ATMPs: Global regulators eye harmonization for ultra-orphan products

Regulatory NewsRegulatory News | 08 April 2021 |  By 

Top regulators from the US, EU, UK and Japan view harmonization as a critical factor in enabling the development of advanced therapy medicinal products (ATMPs) for ultra-rare diseases.
Particularly for cell and gene therapies targeting patient populations numbering in the hundreds worldwide, commercial viability and the feasibility of clinical trials can be complicated by jurisdictional differences in regulatory requirements. Such challenges were the topic of conversation during a panel discussion with regulators at the Alliance for Regenerative Medicines’ Meeting on the Med on Wednesday.
“There are plenty of diseases that the patient population is so small in the United States that it’s not commercially viable. It’s so small in the European Union that it’s not commercially viable, it’s probably too small in the United Kingdom and too small in Japan. The problem is that if a sponsor has to negotiate different regulatory environments in four different locations. that becomes untenable if there are different requirements,” said Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the US Food and Drug Administration (FDA).
Marks said that harmonizing regulatory requirements, especially for diseases with small patient populations, could lead to more treatments for previously untreatable diseases. “But right now we’re not going to get there because it’s very hard to have commercial viability in one location and people are kind of scared of going across different boundaries,” he added.
While broader harmonization could be beneficial, Marks said that regulators could focus on ultra-orphan indications first to get their toes wet.
“I think that this is an excellent point, in ultra-orphan disease, you can never regulate this sufficiently in one jurisdiction and if there are different requirements then there is indeed a problem,” said Christian Schneider, interim chief scientific officer at the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).
Asked whether there is an appropriate forum to kick start discussions about harmonization in this area, Ana Hidalgo-Simon, head of advanced therapies at the European Medicines Agency (EMA) said that regulators are having more and more discussion, especially at the technical level, about ATMPs.
“At the end of the day, even though we are all asking for the same things … we all have to make it fit into a slightly different legislative system,” Hidalgo-Simon said, adding that pursuing greater harmonization in the area of ATMPs for ultra-rare diseases is “excellent as a first step.”
“But anything that we gain in one area that we can prove first that we can do it and second that it is good for the patients, then it’s our role to push it to the next step forward,” she said.
Hidalgo-Simon and Marks pointed out that their agencies do have discussions on these topics and that companies can have conversations with both agencies simultaneously.
“COVID has given us a little bit of a push in that direction as well, in the sense that I think we are collaborating more than we thought was possible to only a year ago,” Hidalgo-Simon said.
Marks added that regulators need to make companies more aware of the opportunities for engagement with multiple regulators. “There’s nothing that says we at FDA can’t have the EMA — or any other regulator for that matter — join in on one of our early regulatory calls with the company so that everyone is hearing the same thing. That way there can be alignment early on,” he said, adding that the more alignment there is between regulators the less uncertainty there will be during development.
Meeting on the Med


© 2023 Regulatory Affairs Professionals Society.

Tags: ATMPs, EU, Japan, UK, US

Discover more of what matters to you

No taxonomy