Healthcare should make people’s lives better. That fact can hardly be contested. Yet for some patients with rare diseases, commercial interests are dictating who gets to access life-saving treatment and who doesn’t. Pharmaceutical companies have long been driven by global demand and the potential for the highest profits. In the past two decades, the market has exploded: pharma revenues worldwide have exceeded $1tn. For patients with common conditions, this investment in healthcare can only be good news. But the narrow focus of this strategy means that, in the UK, the one in 17 of us who will at some point be affected by a rare condition risk being forgotten.
That is until now. Healthcare providers, driven by a desire to make life-saving treatments more widely available, are increasingly finding new ways of getting them to patients for whom they would have previously been out of reach. Great Ormond Street hospital (Gosh) recently announced that it was taking the unprecedented step of attempting to obtain the licence itself for a rare gene therapy on a non-profit basis, after the pharmaceutical company that planned to bring it to market dropped out. If successful, it will be the first time that an NHS trust has the authorisation to market a drug for this kind of treatment. The move could act as a proof of concept for bringing drugs to UK patients that pharmaceutical companies aren’t willing to risk their profits on.
