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European authorities have announced a proposal for a framework to support not-for-profit organizations and academia in repurposing authorized medicines.
This column reported in August of a push by the current Slovenian presidency of the European Union to drug repurposing—and now, just weeks before the end of its term in the EU's revolving chair, its ambitions have received a concrete endorsement in the shape of the formal launch of an EU pilot program aimed specifically at testing the concept further. Right at the end of October, the European Commission, the European Medicines Agency and the Heads of Medicines Agencies announced "a proposal for a framework to support not-for-profit organizations and academia in repurposing authorized medicines."
Presented as a way of making new treatment options available to patients, the scheme aims at assisting evidence generation on an established medicine in a new indication, with the goal of create a data package robust enough to support a future market authorization application. It will apply to conditions to candidate medicines that are no longer covered by any intellectual property protection, and that could respond to unmet need with significant potential public health benefits. And eligible beneficiaries might be academic units, charities, patient organizations, learned societies, research funders or payers, explains the EU.
Slovenia's principal interest in the concept has been overtly the desire to open an avenue to cheaper medicines. The formal EU approach in this scheme, however, is more nuanced. Ideally, in official EU thinking, it could provide some constructive response to the dilemmas posed by extensive off-label prescribing. "The absence of a bespoke regulatory route for, off-patent, authorized medicines already on the market to be developed for new uses and reach formal regulatory approval sometimes poses problems to physicians, patients and health systems," says the background documentation to the scheme. "Well-established evidence based on off-label use is not converted into on-label use", with possible "negative consequences for patient safety and treatment options." Additionally, "potentially useful new or established non-approved indications will be hampered if the medicine is suddenly withdrawn from the market."
The principal assistance available through the pilot will be in terms of scientific advice. Eligible academic sponsors developing orphan medicines will automatically benefit from a fee waiver. And additional fee waivers will be granted to a subset of selected applications taking into account the extent of the expected public health benefits and the strength of the evidence to substantiate the promise held by the proposal.
As observed in this column in August, there is a long history to this ambition. The concept of support for a repurposing framework features in EMA's Regulatory Science Strategy to 2025 and has been extensively discussed in the European Commission’s expert group on safe and timely access to medicines for patients, STAMP, where representatives of EU member states, EMA and stakeholders from not-for-profit organizations, patients, healthcare professionals, industry, health technology assessment bodies and payers contributed.
But a clearly delineated challenge repeatedly arises whenever attempts are made to turn ambition into reality. As the EU announcement makes clear, any success ultimately depends on the engagement of a pharmaceutical company, and that is far from guaranteed. Non-commercial generation of new evidence "should then be followed by the engagement of the marketing authorization holder/applicant of the concerned medicinal products applying for the new indication through standard regulatory processes." Not "will be followed". Just "should be followed".
The pilot will run until the completion of scientific advice for the selected repurposing candidate projects, says the EU announcement—adding that it will continue "optimally until the filing of an application by a pharmaceutical company for the new indication." It has been immediately and warmly welcomed by non-commercial organizations—notably the Anti-Cancer Fund, a longstanding proponent of repurposing. "We are very glad that EMA and the national medicines agencies are now ready to provide regulatory support, especially scientific advice, to not-for-profit organizations and academia who are willing to investigate existing drugs for new indications," said its managing director, Lydie Meheus. But it has also received a qualified welcome from some industry organizations. The generic industry's Medicines for Europe, which is active in the development of value-added medicines, saw it as "a great first step", but insisted that it "must become a part of a broader sustainable repurposing ecosystem" – by which it means some regulatory simplification, and better pricing and reimbursement policies.
The pilot may prove that repurposing really can deliver. Or it may indicate the contrary. For European policymakers, the scheme provides a convenient way of deferring any decisions—and maybe making it possible, in the end, to drop the discussion completely, bring to an end what some regard as a futile distraction.