Using analogue data to substantiate long-term durability of gene therapies: a narrative review.

The number of gene therapies in clinical trials and moving toward licensure is increasing. Most gene therapies are designed to achieve long-term effects, but at licensure the data to support claims of long-term durability are often limited, as long-term monitoring studies are often part of post-approval commitments by companies. Health technology assessors must therefore assess the potential for the long-term durability of a product and the potential cost-effectiveness based on the data available.

The use of innovative payment mechanisms for gene therapies in Europe and the USA.

Innovative reimbursement mechanisms have long been considered potential solutions to the data uncertainty associated with one-off, high-value gene therapies that have long-term therapeutic potential, combined with limited supporting evidence at launch. The launches of increasing numbers of such gene therapies in Europe and the USA in the past 5 years provide valuable exemplars of how innovative reimbursement mechanisms are used by healthcare system decision makers in practice. This review details the use of such reimbursement schemes for recently launched gene therapies in key European countries and the USA, and shows that they are more widespread in Europe than in the USA.

Clinical Adoption of Advanced Therapies: Challenges and Opportunities.

As the cell and gene therapy field matures the powerful therapeutic potential of these innovative therapies is starting to be shown, particularly in the fields of oncology and childhood immune deficiency diseases. However, as more therapies enter late stage clinical trials, advances and innovation are required in manufacturing, logistics, regulation, reimbursement and the healthcare setting to ensure that systems are in place to support wider clinical adoption of these promising treatments. A window of opportunity exists to implement new methodologies for best practice in both the ability to manufacture products reproducibly at scale, as well as ensuring healthcare systems are not overwhelmed by the variety and complexity of these new therapies and the additional burden they will place on already stretched facilities.

Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries.

: The experience of Kymriah® and Yescarta® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. : To provide an overview of the reimbursement schemes used for Kymriah® and Yescarta® in France, Germany, Italy, Spain, and the UK (EU5) as per the final quarter of 2019; to identify challenges and derive learnings for future product launches. Methodology: Secondary research, complemented by primary research with key market access stakeholders.

Upgrading the SACT dataset and EBMT registry to enable outcomes-based reimbursement in oncology in England: a gap analysis and top-level cost estimate.

: Outcomes-based reimbursement (OBR) can reduce decision uncertainty and accelerate patient access to cell and gene therapies, however, OBR is rarely applied in practice in England. Oncology is the therapy area with the most cell and gene therapies in late-stage development, and the Systemic Anti-Cancer Therapy (SACT) dataset and The European Society for Blood and Marrow Transplantation (EBMT) registry are two data collection infrastructures that could potentially act as conduits for implementing OBR in cancer in England. : To perform a gap analysis to identify the key requirements for upgrading the SACT and EBMT databases for the purposes of enabling OBR, and a top-level estimation of how much this upgrade may cost, using either a manual (staff-heavy) workaround or part automation (technology-heavy) approach.