The European Parliament’s plan to base data protection on unmet medical needs has raised concerns, with industry representatives and politicians calling it “ethically difficult” and admitting it has made them “very worried.”
Experts commented on the proposal during an online event hosted by Politico on Friday, two days after the Parliament voted to adopt the unmet need provision as part of a planned package of changes to the European Commission’s draft pharmaceutical legislation. Nathalie Moll, director general at the European Federation of Pharmaceutical Industries and Associations (EFPIA), was on the panel and commented on the Parliament’s proposals. Moll said the changes improve on the original draft but still fall short of providing the predictability that industry wants.
“There’s still a reduction in the incentives and there’s a modulation based on a concept of high unmet medical need, which I find even ethically difficult to evaluate when you’re talking about any disease but especially rare diseases. How do you classify which one has a higher unmet patient need than another?” Moll said.
The classification of unmet need matters under the Parliament’s proposal because it affects how long a drug will have data protection and, by extension, could influence which diseases biopharma companies invest in. The Parliament wants to give an additional year of data protection to medicines that address an unmet medical need. Orphan drugs that address a high unmet medical need could get up to 11 years of market exclusivity.
Kathleen Depoorter, a Belgian politician, joined Moll in raising concerns about tying exclusivity to unmet medical need. Depoorter, who is in touch with the Belgian presidency of the Council of the European Union, said she is “very worried.” The concerns are underpinned by the experience of Belgian Minister of Health Frank Vandenbroucke, who has been investigating unmet medical and social needs.
“He has presented [the research] to the European Council and, as I heard, there were only 30 responses,” Depoorter said. “If you have to base your definition of unmet medical needs on 30 or 50 cases, that’s not enough. So, I’m really worried about the criteria that will be used to define these unmet medical needs and unmet social needs. This will have a huge impact among patients.”
Moll discussed how the proposals could influence R&D priorities and ultimately the drugs that come to market. The European Medicines Agency already has a definition of medical need that informs whether a candidate is eligible for fast-track approval. “That makes sense,” Moll said, but using unmet medical need to determine data protection “is very confusing.”
“How do you know what the unmet medical need is 15 years in advance, when you’re starting to develop a product in a certain disease?” Moll said. “The objective is good but 20 years ago we didn’t know we’d have COVID and that it would be an unmet medical need. I think we’re trying to do too many things in one and we need to remember that what we’re looking at is an ecosystem that needs predictability.”
Michelangelo Mancuso, a specialist at the European reference network (ERN) for neuromuscular disease, laid out another issue with assessing whether a disease is a high unmet medical need. Scientists working on rare diseases often lack a clear picture of the natural history of a condition, particularly when very small numbers of people have the disease. The knowledge gaps complicate attempts to assess unmet needs. Mancuso is working to build a clearer picture of how rare diseases affect the lives of patients.
“One of the most important things that we should develop and know better is what is really bothersome for our patients, which are the most severe symptoms or signs that they experience during the disease course,” Mancuso said. “That’s one of the reasons why ERNs are developing all these patient reported outcome measures in order to learn from the patients the unmet medical needs.”
Moll said it will be “crucial” to involve patients in the definition of high unmet medical need. The EFPIA director said the push for more involvement of patients is one of the areas that the Parliament has improved on the Commission’s draft legislation, explaining that a bed-bound patient who is expected to live five years may value mobility more than increased survival.
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