European patient groups said the definition of “unmet medical need” under the EU’s proposed pharmaceutical reform is too narrow and doesn’t capture the patient perspective on unmet needs. Under the proposed reforms, treatments that address unmet medical needs would be granted additional market exclusivity to incentivize product development.
Speaking at DIA Europe on 13 March, Maria Boulos of the European Medicines Agency (EMA), said that that these definitions are now undergoing “extensive discussions” in the European Parliament ahead of a vote on the proposal by the Committee on Environment, Public Health and Food Safety (ENVI) on Tuesday. Other panelists at the session discussed challenges in applying the term to medicines.
The overhaul of the EU pharmaceutical legislation, which includes the definition of unmet medical need, was adopted by the European Commission in April 2023 and aims to increase patient access to medicines, among other goals. The provision specifies that a medicine meets the definition of an unmet need if it treats a “life threatening or severely debilitating condition” for which there is no treatment and produces a “meaningful reduction in disease morbidity or mortality.” A product would be accorded as meeting a high unmet medical need if it treats a rare disease for which no treatment exists or is considered an “exceptional therapeutic advancement.”
A product meets an unmet need the product gets six more months of market exclusivity under the Commission’s proposal or 12 months under a compromise amendment proposed by the European Parliament. Orphan products would be awarded an additional year of marketing exclusivity under the Commission’s proposal and an additional two years of exclusivity under the European Parliament compromise amendment.
Currently, an unmet medical need is defined as a condition for which “there exists no satisfactory method for diagnosis, prevention or treatment” or is considered a “major therapeutic advantage” over current treatments.
Discussions ongoing
Maria Boulos of EMA said the specifics of the definition are important because they support access tools under the priority medicines (PRIME) program, accelerated assessments, and conditional marketing authorization.
The European Parliament’s health committee is supposed to debate and vote on changes to the proposed pharmaceutical legislation on 19 March, including provisions that would increase market exclusivity and regulatory data protection above the Commission’s proposal. (RELATED: European Parliament committee propose changes to pharma legislation, Regulatory Focus 15 March 2024)
Definition ignores patient realities
Members of the European patient groups said that the proposal adopts a very narrow view of unmet medical need and does not represent the patient perspective.
Claudia Louati of the European Patients’ Forum in Belgium said, “We are a little bit concerned that the definition from the EC is a bit restrictive and does not take into account some aspects that are very important from a patient perspective such as the quality of life, or the impact of treatment on every day life.”
“There are no metrics to measure the quality of life, and an unmet patient need may be a more appropriate term,” she said.
She said that “unmet medical needs cover many different realities for patients.” She added that this is “an emotional topic because each patient has their own definition of an unmet medical need.”
Tina Taube, executive director of public affairs for the European Federation of Pharmaceutical Industries and Associations (EFPIA), asserted that the proposals could potentially exclude some treatments for chronic diseases that may not meet the definition of an unmet medical. Taube said that “we should not narrowly define an unmet medical need.”
She cited a report released for EFPIA by EXON Consultancy which notes that of the 111 non-orphan medicines authorized between 2019 and 2022, only 20 would probably meet the proposed criteria for unmet medical need and that medicines that treat migraines, cardiovascular disease, and diabetes may not qualify.
Thomas Metcalfe, of F. Hoffmann-La Roche, said that most drug development activities can be considered as addressing an unmet medical need. “I think we all have to appreciate that medicines development is a highly regulated activity and I think that you would struggle to get an approval for a clinical trial to test the medicine in a patient population without there being some concept of an unmet need.”
He added that “nobody who is doing this work seriously embarks on what is an extremely expensive and complex activity without there being an unmet medical need at the heart of this.”
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