FDA orphan drug updates at NORD Summit

Officials from the US Food and Drug Administration (FDA) provided updates on the agency’s efforts to support the development of medical products for rare diseases at the National Organization for Rare Disorders (NORD) Summit this week.
 
In his keynote address on Thursday, FDA Commissioner Stephen Hahn said that FDA had already approved 51 orphan indications through July of this year and acknowledged that the agency’s Office of Orphan Products Development (OOPD) has seen, “Large increases in the number of requests for orphan drug designation and rare pediatric disease designation.”
 
Hahn also touched on recent developments related to the agency’s Orphan Products Grants Program, which issued six grants worth $16 million over four years to support clinical trials of products for the treatment of prevention of rare diseases. In addition to the new trials being supported, FDA is providing additional funding to existing grantees to cover unexpected costs incurred due to the COVID-19 pandemic.
 
Additionally, Hahn discussed two new initiatives, one dubbed Orphan Grantees Unite, which, “provides a forum for grantees to share their experiences and challenges in support of common solutions,” and an upcoming effort to support new medical technologies for pediatric patients.
 
“The vision of [System of Hospitals for Innovation in Pediatrics] SHIP is for a national ecosystem that will focus on safe innovation by optimally engaging our nation’s pediatric health systems that have the breadth and depth of infrastructure to support safe evaluation of technologies for children,” Hahn said, adding that FDA will host a public workshop on the initiative in February.
 
Speaking on a separate panel on regulatory science, OOPD Director Janet Maynard also announced two new programs stemming from FDA’s Orphan Drug Modernization Plan.
 
What is new, according to Maynard, is that the agency, “Will move from a paper-based process to a new cloud-based online submission portal. The new online portal will be available this year and will allow sponsors to submit orphan drug designations electronically."
 
Maynard also said her office will implement a new internal workflow management tool to, “Establish and facilitate a defined set of tasks necessary in the review of an orphan designation submission,” to help increase the efficiency of its reviews.
 
Peter Marks, director of the Center for Biologics Evaluation and Research, discussed how COVID-19 has impacted the conduct of clinical trials. “We’re observing patients, some of whom had biopsies as endpoints or other invasive procedures and some of those didn’t happen,” Marks said. “Our concern is that ultimately, after the pandemic … almost like after a hurricane passes, you don’t see all the damage till the weather is clear.”
 
“Some of the things we’ve learned is that complex clinical trials during the time of COVID-19 where there are complex assessments — frequent assessments — those have been challenging. On the other hand, trials that have been more streamlined, trials that relied on remote assessments have been able to continue, and so I think that some of what will happen coming out of COVID-19 is that we may be able to transition into trials that may be more patient friendly,” Marks said.
 
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