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Patient Advocacy Groups And Orphan Drug Development

January 16, 2013

A January 13, 2013, FDALawBlog.net article titled, “Patient Power in Orphan Drugs”, discusses the powerful influence that patients have on the development of orphan drugs. The article reviews the impact that the LAM Foundation had in helping with the development of the drug Sirolimus for the treatment of LAM. The LAM (Lympangioleiomyomatosis) Foundation, founded by a patient’s mother in the 1990’s, was instrumental in getting funds and setting up clinical trials for Sirolimus. Sirolimus receives FDA Orphan Drug Designation (ODD) on October 31, 2012 with “The LAM Foundation” identified as the Sponsor.

Because rare diseases affect small numbers of patients, finding patients to enroll in clinical trials can be difficult. However, with the aid of advocacy groups who have established lines of communication to the rare disease population, this can be a useful tool for generating buzz about a new treatment and also for educating potential patients about taking part in clinical trials.

Some patient advocacy groups provide large amounts of money in both preclinical and clinical research programs studying new orphan drugs for rare diseases :

The Cystic Fibrosis (CF) Foundation gave $75 million to Vertex Pharmaceuticals for the “collaboration of scientific, clinical, and financial support for the development of orphan drug Kalydeco (Ivacaftor)”

Pfizer announces on November 19, 2012, that the company is to receive up to $58 million in a 6-year pre-clinical R&D program, from Cystic Fibrosis Foundation Therapeutics (CFFT) to develop new drugs for the most common mutation of the disease – the Delta F508 mutation.

Other examples of recent patient advocacy groups impacting orphan drug development :

– The Orphan Druganaut Blog has documented for the past few months the role that several advocacy groups have played in helping with the development of Sarepta Therapeutics’ orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD). This is an example where advocacy groups are looking and finding venues to speed the drug’s progress. The Duchenne Alliance and the Parent Project Muscular Dystrophy (PPMD) organizations are 2 advocacy groups for DMD that are talking to both the FDA and Sarepta Therapeutics about ways of accelerating the approval of orphan drug Eteplirsen.

– The use of social media tools by patient advocacy groups to help get Vertex Pharmaceuticals’ orphan drug Kalydeco available to all who need it

– The Orphan Druganaut Blog has documented the ongoing, slow, but important role of the Cystic Fibrosis Trust UK in bringing Kalydeco to patients in the UK

– The Cystic Fibrosis (CF) Canada organization asking for patient input for Vertex Pharmaceuticals’ orphan drug Kalydeco.

Upcoming Patient Advocacy Meetings Events

–   Rare Disease Legislative Advocates (RDLA) Legislative Conference, February 25 – March 1, 2013, Washington, D.C.

–  6th Annual World Rare Disease Day 2013, February 28, 2013

–  CBI’s Orphan Drug Innovation Sumit, Philadelphia, PA, July 17-18, 2013

–  EURORDIS Summer School 2013 for Patient Advocates.

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

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