PMU9 DO NICE APPRAISAL PROCESSES REWARD INNOVATIVE AND CURATIVE THERAPIES? AN ASSESSMENT OF CURRENT AND POTENTIAL FUTURE TECHNOLOGY APPRAISALS FOR RARE DISEASES

Abstract

To assess NICE processes for rare diseases: Highly Specialised Technologies (HST) committee appraisal, weightings of quality-adjusted life years (QALY), and differential discounting. We assessed current and proposed HST and Single Technology Assessment (STA) evaluated technologies for adherence to the HST qualification criteria, eligible patient population/prevalence, undiscounted survival benefit and application of the QALY weighting. We also assessed a targeted selection of pipeline therapies with the potential for substantial survival and quality of life gain. Using published literature, we identified which therapies might qualify for the HST evaluation and, of those, which qualify for the QALY weighting and/or differential discounting. We simulated the impact of these processes on a hypothetical drug with a large survival benefit in a rare disease. To date, eight therapies have been recommended out of 20 referred to the NICE HST committee. Of those recommended, only one qualified for QALY weighting. For the others, the expected QALY benefit did not exceed the 10 QALY threshold. Of 11 selected pipeline therapies, only three (27%) would be expected to meet HST criteria and zero would meet thresholds for QALY weighting. In a hypothetical scenario where a drug for a rare condition improved survival by 20 years with an average utility score of 0.6, failure to qualify for QALY weighting/HST process would reduce the cost-effective price by more than £1M. NICE HST approaches are intended to make allowance for rare, debilitating conditions where treatments may not meet traditional cost-effectiveness thresholds. In practice, however, these allowances are ambiguous and evaluated technologies rarely qualify. Future innovative therapies may deliver substantial benefits, however, access may be limited if traditional thresholds are applied. Over time, this may signal to industry that without extreme survival benefit, treatments for rare disease are unattractive investments.