A page-one story in the May 19th Wall Street Journal examines the ties between Sarepta Therapeutics and parents of boys with Duchenne muscular dystrophy (DMD) who successfully advocated for regulatory approval of eteplirsen: “How the FDA Approved a $300,000-a-Year Drug Its Own Experts Didn’t Believe Worked,” by Susan Pulliam and Brody Mullins, May 19, 2017.
The article recounts how “desperate” executives and parents collaborated to prepare anecdotal videos of children with DMD, presented to regulators in a format “akin” to clinical reports. In addition, a lawyer retained by Sarepta prepared boys and their caregivers to testify at an FDA advisory committee meeting, without the knowledge of panel members.
Based on this report, we see several important trends and actionable insights for rare disease companies and patient organizations alike:
- Rare disease companies face growing scrutiny. The investigative report is part of a series revealing “secret ways companies work the levers of power in Washington.” It follows coverage by Kaiser Health News, NPR and other media suggesting that some companies exploit the Orphan Drug Act. Likewise, orphan-drug pricing continues to draw criticism.
- Patient-centered drug development is a work in progress. The concept of including the patient voice early in the research process appeals to many stakeholders. However, the appropriate steps to make this a reality are not always well defined or consistently adopted. The Sarepta case confirms there are many dynamics at play: scientific, medical, economic, financial and emotional. Efforts rooted in science – patient reported outcomes, videos integrated into clinical trial protocols, or risk-benefit analyses – can help.
- Compliance begins with – and extends beyond – regulations. It’s critical for industry and patient organizations to clearly define their relationships and maintain their independence. The perception of inappropriate activity can be just as damaging as an actual violation. Patient organizations and industry can minimize these risks by adopting guidelines for engagement and following best practices for communication and media relations.
- Critical reports could plant seeds of resistance to continued reforms. Interestingly, the article closes with a brief mention of the 21st Century Cures act, criticized as being too favorable to drug manufacturers. This skepticism could dampen support for other legislative reforms, such as the OPEN Act. Continued education and involvement of all stakeholders will help maintain and protect important tools to support timely review and approval.
Although incomplete, the Journal’s rehash of the eteplirsen approval brings to the surface many challenges of orphan drug development. While there is no simple antidote, industry must show through both actions and words that their primary motive is serving patients, with profits as a valid byproduct of this strategy.
Chris Smith is the President and CEO of SmithSolve, a communications agency helping clients earn trust, manage risk and break through the noise.