Pricing implications for cell and gene therapies

Pricing implications for cell and gene therapies during a global pandemic


Cell and gene therapies – life-saving opportunity for many patients

Cell and gene therapies are some of the greatest examples of how far the innovation in pharmaceutical and biotech research has reached. Thеse breakthrough technologies provide a life-saving treatment opportunity for some people who suffer from rare diseases and cancers – conditions with a significant unmet need. This is one of the reasons why these products are typically associated with exorbitant price tags ranging from $250,000 to $1,000,000 per patient per year. However, at times of a global pandemic due to COVID-19 resulting in an overwhelming healthcare and financial burden for governments across the whole globe, what are the pricing and reimbursement (P&R) implications of the cell and gene therapies for the healthcare systems?

Cell and gene therapies are classified as advanced therapy medicinal products (ATMPs) by the European Medicines Agency (EMA). Following their regulatory approval, to ensure patient access, ATMPs need to be assessed by a health technology assessment (HTA). The value proposition of these drugs appears to be attractive to payers due to their curative potential and improvement in health outcomes represented by longer survival or complete remission.

Uncertainty associated with ATMPs

However, there is some uncertainty in the clinical data as the clinical studies of these products are often single-arm, measured over short periods of time and enrol only a limited number of patients. Another point of uncertainty is around the safety of cell and gene therapies because some of them have demonstrated alarming, possibly life-threatening adverse events in their trials. For instance, the incidence of cytokine release syndrome in patients with acute lymphoblastic leukaemia (ALL) and non-Hodgkin lymphoma (NHL) with Kymriah is estimated at 77%. For this reason, long-term follow-up clinical trials and real-world evidence studies with them have been initiated but it will take years until their completion.

To mitigate the uncertainty associated with the long-term effectiveness and safety of cell and gene therapies, HTA bodies have implemented different mechanisms and mandated the collection additional data. For example, early patient access schemes have been made available for Kymriah and Yescarta in France: Temporary Authorisation for Use (ATU) and the UK: Cancer Drug Fund (CDF). The HTA bodies of France and Germany have asked for reassessment of these breakthrough products, within 1-2 years once some additional long-term data has been collected.

Reimbursement challenges during times of a global pandemic

Cell and gene therapies pose a significant challenge to payers due to their astronomical cost. The manufacturers of these products face the hurdle of setting a new price benchmark among payers. Although they are a one-off treatment, hence a one-off payment for a relatively small number of patients, that still represents a substantial upfront payment for healthcare systems. That is why there have been innovations in the payment models in some countries. In Germany, for example, a nationwide pay-for-performance contract for Kymriah requires the manufacturer to partially reimburse costs of treatment if a patient dies of their condition within a given period of time.

The global pandemic due to COVID-19 certainly will have an impact on the P&R landscape in 2021. Healthcare budgets faced an immediate squeeze from COVID-19, and probably longer-term pressures due to global economic recession are yet to be seen. Payers will be budget pressured and may raise the bar in terms of evidence for access to new medicines, including breakthrough and game-changing products, such as cell and gene therapies. Manufacturers of these therapies are likely to face greater P&R challenges derived from the growing pushback from payers. This, coupled with the lack of extensive experience with ATMPs among HTA bodies necessitates manufacturers to come up with innovative payment and risk-sharing methods (e.g. patient access schemes, pay-for-performance agreements etc.) to mitigate the ever growing uncertainty and unwillingness to pay from payers.

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