A new documentary is being made that will address the controversial subjects of early access to drugs in development, biomarkers as primary outcomes, and placebo-controlled studies. These subjects make for interesting philosophical conversations at meetings but for parents trying to get a drug approved for their child with Duchenne muscular dystrophy, the topics are a matter of life or death.
To the Edge of the Sky is a documentary about parents fighting the FDA to get an experimental drug (Sarepta’s eteplirsen) accelerated approval. Without the experimental drug, the parents know their boys will spend their teen years in a motorized wheelchair and eventually succumb to the disease in their early 20s. With the experimental drug, the boys may continue to walk and they may live longer. The key word in that last sentence is ‘may’. According to the FDA, there is very little properly controlled data to confirm that would be the case. But parents are willing to take that risk. The FDA are not. Accelerated approval by the FDA means that the drug would be approved for treatment based on biomarkers that according to the FDA, have not been properly standardized to allow them to be primary outcome measures in pivotal clinical trials for Duchenne muscular dystrophy. As such, the FDA wants more data. It is their responsibility to only approve drugs that they know are safe and effective. For life threatening rare diseases, that is often difficult to determine. In the meantime, families are watching their children slowly die.
To the Edge of the Sky follows four families who have boys with Duchenne muscular dystrophy as they wage an extraordinary campaign to convince the FDA to allow the accelerated approval for a drug (eteplirsen) which they believe is a potential cure for Duchenne––before it kills their sons. One of the families includes Christine McSherry, who we interviewed last fall about her conversations with the FDA. Click here to see that conversation.
The documentary is being produced by Todd Wider and Jedd Wider, the team behind numerous documentaries including:
- Mea Maxima Culpa: Silence in the House of God (2012, HBO)
- Taxi to the Dark Side (2007, HBO)
- Semper Fi: Always Faithful (2011)
- Client 9: The Rise and Fall of Eliot Spitzer (2010)
- Morgan Spurlock’s What Would Jesus Buy? (2007)
The kickstarter campaign is designed to accomplish two objectives:
- To “accelerate our post-production schedule, increase our staff, finalize editing immediately, and hire the right people to assist in our outreach campaign.“
- To “rally the masses and get the global community involved in reform––the more people who get involved, the more attention rare diseases will receive. And we can do that through crowdfunding.”
The producers hope to have the film completed by October 2015. To see the trailer for the film and consider donating to the project, click here.
( Originally published at http://www.raredr.com/news/Documentary-Duchenne-FDA )