![]() ![]() They require reassurance that the prices being charged are not ‘excessive’. However, health care decision-makers are still likely to find themselves asked to provide funding for orphan drugs on criteria other than the ‘value-based’ price whether at standard or adjusted thresholds. severity, unmet need) that justify departing from standard value for money criteria (Drummond et al., 2007) and recent work has sought to show why risk aversion might lead us to want higher thresholds for treatments for catastrophic diseases (Lakdawalla and Phelps, 2020, Garrison et al., 2019). Alternatively, some argue that there may be characteristics of orphan drugs (i.e. ![]() Many health economists argue there is no justification for a premium for ‘rarity’ orphan drugs should not be judged any differently from ‘value-based’ criteria applied to drugs for common diseases (McCabe, Claxton and Tsuchiya, 2005). The high cost of drugs for rare diseases (often known as orphan drugs) has generated considerable debate. What is the maximum allowable price society should be willing to pay for an orphan drug based on allowing a reasonable rate of return? To answer this question, Berdud, Drummond and Towse (2020) propose a method that adjusts an established payer/HTA body incremental cost-effectiveness threshold (CET) to take account of differences in patient populations and costs of research and development in order to generate prices that sustain rates of return from investments in developing orphan drugs that are no greater than the industry average. ![]()
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