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Patient Advocacy Groups Investing Money In Pharma

March 12, 2013

The power and influence of patient advocacy groups on orphan drug development is tremendous. The trend recently is for the investment of money of patient advocacy groups in pharmaceutical companies that are working on new treatment options for rare diseases. Many rare diseases have patient advocacy groups that provide large capital investment in preclinical and clinical research programs of pharmaceutical companies. It is a mechanism of uniting both the rare disease community and pharmaceutical companies with the common goal of supporting the development of new treatments.

One of the most successful examples of the partnership between advocacy groups and pharmaceutical companies, is the partnership between the Cystic Fibrosis (CF) Foundation and Vertex Pharmaceuticals. The CF Foundation gave $75 million to Vertex Pharmaceuticals for the very successful “collaboration of scientific, clinical, and financial support for the development of orphan drug Kalydeco (Ivacaftor)”. The CF Foundation announces in November 2012 a $58 million CF drug discovery agreement with Pfizer, to develop new drugs to treat patients with the most common mutation of CF, Delta F508.

PTC Therapeutics is developing orphan drug Ataluren for patients with a particular genetic mutation for Duchenne/Becker Muscular Dystrophy and for CF. Ataluren is supported by grants from multiple patient advocacy groups and foundations :

  1. $25    million from the CF Foundation in support of key Phase 2b clinical trials for nonsense mutation CF
  2. $  2.5 million from the Muscular Dystrophy Association (MDA) to support development for nonsense mutation Duchenne Muscular Dystrophy (DMD)
  3. Support from the Parent Project Muscular Dystrophy.

Another successful story is that of the advocacy organization CureDuchenne, founded by Paul and Debra Miller, parents of a son diagnosed with DMD. The goal of the organization is to find a cure for DMD. This is a story of research finding the nonprofit organization instead of the usual nonprofits finding the research to support. Prosensa, a Dutch company that was doing research to moderate the effects of DMD through exon skipping, approached CureDuchenne. The organization gave $1.3 million to Prosensa to support their initial research. Seven years later, Prosensa receives a commitment of up to a $650 million investment for their Duchenne programs.

The CHDI Foundation is a private, not-for-profit research organization whose mission is “to discover drugs that slow the progression of Huntington’s Disease (HD).” The organization provides financial support for research for HD. In February 2013,  CHDI announces a collaboration with KineMed and Isis Pharmaceuticals to develop “novel biomarkers for pre-clinical and clinical use in drug development and therapeutic monitoring.”

Parents are usually the founders or main force behind patient advocacy groups. The partnership between patient advocacy groups and the pharmaceutical industry has influenced greatly orphan drug development. It is a partnership where both parties win.

Please Note: “Working Together Teamwork Puzzle Concept” by lumaxart (Working Together Teamwork Puzzle Concept) [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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