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Translational Research Advisory Committee
Sponsored Meetings: |
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International Coordinating Committee for SMA Clinical Trials: SMA Summit on Drug Development – Bethesda, MD September 28-29, 2007
The SMA Summit on Drug Development was held on September 28th and 29th 2007 in Bethesda, MD. The event was hosted by the Patient Advisory Group (PAG) of the International Coordinating Committee for SMA clinical trials (ICC), which includes Families of SMA, Fight SMA, MDA, and the SMA Foundation. The ICC is a volunteer committee composed of stakeholders from the Spinal Muscular Atrophy (SMA) community who work together to address the opportunities and challenges associated with effectively organizing clinical trials of new treatments for SMA. The ICC consists of six working groups: the Patient Advisory Group, the Outcomes Measures Group, the Protocol Design Group, the Standard of Care Group, the Biomarkers Group, and the Registry / Database Group.
The SMA Summit on Drug Development was convened in anticipation of major drug efficacy trials for SMA in order to foster dialogue between stakeholders, to identify barriers to successful drug development, and to develop strategies to address these gaps in the pathway to regulatory approval. Attendees included representatives from the biotech and pharmaceutical industries, international advocacy groups, clinicians, and government. Participants discussed the currently available clinical infrastructure, the existing SMA therapeutic pipeline, and the regulatory requirements for evaluating new SMA treatments.
In order to bring SMA clinical trials to fruition in the most expedient and successful manner, the Summit Steering Committee has generated a summary of key points emerging from the conference discussion, including action items for the SMA community. These will be discussed in the meeting summary below in order of importance as deemed by the steering committee.
Meeting Agenda - PDF | Meeting Slides
MD Clinical Research Coalition Meeting, Tucson, AZ, June 11-12, 2004
MDA’s Translational Research Advisory Committee emphasized the need for more extensive clinical research infrastructure at its February, 2004 meeting. In response, MDA conducted a workshop in June of 2004 to further assess current needs in clinical research and to explore the potential for collaboration in this area. Attendees included clinicians, researchers, industry staff and representatives from the National Institutes of Health and the Centers for Disease Control.
Meeting Agenda
Summary and Recommendations of the Break-Out Groups
Challenges in Drug Development for Muscle Disease – A Stakeholders' Meeting - Bethesda, MD, August 4-5, 2005
The original certificate of incorporation for the Muscular Dystrophy Association in June, 1950, states that its purpose is “To foster and promote the cure and alleviation of the condition of persons suffering from the disease known as muscular dystrophy or from any similar or allied disease…” Since that time, MDA-funded investigators have contributed greatly to the field of muscle disease physiology and pathology, and to landmark research advances such as the first identification of a disease-causing gene through positional cloning in the 1980’s, and the first human clinical trial of gene transfer for a muscle disease in the 1990’s.
Now, as more and more potential treatments for muscle disease approach the human testing phase, academic and corporate investigators are struggling to determine what endpoints are meaningful in the clinical assessment of these therapies. Also, in some cases there is still a lack of appropriate natural history data and the field lacks certain necessary analytical tools. The goal of this meeting is to assess needs in this area from the perspective of various groups of stakeholders from academia, industry, the patient community and government in order that these obstacles to therapy development for muscle disease may be overcome efficiently. We would like to thank all participants in this meeting, whether your primary interest is in genetic muscle disease, or muscle wasting related to age, injury or illness, for coming together to address this common cause.
We would also like to acknowledge particularly our appreciation for the time of the representatives of federal regulatory and funding agencies who have agreed to share their perspectives and expertise.
MDA gratefully acknowledges the following for their support: Amicus Therapeutics, CepTor Corporation, Genzyme General, PTC Therapeutics, Pfizer, and Wyeth Pharmaceutical.
Meeting Agenda - PDF | Meeting Slides
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