MDCC July 1, 2003 Meeting Agenda

Muscular Dystrophy Coordinating Committee (MDCC)
First Meeting
July 1, 2003
9 am to 3:30 pm
Democracy II, Room 701
6707 Democracy Blvd., Bethesda, MD

Goals of Meeting:

• Inform committee members about the role of the MDCC and the proposed structure for yearly meetings.
• Learn about the collective expertise on the committee.
  • Hear the perspective of committee members.
  • Be informed of the ongoing and planned initiatives at different Federal agencies and other organizations relevant to MD research and education.
• Give committee members an opportunity to present their ideas and expectations for the MDCC.
• Design a strategy for developing a research and education plan within the required one year time period.
  • Develop a timeline for completing the plan.
  • Determine what information is needed to begin and plan ways to bring together the needed expertise.

Agenda:

Morning (9 am -12:30 pm)

1. Opening Remarks (Dr. Elias Zerhouni, Director, NIH)

2. Welcome and Introductions (MDCC Chair)

3. Review of Committee Charter and Charge to Committee (MDCC Chair) Excerpted from the MD-CARE Act:

(1) IN GENERAL- Not later than 1 year after the date of enactment of this section, the Coordinating Committee shall develop a plan for conducting and supporting research and education on muscular dystrophy through the national research institutes and shall periodically review and revise the plan. The plan shall--

(A) provide for a broad range of research and education activities relating to biomedical, epidemiological, psychosocial, and rehabilitative issues, including studies of the impact of such diseases in rural and underserved communities;

(B) identify priorities among the programs and activities of the National Institutes of Health regarding such diseases; and

(C) reflect input from a broad range of scientists, patients, and advocacy groups.

(2) CERTAIN ELEMENTS OF PLAN- The plan under paragraph (1) shall, with respect to each form of muscular dystrophy, provide for the following as appropriate:

(A) Research to determine the reasons underlying the incidence and prevalence of various forms of muscular dystrophy.

(B) Basic research concerning the etiology and genetic links of the disease and potential causes of mutations.

(C) The development of improved screening techniques.

(D) Basic and clinical research for the development and evaluation of new treatments, including new biological agents.

(E) Information and education programs for health care professionals and the public.

4. Discussion of Goals and Outcomes of First MDCC Meeting and Overview of Plans for Future Meetings
It is expected that the Committee will meet once or twice a year, likely for one day per meeting. During the first year, the committee may need to schedule two or three meetings, to allow sufficient time to develop and refine the research and education plan.

10:15 - 10:30 Break

5. Short Presentations by Members Representing Government Agencies -- 10 minutes each (* please note that this time limit will be strictly enforced to give all members an opportunity to present, and to allow for discussion time):
Government members can each provide an overview of his/her agency or institute's activities relating to MD research and education. A committee member from NIH will describe the role and activities of the NIH MD Research Task Force. Each member should also discuss his/her expectations for the MDCC.

o Stephen Katz (NIAMS)
o Audrey Penn (NINDS)
o Duane Alexander (NICHD)
o Coleen Boyle, ad hoc member (CDC, alternate for Jose Cordero)
o Russell Katz (FDA)
o Mary Jean Duckett (CMS)
o Merle McPherson (HRSA)
o Patricia Morrissey (Administration on Children and Families)
o Robert Pasternack (Department of Education)

Discussion

Lunch (12:30 - 1:30)

Afternoon (1:30 - 3:30)

6. Short Presentations by Public Members --10 minutes each (* please note that this time limit will be strictly enforced to give all members an opportunity to present, and to allow for discussion time):
Public members can each discuss the experience and perspective they bring to the committee. Committee members affiliated with a specific organization can describe the organization's activities with regard to MD research funding, and MD research and education. Each member should discuss his/her expectations for the MDCC.

o Patricia Furlong (Parent Project MD)
o Sharon Hesterlee (MDA)
o Daniel Perez (FSH Society)
o Donavon Decker (patient advocate)
o Bradley Stephenson (patient advocate)

Discussion

7. Proposed Strategy for Developing a Research and Education Plan
We will discuss various approaches to developing the research and education plan for the muscular dystrophies.

o Discuss ideas for the process of developing a research and education plan. o Discuss the scope of the plan. o Determine what information is needed to begin to develop the plan and how to bring together needed expertise. o Identify committee members and outside experts who can provide the committee with necessary background information.

8. Identify Action Items:

o Designate work that can be done prior to next meeting.
o Establish a schedule for future meeting dates.