Market access of gene therapies in a nutshell
challenges in a nutshell
With the promise of one-time therapy, gene therapies are an attractive and innovative way to treat patients. For Advanced Therapy Medicinal Products (ATMPs) addressing an unmet medical need or providing novel therapies for serious or life-threatening diseases, the FDA and EMA have implemented a regulatory framework to expedite their market approval. Regardless of the programs including Fast Track, Breakthrough Therapy or Priority Medicine (PRIME) to accelerate the approval process, gene therapies undergo vigorous scrutiny by regulatory bodies. This is due to the size and physiochemical properties of these biologics that are different from the traditional small molecules manufactured. Hence, measures were adopted by regulatory bodies to ensure the safety of ATPMs not only in the clinical trials but beyond market authorization.
Besides these stringent measures, gene therapies also face several barriers pertaining to their market authorization. Based on the precedents available to date, three main critical issues affecting the market entry of gene therapies could be identified. The first obstacle is about generating clinical evidence. As gene therapy products (GTPs) are often orphan drugs, they are faced with the limitation of small population sizes. Together with ethical issues arising with placebo controls for instance due to invasiveness (e.g. Glybera®, 60 painful injections per one-time treatment), these practical obstacles hinder the use of randomized controlled trials (RCTs). Furthermore, the inability to clearly assess the potential therapeutic value of GTPs is another challenge. In the absence of long-term safety data, it is difficult to effectively evaluate the incremental clinical gains and the long-term durability of the treatment benefit for gene therapies. This uncertainty about the real value of GTPs can result in budget constraints by payers. Lastly, the obstacle of the affordability of GTPs is the biggest concern. Indeed, the exorbitant prices of these drugs (e.g. the price of ZyntegloTM is set at 1.575.000 €) force payers to come up with innovative reimbursement solutions.
In our latest White Paper "Successful market access for gene therapies – strategic challenges and possible solutions", we describe and discuss in depth challenges related to the market authorization of gene therapies and present possible solutions to unresolved problems. Please feel free to request the White Paper free of charge further down on this page.
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