Tuesday, November 29, 2016

Neuroscience Center, Conference Room C/D
6001 Executive Blvd.
Bethesda, Maryland 20892

Meeting Agenda

8:30 am

Welcome and introductions

Stephen I. Katz, MDCC Chair

8:45 am

Conflict of interest policies and general meeting information

Glen Nuckolls, MDCC Executive Secretary

8:55 am

Meeting Reports

2016 FSHD International Research Consortium and Research Planning Workshop

Dan Perez, FSH Society

Bone Health and Osteoporosis in Duchenne Muscular Dystrophy

Kathi Kinnett, Parent Project Muscular Dystrophy (PPMD)

2016 Myotonic Dystrophy Foundation Patient-Focused Drug Development Meeting

John Porter, Myotonic Dystrophy Foundation

2016 Dystroglycanopathy Conference

Kevin Campbell, University of Iowa

10:00 am

Topic I: Ethics of Pediatric Clinical Trials
Ethical Challenges in Clinical Trial Design: Lessons Learned from DMD

Skip Nelson, FDA

10:30 am Break
10:45 am

Patient-Centered Benefit-Risk and Participant Burden in DMD Trials

Pat Furlong, PPMD

Pediatric Clinical Trial Design

Anne Zajicek, NICHD

11:45 am

Discussion of best ethical practices in clinical trial design

MDCC members and meeting attendees

12:30 pm


Break out session with working group on Access to Care and Services

Working group members (only)

1:30 pm

MDCC meeting resumes

Report from working group on Access to Care and Services

Valerie Cwik, Muscular Dystrophy Association, and Brian Denger, PPMD

1:45 pm

Topic II: Prospects for New Investigators in the Muscular Dystrophies

Promoting the Success of New Investigators

Kevin Campbell, University of Iowa

Promoting the Success of Newly Independent Physician/Scientists

Kathryn Wagner, Kennedy Krieger Institute

Success Rates for New Investigator Grant Applications

Tom Cheever, NIAMS

Perspectives of New Investigator Applicants and Grantees

Glen Nuckolls, NINDS

3:30 pm

Discussion of strategies to support and promote the success of new investigators

MDCC members and meeting attendees

4:15 pm

Closing remarks

Stephen I. Katz














Meeting Summary



Dan Perez of the FSH Society presented a summary of the FSHD International Research Consortium and Research Planning Workshop. The meeting included a review of priorities in the research field and discussion of priorities going forward, including the need for surrogate outcome measures, additional natural history studies, a focus on uniformity of genetic testing, models that recapitulate disease progression, and genetic modifying techniques. Kathi Kinnett of Parent Project MD summarized PPMD’s meeting on Bone Health and Osteoporosis in DMD. The meeting highlighted the large variability in clinical management of these conditions and raised important issues for consideration in maintaining bone health and managing osteoporosis in patients with DMD including the role of exercise, controversies around the use of bisphosphonates, and potential non-bisphosphonate treatments. John Porter of the Myotonic Dystrophy Foundation (MDF) spoke about the MDF’s Patient-focused Drug Development workshop, which included many advocates as well as FDA staff. A Voice of the Patient report is being developed. Discussion from MDCC members included the need to collect data from natural history databases and a call for a means for researchers to be able to access patient-reported data. Kevin Campbell of the University of Iowa Wellstone Center gave an overview of the 2016 Dystroglycanopathy Conference, a conference to connect patients and families with researchers at the Wellstone Center. Investigators at all career stages were involved in the conference which gave patients and families the opportunity to visit labs and learn first-hand about research at the Wellstone Center. Dr. Campbell stressed the value to the researchers in speaking with, meeting, and learning about the patients who their research is designed to ultimately help.

The MDCC meeting also included a brief update from the newly formed MDCC working group on Access to Care and Services, which is co-chaired by Valerie Cwik (MDA), Brian Denger (PPMD), and Glen Nuckolls (NIH). The group will be working to develop strategies to overcome obstacles that individuals and families affected by muscular dystrophy encounter in accessing quality medical care, obtaining durable medical equipment, integrating into the workforce, and obtaining other services across all age ranges.

Topic I: Ethics of Pediatric Clinical Trials 

Three presentations helped to frame the discussion regarding balancing risks and benefits to patients and their families in designing pediatric clinical trials. Skip Nelson of the FDA gave an overview of the basic ethical framework in pediatrics trials, including a discussion of the issues associated with direct clinical benefit and minor increase of minimal risk, using muscle biopsy as an example that occurs routinely in the muscular dystrophy patient population. He discussed rigorous practices in trial design and execution as well as ideas for how to improve the system, including sharing existing clinical data, developing meaningful clinical endpoints that don’t rely on walking, developing non-invasive biomarkers, and allowing patients enrolled in a trial the ability to cross over to active treatment. Pat Furlong of PPMD talked about a benefit risk pilot study of 11 clinicians and 115 parents carried out by PPMD. The highest priority was found to be slowing or stopping the progression of disease, even if that came with accepting serious risks. PPMD will follow up with a treatment preference study in teens and adults, and will work to further understand patients’ and families’ tolerance for uncertainty in benefits and acceptance of risks. Ms. Furlong also spoke about the importance of understanding parent perceptions of clinical trials and also considering clinical benefit vs. meaningful benefit to patients. She stressed the need to consider the burden of participation (financial, emotional, etc.) in a trial on both the participant and the family, the need to give patients and families continued support during trial, and to report back to participants and their family on trial results. Anne Zajicek from NICHD spoke about good clinical. practice, data resources and the importance of data sharing. She shared lessons learned from the NICHD’s Pediatric Trials Network.

Topic II: Patient access to care, services and medical equipment

The second major topic discussed at the meeting was trends seen in the MD new investigator workforce and ways to promote the success of this part of the workforce. Kevin Campbell stressed the importance of mentoring newly independent investigators on managing their budgets, considering the costs of lab personnel, and avoiding overly ambitious research projects. For many new investigators, their former post-doc advisor may be a good source for this mentoring. He mentioned the need for institutional and private foundation support as well as institutional resources including accessible and affordable childcare. In addition to finding ways for investigators to start labs at a younger age, he also mentioned in importance of promoting the success of newly established investigators (e.g., those applying for their second R01). Kathryn Wagner emphasized challenges with regard to the length of training of clinical investigators and the large amount of time needed to devote to both clinic and lab work (upwards of an 80-100 hour workweek). Both Drs. Campbell and Wagner emphasized the need for time protected from teaching, clinics and administrative activities so that new investigators can conduct research and develop collaborations. Dr. Wagner felt that applicants for NIH K awards at her institution tend to be much more successful that the average applicant because her institution strongly encourages applicants to have their applications reviewed by an internal panel prior to submitting them to NIH. She suggested the possibility of adding a junior physician-scientist position to the NIH-funded Wellstone Centers.

Tom Cheever showed data on the success rate of muscular dystrophy NIH-funded new investigators, as a follow-up to the last MDCC meeting. In analyzing the outcomes of MD new investigator applications, he found a fair amount of variability and small numbers overall, but noted that the success rate of MD new investigators at obtaining an NIH R01 trends lower than the success rate for established investigators over the last several years. Established MD investigators actually do as well or better than all NIH established investigators. As noted by Dr. Campbell, the success in obtaining a second R01 is also a significant challenge for MD researchers, as it is for all NIH researchers. This highlights the importance of other non-NIH funding in supporting researchers during these critical junctures in their careers.

Glen Nuckolls talked about information collected through phone conversations on the personal perspectives of some new and early stage investigators in the muscular dystrophies. A number of themes emerged from these conversations including the fact that start-up packages and protected time for research varied widely across institutions, the challenges of competing with established labs for funding, the need to network and seek out speaking and grant reviewing opportunities, and the importance of initiating and maintaining collaborations. Some investigators mentioned the attractiveness of industry since they no longer needed to apply for grant funding. The investigators had a number of suggestions including setting up sessions at regional or national meetings on career development topics, organizing meeting sessions to showcase the work of new investigators, developing ways to promote collaborations, and the utility of an online portal or other system that compiled all relevant funding opportunity announcements. Some of these may be opportunities for MDCC member organizations.

The discussion during this session included ways that applicants who just miss the NIH payline can get funding from other organizations, and Dr. Cheever mentioned the Online Partnership to Accelerate Research (OnPAR) as a potential resource.

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