Industry partners receive FDA’s Pediatric Rare Disease Designation – and YOU helped make it possible!

In the last few weeks we’ve heard the news from several companies working to develop therapies for Duchenne that they have received Rare Pediatric Disease Designations from the FDA. Santhera, Sarepta, Catabasis, and Biomarin.


We as a community have celebrated this news. But what does it actually mean?


And did you know that it would not have been possible without YOUR tireless advocacy and the infrastructure you’ve long worked with PPMD to create?


So… here’s the story…


The Rare Pediatric Disease Designation being celebrated actually refers to both an FDA drug development designation, as well as the potential for eligibility for an incredibly valuable voucher known as the Rare Pediatric Disease Priority Review Voucher (sometimes referred to as ‘PRV’).  Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, at the approval of a qualifying new drug application (NDA) or biologics license application (BLA) for the treatment of a rare pediatric disease, the sponsor is eligible for a Rare Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent NDA or BLA.


So what does this mean?


As we all know, we are a rare disease community. Technically speaking, this means that Duchenne affects fewer than 200,000 people in the U.S. From the perspective of attracting industry into our space, our rare disease communities have worked collaboratively for decades to seek ways to incentivize industry to develop therapies for patients for rare disease. If you are a company and you have a choice of spending hundreds of millions of dollars working to develop a therapy (that may or may not go to market) for a small patient population – versus a large patient population, there needs to be some sort of financial incentive to work with the smaller patient populations. Thus, the PRV program has been one of the ways these incentives have been approached. Here’s how the PRV works.


Typically, the process usually looks something like this:

  • A company requests priority review designation from the FDA
  • FDA determines whether the condition drug targets meets criteria for rare pediatric disease
  • FDA awards a priority review designation
  • Drug review decision takes place within 6 months


Under Priority Review Voucher (PRV) System, the Process looks more like this:

  • Company is granted or purchases a PRV
  • Company informs FDA of intent to use voucher on upcoming submission (within 90 days of submission)
  • FDA accepts voucher, agrees to review product within 6 months


And there’s more…. While in order to qualify for the voucher in the first place the company’s product must target a rare pediatric population, the voucher can be sold or transferred an unlimited number of times. Thus, the incentive is to qualify for the priority review in a rare pediatric product and then sell the voucher and apply it to an expedited review for a product with a potentially much larger market.


So, how’s it working? Well, recently United Therapeutics sold their Priority Review Voucher to Abb Vie for $350 million. So, I’d say that it’s working.


The program was actually first implemented for tropical diseases under The Food and Drug Administration Amendments Act of 2007 (FDAAA) and then expanded to include rare pediatric diseases by Section 908 of the Food and Drug Administration Safety Innovation Act (FDASIA) which added Section 529 to the Federal Food, Drug, and Cosmetic Act.


Our role


Here’s how PPMD and our Duchenne community’s investment into advocacy and infrastructure became a critical part of this story.


In June, several companies reached out to me and alerted us that while pursuing pediatric rare disease designations, they had received some push-back from the FDA on their submissions and we were asked to help justify that Duchenne is indeed a rare pediatric disease. While no one was questioning the issue of ‘rare’, what was being questioned was whether Duchenne indeed still met the qualification of ‘pediatric.’


Following these inquiries, I pulled the statute and was at a loss for how Duchenne would not easily fulfill the required definition that greater than 51% of the Duchenne population in the U.S. was aged birth through 18 years? While clinical care advances have improved quality of life for people with Duchenne and – in many cases – have extended life expectancy, we have not advanced to an extent that the majority of our Duchenne population are older than 19. We look forward to that day.


These companies’ clocks were ticking and they needed assistance in providing justification of our population’s pediatric status.


At PPMD we maintain the DuchenneConnect registry, the only national Duchenne registry in the United States. While our industry partners had included some of this data in their previous submissions, we began to delve for further information to respond to the FDA’s questions.


But as it turns out, our community had invested in an even more comprehensive data set. We had worked to pass the MD-CARE Act, and we had spent decades developing relationships with our federal agency partners.


You may recall that one of the first elements authorized by the MD-CARE Act in 2001 was a national Duchenne/Becker surveillance program (MD STARnet). I reached out to our friends at the Centers for Disease Control and Prevention (CDC) and asked whether they would be able to work with us on this matter. The CDC understood the urgency and agreed to work directly with the FDA. I then reached out to our partners at the Office of Orphan Products at the FDA and explained the inquiries that we’d been receiving and offered to convene a discussion between the FDA and CDC – they were eager to participate. Within 48 hours, 9 key personnel at the FDA and CDC – all of whom were deeply committed to our Duchenne community – were reviewing MD STARnet data, the pediatric rare disease designation criteria, and discussing whether Duchenne met the statute and the protocol for informing the companies of the Agencies’ determination…and it did!


And what’s the moral of this story?

Without YOU – our Duchenne community:

  • There would have been no MD-CARE Act, which would have meant no MD STARnet data.
  • We wouldn’t have the wonderfully rich relationships with our congressional champions and federal agency partners which we build on daily.
  • We wouldn’t have DuchenneConnect, which evolves and grows richer daily.
  • We wouldn’t have a rapidly developing therapy pipeline – with potential approvals in 2015!


And – as for the Rare Pediatric Disease Priority Review Voucher program…

Remember the 21st Century Cures Act that passed the House of Representatives this summer and that our community rallied for during the Connect Conference? Well, it included a provision that would extended the PRV for another three years (until December of 2018). Once again our community has been working to keep this critical industry incentive accessible to the companies working so hard on behalf of our community. And – if recent weeks are any indication – the pediatric rare disease designation is an incentive that matters to the industry sponsors in our Duchenne space.


So, the next time you read a release from one of our industry partners that announces another FDA Pediatric Rare Disease Designation – or even better yet – the awarding of a Pediatric Rare Disease Voucher, know that YOUR outreach/ Hill visits/ participation helped make that possible. 

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