled, if no standard treatments are available

 Very closely monitored for effectiveness and side effects

Preclinical

Clinical Trial Phases

ANIMAL STUDIES: Examine safety and potential for efficacy

PHASE 1

PHASE 2

FIRST INTRODUCTION OF AN INVESTIGATIONAL DRUG INTO HUMANS

• Determine metabolism and PK/PD actions, MTD, and DLT

• Identify AEs

• Gain early evidence of efficacy, studied in many conditions; typically, 20 to 80 patients; everyone gets agent

EVALUATION OF EFFECTIVENESS IN A CERTAIN TUMOR TYPE

• Determine short-term AEs and risks; closely monitored

• Includes up to 100 patients, typically

PHASE 3

GATHER ADDITIONAL EFFECTIVENESS AND SAFETY INFORMATION

COMPARED TO STANDARD OF CARE

• Placebo may be involved if no standard of care exists; hundreds to several thousand patients

• Often multiple institutions; single or double blind; sometimes open label

PHASE 4

APPROVED AGENTS IN NEW POPULATIONS OR NEW DOSE FORMS

Clinical Trials: Benefits of Participation

Possible Benefits:

• Patients will receive, at a minimum, the best standard treatment

• If the new treatment or intervention is proven to work, patients may be among the first to benefit

• Patients have a chance to help others and improve cancer care

Risks of Participation

Possible risks:

• New treatments or interventions under study are not always better than, or even as good as, standard care

• Even if a new treatment has benefits, it may not work for every patient

• Health insurance and managed care providers do not always cover clinical trials

Why Do So Few Cancer Patients Participate in Trials?

Patients may:

• Be unaware of clinical trials

• Lack access to trials

• Fear, distrust, or be suspicious of research

• Have practical or personal obstacles

• Face insurance or cost problems

• Be unwilling to go against their physicians’ wishes

• Not have physicians who offer them trials

• Have a disconnect with their healthcare team

Diversity in Clinical Trials

There has been a lack of diverse representation in clinical trials in myeloma.

 In the U.S., approximately 20% of all myeloma patients are of African descent, but only 5%–8% of patients in myeloma clinical trials are of African descent.

This is significant for the following reasons:

 All patients of all races and ethnicities should be able to benefit from clinical trials.

 Diverse patient representation in clinical trials is required to ensure that the outcomes are applicable to all patients.

Reasons for underrepresentation in clinical trials are complex and include:

 Systemic racism, accessibility of clinical trials, sensitivity to diversity by medical professionals

 Misconduct in medicine in the past, the lack of trust in the system, and more.

Importance of Clinical Trial Participation by Diverse Populations

[P]eople from racial and ethnic minorities and other diverse groups are underrepresented in clinical research. This is a concern because people of different ages, races, and ethnicities may react differently to certain medical products.

– FDA

Leadership and commitment

Community engagement practices

Investigator hiring, training, and mentoring practices

Patient engagement practices

US Cancer Centers of Excellence: Strategies for Increased Inclusion of Racial and Ethnic Minorities in Clinical Trials

Commonly Asked Questions

How does the study work? How often will I need to see my doctor or visit the cancer center?

Will I need to undergo additional tests?

What is currently known about the new drug or combination?

What benefits can I expect?

What side effects should I expect? Who should I notify if I have side effects?

Can I take my vitamins or other medications?

Can I get the treatment with my local doctor?

Will my insurance pay for my participation in the clinical trial?

Is A Clinical Trial Right For Me?

 Discuss with your physician if you are eligible for a clinical trial

 Work with your physician to determine the best trial for you

 Meet with the clinical research nurse or trials coordinator to discuss the trial

 Carefully review the provided “Informed Consent”

 Describes the study and any potential safety concerns related to the experimental medication

Accessed March 15, 2024. https://www.accccancer.org/home/attend/webinartemplate/2022/07/25/on-demand/just-askincreasing-diversity-in-cancer-clinical-research

Q&A WITH GUEST PANEL

Program Evaluations

We greatly appreciate your time and feedback!

IMF PATIENT AND FAMILY SEMINAR

BOCA RATON

AGENDA

SATURDAY MORNING

IMF PATIENT AND FAMILY SEMINAR

BOCA RATON

AGENDA

SATURDAY

AFTERNOON

Thank you to our sponsors!

5:00 – 7:00 PM Welcome Reception

Royal Palm Ballroom

OUR VISION:

A world where every myeloma patient can live life to the fullest, unburdened by the disease.

OUR MISSION:

Improving the quality of life of myeloma patients while working toward prevention and a cure.

IMF Core Values:

These are the core values we bring to accomplishing our mission each day.

Patient Centric

The patient experience is the focus of everything we do. Every interaction is an opportunity to establish a personal connection built on care and compassion which is the basis for continued support.

Respect All

As a team, we value honesty and transparency while creating a culture of mutual respect. We foster a myeloma community built on sincerity, authenticity, and kindness.

Excellence and Innovation

We value accountability, personal responsibility, and a steadfast commitment to excellence. We respect the legacy and reputation of our organization while seeking new solutions and advancements to improve outcomes, quality of life, and access to the best available resources for everyone impacted by myeloma.

Honor differences

We recognize each team member's skills and talents through collaboration and cooperation. Our programs aim to celebrate and support the diversity of our patients and their communities.