Breakthrough Business Models Drug Development for Rare and Neglected Diseases and Individualized Therapies: Workshop Summary (2009) / Chapter Skim
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8 Summary
8 Summary
Pages 98-103
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From page 98... ...
Still, barriers to progress persist. Tom Caskey reviewed financial barriers to orphan drug development during discovery, utility or proof of concept, and clinical development, and discussed drivers of investment (see Chapter 2)
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From page 99... ...
CFFT establishes business partnerships with pharmaceutical companies, lowering their risk in the development of drugs for rare disorders by providing financial support, access to leading cystic fibrosis experts and research tools, and access to the Cystic Fibrosis Therapeutic Development Network of Cystic Fibrosis Care Centers for facilitation of clinical trials. Genzyme approaches the development of drugs for rare diseases as a for-profit venture.
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From page 100... ...
All data, including images, biological samples, and clinical data, will be available in a public global database. The Genetic Alliance BioBank is an advocacy-owned repository for clinical data and biological samples, designed so as to create a firewall between researchers and many of the burdensome administrative and regulatory tasks associated with working with patients.
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From page 101... ...
Vertex Pharmaceuticals stressed alliances as the way forward and offered a list of lessons learned for alliance partners. The Myelin Repair Foundation, which has structured itself like a start-up business, showed how it was able to build networks, establish patient repositories, and share intellectual property to accelerate drug development.
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From page 102... ...
The Muscular Dystrophy Association, an umbrella organization addressing 40 rare neuromuscular diseases, has focused on establishing partnerships with other private-sector groups and developing patient registries, research clearinghouses, and international resources and standards. It also convenes collaborating groups in face-to-face meetings.
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From page 103... ...
Some of these compounds may still hold promise, and an assessment of their current disposition is needed. Bond suggested as a further area for discussion policies applied to the review of orphan drugs and what new or revised policies might facilitate the approval of such drugs.
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