Presentation (CTI)

OBJECTIVES: Individualised therapies including adeno-associated virus vectors, antisense oligonucleotides, small interfering RNA and gene editing technologies (e.g., CRISPR/Cas-9) have the potential to be reprogrammable across multiple conditions (“platform technologies”). This may accelerate innovation, particularly for rare diseases. However, rare diseases pose complex challenges for health technology assessment (HTA) decision-making. These challenges could become more prominent if the advent of platform technologies increases the number of individualised therapies seeking reimbursement. This study examines the specific challenges they might pose for HTA organisations.
METHODS: HTA documents and published economic evaluations of individualised therapies were identified systematically and assessed against a prespecified selection criteria. From the HTA reports, uncertainties discussed by committees were extracted. From the published economic evaluations, author-reported challenges and economic modelling approaches (e.g., model type, cycle length and model inputs) were extracted. Themes were drawn to assess the challenges individualised therapies pose for HTA. At each review stage, a 10% quality assessment check was conducted by a second reviewer.
RESULTS: A total of 69 HTA reports, and 27 journal articles were identified. All studies reported cost-utility analyses. Identified challenges to decision-making included evidence from small single-arm trials, short trials, lack of long-term efficacy data, use of surrogate outcomes, methodology used in indirect treatment comparisons, economic modelling structure, identifying economic model inputs, lack of health-related quality of life data and absence of service redesign costs. These challenges led to uncertainty in the cost-effectiveness assessment of individualised therapies.
CONCLUSIONS: The assessment of individualised therapies highlights novel challenges for HTA decision-making. The anticipated scale of individualised therapy development, and their potentially transformative effect on rare diseases, means HTA organisations may seek to consider the appropriateness and proportionality of their evaluation processes, in order to avoid unwarranted delay to access. A NICE ‘HTA Lab’ project looking at individualised therapies is exploring these challenges.