Payer and Implementation Challenges with Advanced Therapy Medicinal Products (ATMPs)
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CURRENT OPINION
Payer and Implementation Challenges with Advanced Therapy Medicinal Products (ATMPs) John Spoors1,2 · Alec Miners1 · John Cairns1 · Danny Palnoch2 · Ash Summerfield2 · Joanne McEntee3 · Sheena Vithlani3
© Springer Nature Switzerland AG 2020
Abstract Advanced therapy medicinal products (ATMPs) are a dynamic and current topic for healthcare systems, with new products progressing to market at an increasing rate. ATMPs highlight the growing gap between payer and regulator requirements; the limited evidence base combined with pressure to implement rapidly is exacerbating the clinical and financial uncertainties associated with these products. There are a number of key uncertainties with ATMPs related to implementation and healthcare planning—these uncertainties at the time of evaluation have the ability to change the value proposition of products. ATMPs also have the potential to reduce the amount of net health gain available to healthcare systems, and evaluators should consider the opportunity cost when seeking to accelerate access to one-off therapies with a limited clinical evidence base. Therefore, ATMPs have the potential to transform clinical care pathways, but implementation challenges and application of key health economic principles may highlight the requirement to exercise caution.
Key Points Advanced therapy medicinal products (ATMPs) have transformative potential, and healthcare systems are preparing for their evaluation and implementation. However, a balance must be struck between accelerating access to these therapies and the uncertainties/risk associated with their often-limited evidence base and implementation requirements. As excitement around these therapies builds, it is vital that healthcare systems consider the opportunity cost when accelerating evaluation and implementation, a phenomenon that is amplified if a one-off product does not deliver the health benefit promised at launch.
* John Spoors [email protected] 1
London School of Hygiene and Tropical Medicine, London, UK
2
NHS England and NHS Improvement Medicines Analysis Team, London, UK
3
Specialist Pharmacy Service, London, UK
1 Introduction Advanced therapy medicinal products (ATMPs) are a dynamic and current topic for healthcare systems, with new products progressing to market at an increasing rate [1]. As ATMPs begin to emerge in the marketplace, expectations that these therapies have the potential to offer transformative benefits to patients are high. Three types of ATMPs exist: gene therapies, somatic-cell therapies and tissue engineered therapies [2]. The US FDA predicts that, by 2025, it will be approving 10–20 cell and gene therapies per year [3]. A good comparison is the rapid development of antibody products in the late 1990s and the mainstreaming of biological therapy as a core element of therapeutic regimens. Key recent examples of approved gene therapies include (1) the ex vivo chimeric antigen receptor T cell therapies axicabtagene ciloleucel [4] and tisagenlecleucel [5] for the treatment
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