Can Fast Access Be Achieved for Million-Dollar Therapies? A Review of the Preferred Evidence Package and Access Strategies That Can Help Maximize Access to High-Cost Therapies

OBJECTIVES: With the development of innovative drugs, there has been an increase in drugs priced >US$1 million per treatment. Despite considerable improvements in clinical outcomes, their acquisition cost often limits reimbursement. This research aims to evaluate the acceptability of evidence packages at launch for high-cost drugs and the components of these packages that influence reimbursement recommendations.

METHODS: 13 orphan drugs with >US$1 million list price per treatment at launch were identified via grey literature search. Of these, eight had 21 HTA reports issued by four HTA agencies (HAS, NICE, G-BA and TLV). Relevant data were extracted from the HTA reports to assess the components of the reimbursement strategy and evidence package.

RESULTS: Among the HTA evaluations, 14, four, and three were positive, negative, and restricted recommendations, respectively. NICE and TLV showed higher negative recommendations, mostly driven by the inability to demonstrate cost-effectiveness, due to limitations in the clinical data. 16 of the evaluated clinical trials had a population size of 10-100, which was mostly accepted by HTA agencies given disease prevalence. There were 17 single-arm trials, resulting in failure to demonstrate additional clinical benefit with HAS and G-BA. Overall, HTA agencies were aligned with the outcomes reported, with 18 trials including mortality endpoints that were accepted in the absence of mortality data. Although quality of life may be an important consideration for orphan drugs, <50% of trials included related endpoints.

CONCLUSIONS: Manufacturers often come to market with evidence packages that fail to demonstrate the value for money with payers. The use of endpoints that can be measured in the real-world setting, and early real-world evidence discussions and commitments can help improve access. Moreover, managed entry agreements and innovative pricing solutions should be considered to mitigate evidence generation challenges and ensure that pricing strategies align with clinical and economic value expectations from payers.