Health Technology Assessment Decisions in Immuno-Oncology Therapies: Results, Rationales, and Trends


      Immuno-oncology (IO) therapies have emerged as a promising drug class in cancer treatment with targeted mechanisms of action. Some IO therapies have demonstrated durable clinical responses beyond conventional standards of care. As the IO landscape continues to expand, it becomes important to assess their value with regard to clinical benefit and costs. Health technology assessments (HTAs) aim to manage access and expense of new treatments to provide cost-effective treatment options. The objective of this analysis was to evaluate recent IO HTA decisions and their rationale to identify trends in selected countries.


      HTA surveillance was conducted for Australia, Canada, France, Germany, and the United Kingdom (UK) from January 1, 2012 to April 30, 2017 (64 months). HTAs for IO therapies were evaluated by therapeutic area, decision, and rationale for each decision. Decisions were categorized as favorable, unfavorable, mixed (both favorable and unfavorable), or neutral (eg, deferred decision).


      41 IO HTAs were published during the study timeframe: 23 (56%) in melanoma, 12 (29%) in non-small cell lung cancer, and 6 (15%) in renal cell carcinoma. Across HTAs examined, 26 (63%) decisions were favorable, 11 (27%) were unfavorable, 2 (5%) were mixed, and 2 (5%) were neutral. All decisions were deemed favorable in France (6/6, 100%) and the UK (8/8, 100%), followed by Canada (6/7, 86%), Germany (4/9, 44%), and Australia (2/11, 18%). Favorable decisions stemmed from strong evidence of clinical benefit and high unmet need, whereas unfavorable decisions were typically due to inappropriate comparators or unacceptable incremental cost-effectiveness ratios. Mixed and neutral decisions were heavily dependent on effectiveness in specific subpopulations, including patients previously treated, patients with specific gene mutations, or elderly patients.


      As new IO therapies emerge and attain additional indications, it is critical that HTA submissions provide strong clinical and pharmacoeconomic evidence to achieve access.