Progress Update, March 2014
A message from Bill Murray, CEO
When MDIC was created in the fall of 2012, the Board recognized the importance of clearly articulating our mission and areas of focus so that this innovative collaboration would be able to deliver measurable results that validate the vision for creating MDIC. In addition to being clear on who we are and our focus areas, we established a governance model for our projects with Board champions supported by MDIC project management staff to lead our key initiatives. This construct allows for efficient member engagement on projects that is coordinated and focused. As you read the project updates since our December newsletter, a common theme emerges. We are transitioning from planning and organizing to implementation and action. Our three projects, Clinical Trial Innovation & Reform, Patient Centered Benefit-Risk, and Computer Modeling & Simulation have formed steering committees and defined milestones for 2014. Of particular note, in January, we held our first Chief Medical Officer meeting with our member organizations including industry, non-profits, NIH, CMS, and FDA. Priorities were identified and are outlined in the project update. We also submitted grant requests for our Patient Centered Benefit-Risk project and finalized an agenda for our Computer Modeling & Simulation Summit. Working groups for each of these initiatives are forming, and I encourage our members to contact MDIC if you have interest in engaging. I believe you will find it to be time well spent.
Additionally, at our February Board meeting, we approved the initiation of a forth project focused on clinical diagnostics; an important emerging area as the technology and regulatory landscape is rapidly evolving. Our membership continues to grow as more people have become aware of MDIC. During the past quarter we added 4 new members. Thank you for your continued interest and support of MDIC.
CTIR Project Report
Stephanie Christopher, Program Manager
MDIC Clinical Trial Innovation and Reform envisions that the U.S. FDA and the medical device industry will recognize dynamic medical knowledge generation trends, adopt advanced clinical research methodologies, adapt regulatory policies and define new expectations for clinical research that will restore the United States to a leadership role in establishing standards for clinical excellence and medical technology innovation. Medical device clinical trials are increasingly complex, expensive and slow. Clinical trial innovation has the potential to improve the safety and effectiveness of medical device products being introduced into the market and yield earlier access to beneficial innovative technologies for U.S. patients. Success will require a sustained effort over many years and a significant investment from government, industry and non-profit groups.
As part of the effort to identify member priorities in medical device clinical trial reform, MDIC surveyed our member organizations in December 2013. Top priorities are reflected in the graphic below.
In January, MDIC hosted its first Chief Medical Officer workshop at the FDA campus in Silver Spring, MD. The day started with a tour of the CDRH Office of Science and Engineering Laboratories (OSEL), where workshop attendees learned about the research being conducted at CDRH. After the tour, the workshop started with brief introductions from MDIC, NIH, FDA and CMS and an overview on recent guidances on early feasibility studies and MDDT. The rest of the workshop was devoted to facilitated discussions of the problems and barriers to medical device clinical trials and where MDIC should focus its clinical trial innovation and reform efforts. Hot topics of discussion included: simplification of clinical trial design; early feasibility studies in the U.S.; patient reported trial outcomes; and pre-submission meetings.
After the input from the input from the CMO workshop and the pre-meeting survey, the CTIR steering committee prioritized focus on simplification of clinical trial design and early feasibility studies. Working groups are being formed to establish the goals and deliverables for these two projects. The working groups will focus on the unique role the MDIC and public-private partnership can contribute in medical device trial design and will work to complement other efforts in clinical trial reform, including CTTI. The simplification of clinical trials group is considering activities including a survey of physician societies and clinical researchers about clinical trial designs, a catalog of alternative clinical trial designs, and a review of clinical trial data including what data was collected and what data was published. The early feasibility working group is considering activities including a survey about the barriers in early feasibility studies in the U.S., identifying the medical device studies where early feasibility studies would be appropriate and the development of templates for early feasibility studies, including templates for research contracts and IRB applications. The steering committee is working on a position paper about medical device clinical trial reform and the importance of maintaining data quality in alternative trial designs.
By documenting the problems that exist in the current regulatory environment as well as defining, cataloging and testing possible solutions, industry, non-profits and government can work together to create a regulatory science environment flexible enough to evaluate the latest in medical technology, while still maintaining rigorous safety and efficacy data standards.
The CTIR steering committee’s next meeting is in March.
CM&S Project Report
Dawn Bardot, PhD, Sr. Program Manager
The future of rapid virtual prototyping, digital patients and populations, virtual clinical trials and personalized medicine is upon us; making possible our ability to bring better medical devices to patients. One of the necessary advancements is establishing computational modeling and simulation as valid scientific evidence which will transform the regulatory pathway for future medical products.
The MDIC CM&S vision statement is to reduce time and cost required to develop and approve medical innovation, while improving patient safety, through the consistent application of validated computational modeling and simulation in device development and regulation. To facilitate collaboration across industry, academia and government toward the ongoing advancement of the application of computational modeling and simulation to the development and regulation of medical devices.
In February the CM&S Steering Committee held their second teleconference. Milestones are identified for the first half of 2014 that demonstrate measurable progress around the three aims of 1) Define, Standardize, and Education on CM&S Validation Requirements, 2) Foster Collaboration, and 3) Activate Seed Grants.
The first CM&S Executive and Fellows meeting will be held on May 2nd at the FDA campus in Silver Spring, MD to align on the Modeling and Simulation Roadmap activities and initiate working groups. This meeting will be guided by the results of a membership survey on CM&S conducted in April.
In the second quarter of 2014, MDIC will launch a mock submission prepared by MDIC members including industry, FDA and nonprofits. This will help elucidate both the preparation of a submission using modeling and simulation and thought process on the review of the materials. The objective is to make publicly available this mock submission and hold a workshop to demonstrate and improve the Credibility Methodology, establishing Validation Requirements for CM&S.
The MDIC is hosting the Computational Modeling and Simulation Summit on June 25 and 26 at the Pew Research Center in Washington DC, save the date: June 25 & 26.
This two day conference is open to MDIC members and the public to build a community of practice striving to elevate CM&S to regulatory grade to reduce the burden on bench/animal/human models.
Don’t forget, there is less than a month left to comment on this Draft Guidance: Reporting of Computational Modeling Studies in Medical Device Submissions - Draft Guidance for Industry and Food and Drug Administration Staff:
PCBR Project Report
Stephanie Christopher, Program Manager
The science of measuring patient preference regarding probable benefits and risks for medical therapies is new and not well established. CDRH guidance published in 2012 outlines benefit-risk factors considered by FDA during medical device pre-market review, but there is no accepted mechanism for collecting patient preference data and integration of this data into the submission to aid the actual review process.
To address this gap in the science of measuring patient-centered benefit-risk assessments, the PCBR working group is working on the following deliverables:
- Development of a catalog of methods that can be used to assess patient preferences regarding the benefits and risks of a medical technology (the “Catalog”). Draft 2nd quarter 2014; final 3rd quarter 2014
- Development of a Framework for incorporating information on patient preferences regarding benefit and risk in to benefit/risk assessments of new technology (the “Framework”). Draft 3rd quarter 2014; final 4th quarter 2014
- Development of an agenda for further research and deliberation regarding the incorporation of patient preference information into medical device regulatory approvals (the “Agenda”). 4th quarter 2014
MDIC is partnering with RTI Health Solutions to develop the patient preference assessment methodology catalog (deliverable 1). RTI Health Solutions is a thought leader in developing and applying state-of-the-art benefit-risk preference elicitation methods and conducted the only benefit-risk preference studies that have been submitted to the FDA in support of drug approval applications. RTI will work in conjunction with the steering committee on identifying, evaluating and cataloging patient preference elicitation methods. At the February MDIC board meeting, the board approved seed funding to begin the RTI project.
The PCBR steering committee has applied for additional funding for the patient-centered benefit risk assessment project through PCORI. We have not yet received a final decision on the BAA proposal submitted to the FDA in November 2013.
The steering committee held its first meeting of 2014 in February with a full-day meeting at the Pew Center in Washington, D.C. At the meeting, the committee heard from Bret Hauber of RTI and Bennett Levitan from Janssen R & D about the strategy and steps for the methodology catalog project. Telba Irony, Chief, General and Surgical Devices Branch, Division of Biostatistics, Office of Surveillance and Biometrics, CDRH, presented on CDRH’s recently published patient preference study with regards to obesity devices. PCBR Board champion Ross Jaffe led a discussion on the patient preference framework and the questions the group should consider as they begin drafting the framework.
The next PCBR steering committee meeting is in April.