Cell and gene therapies, also known as advanced therapy medicinal products (ATMPs), are potentially life changing. For patients with rare diseases, they extend the hope of a longer, healthier life and even of a cure. But these therapies are exceptionally expensive with up-front costs ranging from $500,000 to $2 million. Additionally, some incur heavy ongoing costs throughout the life of the patient.
For health technology assessment (HTA) organisations – which must balance clinical effectiveness, safety and efficacy with cost effectiveness, social outcomes and ethical considerations – the decision to support market access for ATMPs is a complex one. Budget constraints mean HTAs and insurers often must make tough decisions balancing the ATMP reimbursement with a reduction in spending elsewhere in the healthcare system. Consequently, therapies that are not viewed as compelling, face rejection. Moreover, the decision-making process can vary from region to region: vastly different decision criteria, for example, are adopted in the UK, the USA and China.