The market of rare disease drugs

Rare diseases are surely among the most interesting therapeutic areas for the pharmaceutical industry, and they have been since the 1990s, when US-based Genzyme proposed a treatment for Gaucher’s disease at up to $300,000. According to a recent report by EvaluatePharma, the drug market includes 4,000 orphan drugs approved since the 1980s, with more than 300 last year, and generates approximately $114bn. This proportion accounts for 16% of the global pharmaceutical market and will rise to 21% by 2022. There are a number of reasons why the industry invests so heavily in orphan drugs. Firstly, those companies which develop and commercialize such drugs receive higher government incentives and are granted –on average – quicker and easier regulatory approval than for non-orphan drugs. Secondly, new technologies have made production easier and cheaper. Moreover, cooperation and networking among patients, physicians and the pharmaceutical industry have been increasing over the last years, which has made drug development easier. However, supervisory authorities have increased their scrutiny of orphan drugs, as concerns have spread that many of these highly expensive drugs only modestly improve patients’ quality of life. For instance, the NHS will thoroughly check the effectiveness of drugs which cost more than GPB100,000 per treatment. This is the case of Alexion Pharmaceutical’s Kanuma, for lysosomal acid lipase deficiency, sold at £500,000. Its clinical benefits arguably do not justify the price. Alexion also sells Soliris, to treat a rare blood disease, at $700,000 per treatment in the US. In the same US, a health insurer has recently refused to pay for Sarepta’s Exondys51 for Duchenne muscular dystrophy. (Source FT)