The right recipe for the repurposing omelette
The repurposing of drugs that have been approved for treating other viruses has gained unprecedented media attention during the COVID-19 pandemic. Lawyers persistently criticise second medical use patent claims as pointless, yet pharmaceutical companies continue to obtain and litigate them. Second medical use patents are probably valuable only occasionally and if standing alone, they are an unstable foundation for a business model.
The EU regulatory system provides incentives for repurposing. When a drug is authorised for the first time, the authorisation holder receives eight years of data exclusivity that runs concurrently with ten years of market protection. These two exclusivities are often summarised as 8+2, with the 2 referring to the two years remaining of market protection (after the expiry of data protection).
There’s a specific incentive for companies to repurpose drugs: market protection will be extended to 11 years if, within eight years of authorisation, the drug is repurposed to treat a new indication that brings a significant clinical benefit compared to existing therapies.
The repurposing incentive is even greater for companies that repurpose another company’s drug: they’ll receive the full 8+2 and they’ll be eligible for the +1 if they repurpose the drug again. This means that one incentive for companies to repurpose their own drugs is that if they do not, perhaps their competitors will.
Designation as an “orphan” drug is another option if the drug treats a disease that afflicts five people or fewer in 10,000 in the EU. Common (non-orphan) drugs are being repurposed for orphan indications and orphan drugs are being repurposed for common indications—sometimes even becoming blockbusters, such as Epogen and Procrit (epoetin alfa).
One possible legal dispute pertaining to orphan-common drug repurposing will be the medical plausibility criterion, which was designed to counter the creation of dubious subsets of diseases in order to meet the prevalence criteria. For infectious diseases such as COVID-19 or HIV, the prevalence criterion is a moving target. However, it seems plausible to identify a subset of patients who develop distinct symptoms, such as those who must be placed on a ventilator.
Previous experiences show that effective treatment of new diseases is often obtained not by employing one drug but by using a combination of drugs, as seen in the treatment of HIV infection. Drug authorisations of fixed combinations are considered different from their individual components, attracting the full 8+2 protection. However, the combination must be justified for therapeutic reasons (such as ensuring efficacy).
One problem, however, is that this incentive is available to the rights holders of the other components as well (or any party if the components are all off-patent).
How to get it right
Successful repurposing is contingent upon several aspects of science, policy, law and business strategy. That does not mean that pharma innovators should disregard them. There is no doubt about the value created through repurposing for society and companies. The burning issue is how a suitable amount of value can be captured.
Data is the key in identifying repurposing potential, and the constellation of incentives and exclusivities are important determinants in how to go about developing repurposing opportunities and bring them to the market. Spotting these opportunities revolves not on patent portfolios but on companies’ abilities to gather and analyse data.
Pharmacovigilance reports, off-label use and all kinds of real-world evidence garnished with artificial intelligence and old-fashioned curiosity are key ingredients in this well-baked repurposing omelette. There is a plethora of repurposing avenues to pursue, and timing and understanding of these avenues are key. Scientific and data collaborations will likely be of paramount importance to success.
This should make the bell of open innovation ring at the back of executives’ heads. Sourcing knowledge across organisational barriers to enhance and expedite innovation are the distinctive features of open innovation and likewise of effective and timely repurposing—something obvious to all in the rapid progress towards addressing COVID-19.
Who will prevail in the repurposing of medicinal products? The open and the collaborative who master sourcing of knowledge while securing synergies through patents and regulatory exclusivities.
Jakob Wested is a postdoctoral researcher at Center for Advanced Studies in Biomedical Innovation Law, (CeBIL), University of Copenhagen. He can be contacted at: jakob.blak.wested@jur.ku.dk
John Liddicoat is a senior research associate at the University of Cambridge. He can be contacted at: jel68@cam.ac.uk
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