When scientists within the FDA advised against the approval of Exondys 51, a drug to treat Duchenne muscular dystrophy, parents of children with the rare genetic disorder and patients rallied to lobby for it in Washington. They were seen as pivotal to the FDA’s 2016 decision to grant approval for the drug, made by Sarepta Therapeutics. The decision was controversial in part because the FDA noted that clinical benefits of the drug – aimed at a subset of people with Duchenne muscular dystrophy – were not yet established.
Sarepta Therapeutics, which is not featured in the database, has taken measures to support its patient base. In March, it announced an annual scholarship program – 10 grants of up to $10,000 each for students with Duchenne muscular dystrophy to attend university or trade schools. Sarepta is also among the funders of Parent Project Muscular Dystrophy, a patient advocacy group at the forefront of the push for Exondys 51’s approval.
The Pre$cription for Power database will grow to include new disclosures. Not all drugmakers are willing to disclose their company giving. Eleven of the 20 companies examined – Allergan, Baxter International, Biogen, Celgene, Endo International, Gilead Sciences, Mallinckrodt, Mylan, Perrigo Co., Regeneron Pharmaceuticals, and Vertex Pharmaceuticals – declined to disclose their company giving or did not respond to repeated calls.
Paul Thacker, a former investigator for Sen. Chuck Grassley (R-Iowa) who helped draft the Physician Payments Sunshine Act in 2010, said there is reason to question the flow of money to patient advocacy groups. The pharmaceutical industry has fostered relationships in every link of the drug supply chain, including payments to researchers, doctors and professional societies.