Rare Diseases and Orphan Products Accelerating Research and Development (2010) / Chapter Skim
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Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples
Appendix F: Advocacy Group Approaches to Accelerating Research and Product Development: Illustrative Examples
Pages 371-386
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From page 371... ...
Although the number of rare conditions for which there are advocacy groups has grown, a great many rare conditions lack research-focused advocacy organizations. Moreover, many existing rare diseases advocacy organizations have very limited funds to support research and are still developing the expertise and experience to support a focused research effort.
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From page 372... ...
. Among rare diseases organizations, the Cystic Fibrosis Foundation has, in many respects, led the way in developing and implementing a systematic research strategy that is tailored to evolving research progress and scientific and technological opportunities.
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From page 373... ...
3.7 Spinal Muscular Atrophy Foundation 3.9 Cystic Fibrosis Foundation 71.6 SOURCE: For Cystic Fibrosis Foundation and Scleroderma Research Foundation, 2008 financial statement. For Alpha 1 Foundation and Friedreich's Ataxia Research Alliance, 2009 annual report.
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From page 374... ...
and 5 private institutions over this past decade and representing $107.5 MM, were categorized using the Biomedical Research Classification Scheme specifically developed for this analysis. Each grant was mapped along a continuum from basic and etiologic research, through the stages of drug development and the clinical evaluation of treatments.
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From page 375... ...
This bottleneck is illustrated by a steep decline in funding beyond the target validation stage of the drug discovery and development process. While it is clear there is still much to be learned regarding the complex biology of MeCP2, this analysis nevertheless underscores a need for future funding to be directed towards specific programs and resources that will help alleviate the current obstacles to translation and facilitate treatment development for RTT.
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From page 376... ...
The ad hoc committee produced the research agenda shown below and it serves as a working document used by the grants award program for prioritizing the relevance of grant applications to the Foundation's overall research goals. The use of the strategic plan and research agenda for evaluation of grant applications is only one use envisioned for the research agenda document.
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From page 377... ...
In 000, the foundation created Cystic Fibrosis Research Foundation Therapeutics, a nonprofit research affiliate, to oversee drug discovery and development activities.
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From page 378... ...
CFFT supports and governs activities related to cystic fibrosis (CF) drug discovery through the many stages of drug development and clinical evaluation.
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From page 379... ...
Further, with increasing demands being placed on pharmaceutical and biotechnology companies, especially the small "start-ups," investors are often hesitant about making major capital investments for orphan diseaseclassified drugs. The Therapeutics Development Program attracts researchers to the CF drug development process and shows a level of commitment unrivaled by any other voluntary health organization.
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From page 380... ...
Animal Models There are several SMA mouse models available. Many of these models are genetically modified to be deficient in mouse SMN protein and also have varying types and amounts of human SMN genes introduced to their genome in an effort to recapitulate a range of disease features and severities seen in patients.
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From page 381... ...
Licenses The SMA Foundation has entered into licensing agreements with several Institutions in order to facilitate access to critical research tools, while giving the Inventors and Institutions acknowledgment of key contributions and fair compensation for their intellectual property. The Foundation offers the opportunity to obtain sublicenses for the research and therapeutics development tools listed in the section below.
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From page 382... ...
Information Page on SMA • MedlinePlus Information on SMA • GeneClinics Clinical Review of SMA • Genetic Testing Resources from GeneTests • Columbia University Medical Center Spinal Muscular Atrophy Clinical Research Center • Treat-NMD Clinical Research Initiatives in Europe Scientific Literature Resources: • SMA Foundation Bibliography at http://www.smafoundation.org/ bibliography EXAMPLE 5 FRIEDREICH'S ATAXIA RESEARCH ALLIANCE The Friedreich's Ataxia Research Alliance (FARA) raises funds for scientific research on the disease with a focus on translational and clinical research and on national and international public-private collaborations and partnerships.
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From page 383... ...
Scientific Conference Program (This material is used with permission and excerpted from http://www.curefa.org/conference.html.) FARA has organized and supported a number of scientific conferences to keep the field informed of research progress and build collaborations and synergistic connections between FA researchers.
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From page 384... ...
Therapeutics Symposium More than 100 FA researchers and our advocacy partners from around the world gathered July 15-17, 2009 for the FA Therapeutics Symposium in Philadelphia, PA. Presentations and discussions highlighted: • progress in the development of previously identified therapeutic candidates, such as HDACI and TAT-Frataxin results from clinical trials including the Phase I study of A0001 and Phase III of Idebenone; • recent discoveries that point to new therapies; • advancements in new cell models and drug discovery and development assays; and • clinical research including biomarker studies and new clinical outcome measures.
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From page 385... ...
. The Scleroderma Research Foundation's Postdoctoral Fellowship program funds grants aimed at focusing talented young investigators on specific research questions in the nation's top laboratories.
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From page 386... ...
Review Considerations Completed applications will be evaluated by the SRF Scientific Advisory Board. Fellows will be selected on the basis of previous achievements, the commitment of the applicant, sponsor and sponsoring institution to scleroderma research, the scientific and technical merit of the research proposal, and the relevance of the proposal to the SRF's ongoing research program.
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