Considering potential solutions for limitations and challenges in the health economic evaluation of gene therapies

ABSTRACT

Introduction: The limited evidence in the clinical trials of gene therapies (GTs) posed substantial challenges for a reliable health technology assessment (HTA). This paper provides insights into the relationship between the background of diseases and the health economics assessment of GTs.Areas covered: The impacts of differentiated severity and unmet needs of genetic diseases, on the economic analysis of GTs, were discussed.Expert opinion: GTs offer a potential cure or significant clinical improvement, while limitations in clinical evidence constitute major obstacles for a robust assessment of clinical effectiveness and economic outcomes. This uncertainty may be balanced by the severity of the targeted condition and the associated unmet needs, thus leading to a relatively higher acceptance for GTs. Overtime, HTA agencies will become more demanding on comprehensive evidence of long-term effectiveness. With a growing number of GTs on the horizon, to what extent the unmet needs of previously devastating diseases will be fulfilled remain unclear. Nonetheless, comparative studies, either with a historical control group or existing treatments, will be necessary to demonstrate the additional benefits associated with GTs.

KEYWORDS:

• Gene therapies
• health technology assessment
• cost effectiveness analysis
• rare diseases

Article highlights

• Genetic diseases targeted by the gene therapies showed varying magnitudes of severity and unmet need measured by the existence of alternative treatments.

• The potentials of gene therapies to alleviate the disease severity and fulfil the unmet needs will largely determine how their values will be assessed and appreciated by the health technology assessment (HTA) agencies.

• Although the HTA and economic analyses of gene therapies are facing substantial challenges due to the insufficiency of robust evidence on long-term outcome, they are highly likely to be endorsed once being perceived as potentially curative treatments for life-threatening diseases out of effective treatments.

• With the growing number of gene therapies for more prevalent diseases will be approved in the coming years, the sustainability of healthcare affordability is at stake. Accordingly, it is questionable if the payers’ willing to pay for gene therapies will be altered in the future.

This paper summarizes and discusses the recommendations for the cost-effectiveness analysis for gene therapies. There is agreement that urgent actions are needed to standardize the methodology for assessing the disease severity and unmet needs. Such efforts will enhance the transparency in the value assessment of gene therapies, thus supporting a more informed HTA and reimbursement decision-making.

Declaration of interest

M Pochopień and E Clay are employees of Creativ-Ceutical. T Qui, S Aballea and M Toumi report personal fees from Creativ-Ceutical outside the submitted work. Creativ-Ceutical is an international life science consulting firm dedicated to supporting the life science industry, health authorities and non-profit organizations in strategic decision making. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.