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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary (2008)
Board on Health Sciences Policy (HSP)

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Wizemann, Theresa, Robinson, Sally, Giffin, Robert. "7 Strategies for Facilitating Clinical Trials." Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary. Washington, DC: The National Academies Press, 2008.

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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary

BOX 7-1

Examples of Disease Foundation Strategies for Facilitating Clinical Trials

MULTIPLE MYELOMA RESEARCH FOUNDATION (MMRF)

Founded in 1998 by a newly diagnosed multiple myeloma patient, Kathy Giusti, and her sister, Karen Andrews, MMRF’s mission is to urgently and aggressively fund research that will lead to the development of new treatments for multiple myeloma.

As part of its long-, mid-, and short-term research strategies, MMRF funds a portfolio of research worldwide comprising the basic science of multiple myeloma, validation, and Phase I and II clinical trials (conducted by MMRC; see below).


http://www.multiplemyeloma.org


MULTIPLE MYELOMA RESEARCH CONSORTIUM (MMRC)

A sister organization to MMRF, founded in 2004 by Kathy Giusti, MMRC has as its mission accelerating the development of novel and combination treatments for multiple myeloma by facilitating clinical trials and correlative studies. MMRC integrates the research efforts of 15 member institutions that represent the leading myeloma centers in the United States and the world. Centers are bound by a common membership agreement.

MMRC is designed to operate like a drug development organization. Its leadership team includes:

  • A chief executive officer with 10 years of experience in drug commercialization at two major pharmaceutical companies (also the founder and a myeloma patient).

  • A chief scientific officer with 16 years of industry experience in drug and target discovery.

  • A chief medical officer who is a trained hematologist/oncologist with more than 20 years of clinical research and drug development experience in industry.

The MMRC Progress Review Committee comprises experts from the member institutions, and selects and prioritizes targets. The MMRC Tissue Bank integrates myeloma tissue samples with corresponding genomic and clinical data. To facilitate accrual, MMRC has a program in place to allow patients to donate tissue directly. The MMRC Data Bank enables sharing of standardized data among consortium member institutions.

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