6 minute read
Clinical Science
by MDIC
MDIC’S INITIATIVES AND PROGRAMS
MDIC operates initiatives focused on four key areas: Clinical Science, Data Science and Technology, Health Economics and Patient Value, and the National Evaluation System for health Technology Coordinating Center (NESTcc). Here we present 2019’s updates and accomplishments of each initiative and the programs that fall within the initiatives, when applicable.
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CLINICAL SCIENCE
The Clinical Science initiative, which is foundational to us as an organization, addresses the barriers to collecting adequate clinical evidence in support of new medical technology by creating blueprints for innovative clinical trial techniques, developing standards and metrics for effective clinical trial design, and encouraging the collection of adequate and appropriate clinical and patient preference data.
Programs:
• Clinical Diagnostics • Early Feasibility Studies • Science of Patient Input
A panel at MDIC’s 2019 Annual Public Forum discusses “SHIELD: Leading the way to IVD Semantic Interoperability”
2019 Clinical Science Year in Review:
CLINICAL DIAGNOSTICS
The Clinical Diagnostics initiative focuses on projects that are intended to improve diagnostic testing and product development using novel regulatory science approaches developed through collaboration among MDIC stakeholders. Providing a predictable path for innovation will help patients benefit through quicker access to more costeffective advanced diagnostic technologies.
Harmonized Education for Surrogate Samples Completed
The MDIC Surrogate Samples Framework proposes uniform terminology and recommendations where surrogate samples might be justifiably used in IVD validation studies. In 2019, our harmonized education materials for FDA and industry were completed. These modules are intended to provide the same education to FDA reviewers, industry R&D, and regulatory professionals on how to use the framework. The modules include recommendations on how to use the framework and case studies for selecting surrogate samples.
IVD Clinical Evidence Framework Published
The Framework for Developing Clinical Evidence for Regulatory and Coverage Assessments in In Vitro Diagnostics (IVDs) was completed and released. This framework is intended to help IVD manufacturers make decisions on how to develop credible evidence of analytical validity, clinical validity, and clinical utility. As part of the communication efforts for the Framework, MDIC participated in a poster presentation at the American Association of Clinical Chemistry Annual Scientific meeting and organized a panel discussion with representatives from FDA, payers, and industry at the Regulatory Affairs Professionals Society annual meeting.
Phase I Milestones Reached in Cancer Genomic Somatic Reference Samples (SRS) Project
The goal of the SRS project is to create physical reference samples that can be made available to the public to improve the accuracy, reliability, and transparency of Next Generation Sequencing (NGS)- based oncology tests. In 2019, a landscape analysis report, Somatic Variant Reference Samples for NGS, Landscape of Available Reference Samples, was released at the American Association of Cancer Research meeting and the SRS Prioritized Variant List was published and released. A request for information leading to the creation of a request for proposals (RFP) was completed. The committee continues to make its selection of vendor(s) to manufacture the somatic reference samples.
EARLY FEASIBILITY STUDIES
The Early Feasibility Studies (EFS) program aims to develop tools and best practices for EFS for sponsors and clinical researchers in order to drive overall EFS efficiency and support continuous assessment of EFS efficiency and effectiveness for US patients.
Recruitment for EFS Demonstration Pilot Completed
By the end of 2019, 31 sites and 15 sponsors were enrolled in the MDIC EFS Site Network Demonstration Pilot. MDIC’s role is to connect EFS sponsors and sites, provide EFS tools, and collect and disseminate metrics on EFS studies, tool implementation, and impact. The EFS Working Group will use those trends and data from the pilot to update EFS tools and best practices to further facilitate U.S. patient first-in-world access to innovative medical technologies.
31 Sites
15 Sponsors
EFS Newsletter Launched
EFS Express—a monthly email newsletter— was launched and 11 editions were published. EFS Express is a mechanism by which to engage and inform the EFS community with updates on the EFS Site Network Demonstration Pilot, educational materials, and event schedules and recaps.
2017/2018 EFS Metrics Collected
To understand the evolution of the EFS landscape, MDIC repeated the exercise of gathering EFS performance metrics from sponsors including IDE, IRB approval, contract execution, budget execution and first patient enrollment timing. Sponsors provided data from nine early feasibility studies conducted at 60 sites. The recent data showed a two-month reduction in time from IRB approval to first patient enrollment. However, the total average time of approximately eight months greatly exceeded MDIC’s target of four months. Additionally, contract and budget negotiations between sites and sponsors remained a challenge and thus remain a focus area for the EFS program.
Master Clinical Trial Agreement Template Revision
MDIC and Baylor Scott & White Research Institute hosted a virtual roundtable to further refine the EFS Master Clinical Trial Agreement (MCTA) template. A revised version of the template was released in 2019.
Communications Activities
As part of the EFS communication efforts, a number of workshops, meetings, and webinars on various topics were held, including site best practices and IRB/ICF workshops, a metrics presentation at the MDIC Annual Public Forum, a virtual legal roundtable focused on contracting, and a symposium on implementation strategies for EFS held at the Transcatheter Valve Therapies Structural Heart Summit.
SCIENCE OF PATIENT INPUT
The Science of Patient (SPI) Input program aims to advance the art and science of patient engagement in regulatory science, including advances in methodologies and tactical considerations for integrating the patient’s perspective and preferences in the design, clinical development, and regulatory review of innovative medical technologies.
Several Patient Engagement Resources Published
SPI working groups published several draft and final resources:
Landscape Literature Review of Existing Work in Patient Engagement in Clinical Trials Survey Report on Patient Engagement in Clinical Trials Best Practices for Communicating Benefit, Risk, and Uncertainty (Draft Report) Development and launch of survey of patient engagement methods used by the Patient-Centered Outcomes Research Institute (PCORI)-funded medical device researchers
Patients shared their unique experiences with medical devices and their perspectives on the medical device industry at MDIC’s 2019 Annual Public Forum.
Draft Report Completed on Research Collaboration to Move Clinical Trials from Generic p-value of 0.05 to TherapySpecific Patient-Values
In 2018, collaborators from MDIC, FDA, MIT, RTI Health Solutions, and the Michael J. Fox Foundation for Parkinson’s Research completed a patient preference study in persons with Parkinson’s disease and used the data from that preference study to test the MIT model for using patient preference information to set the p-values of a clinical trial. In 2019, the MDIC SPI Statistics and Outcomes Working Group developed a draft report in collaboration with the Center for Medical Technology Policy (CMTP) to document lessons learned from this project, including guidelines to integrate patient preferences in the statistical design of clinical trials.
Milestones Achieved in Heart Failure Project
In 2018, SPI launched an engagement to understand patient preferences in patients with heart failure. In 2019, MDIC partners, medical device manufacturers, heart failure patients, and patient preference experts selected a target study population, the recruitment strategies and types of treatments to be compared in the study with appropriate heart failure study attributes, developed the study protocol, and completed pretesting of the preference survey instrument.
Communications Activities
Representatives of MDIC’s SPI program had the opportunity to present at a Patients as Partners conference, the Association of Clinical Research Professionals conference, the MedTech conference, the World Parkinson’s Congress, the Excite International Summit, and the Professional Society for Health Economics and Outcomes Research.
