Since late February, the Race to Yes has been shouting from the rooftops: “Our children are dying, and we need help.” Treatments are on the verge of saving our children, but the system is getting in the way. Help us, FDA. Help us, Mr. President. You have the power to ensure our system helps rather than hurts our kids. Say yes to safe and effective treatments. Say yes to exploring all pathways to approve new drugs to save our dying children.
Last night, we heard a strong, clear voice in response to our pleas. On behalf of The White House, a high ranking FDA official announced to 106,000 people that yes, we share your urgency to develop treatments for Duchenne and yes, we are willing to explore the use of all potential pathways for the approval of new drugs to save your dying children.
On first glance this may seem like yet another form letter, yet another example of lip service while our children get weaker with each passing moment. But the White House response to our petition is important because it clearly states — not just once, but twice — that the White House supports the use of accelerated pathways for Duchenne treatments.
Now this may seem blatantly obvious if you’re not familiar with how new drugs are typically developed and approved. But it’s not, and that’s why the weight of White House support matters. In the drug development enterprise, there is a pervasive business-as-usual mentality that has become the standard operating procedure for developing new medicines. This involves large-scale, double-blind, placebo-controlled clinical trials that take on average 15+ years and cost hundreds of millions of dollars per drug. Much has been written about how entrenched this conservative mindset has become.
But Duchenne is not business as usual, and business as usual approaches won’t save our children. Duchenne is an aggressive, fatal disease with zero approved treatments. Our children are dying, and we’ve had no way to stop it. For the first time, the science is ready to change that. We face an extraordinary opportunity, and our system needs to support it. That starts with leadership saying yes – we support accelerated approval for treatments for Duchenne.
With its response, the White House has said to the world, we get it – the old way of doing things isn’t enough for Duchenne, we are willing to be flexible. We are willing to work with you to speed things along, even it requires a different format than what we’re used to. Show us what you’ve got.
So now we say to drug developers: Do it. Show them what you’ve got.
With this response, the White House has squarely handed the baton to companies coming around the bend in the race to develop new therapies. It’s shouting “Go, Go Go!” And for the company leading the pack, the timing couldn’t be more critical: Sarepta Therapeutics is in the crucial final lap to submit their drug eteplirsen for approval. As the frontrunner, Sarepta will set the pace for all other companies and treatments that will follow. Will they keep arguing about who should take the baton and how to pace themselves? Or will they will they hand the baton to their fastest runner and urge him to sprint across the finish line?