323

Cutting-edge therapies exist, but the market cannot deliver them cheaply. Britain must build NHS capacity so that cures become collective goods, not expensive productsJust a small fraction of our 20,000 genes can cause disease when disrupted – yet that sliver accounts for thousands of rare disorders. The difficulty is: what can a doctor do to treat them? In a common condition such as type 2 diabetes, the underlying biology is similar for millions of patients. The doctor can prescribe metformin. But with a genetic disorder, the mutation might only affect a small number of people worldwide. In many cases, doctors won’t even know which mutation is responsible, let alone how to fix it.Novel gene-editing breakthroughs are making headlines. But therapies are expensive and complex to develop. The cost of bringing any new drug to patients is now around $2bn, in part because, as Brian David Smith notes in New Drugs, Fair Prices, the “success rate, from discovery to market, is tiny” and there are approved treatments for “less than 10% of the 8,000 diseases that affect humans”. Commercial incentives, he argues, skew innovation towards lucrative cancer drugs and long-term treatments for large populations. Complex gene therapies for very rare conditions are seen as too costly to develop and too small to profit from. Continue reading...
Be respectful and constructive. Comments are moderated.

No comments yet.