15 Aug, 13 | by BMJ
Post by Julian Savulescu
Cross-posted from the Practical Ethics blog, and relating to this paper in the JME.
Andrew Culliford, whose story is featured in the Daily Mail, is one of the estimated 7 in 100,000 people living with Motor Neuron disease, a progressive degenerative disease which attacks muscles, leaving those affected eventually unable even to breathe unassisted. For Andrew, a young father who has a severe form of the disease, it means a two to five year life expectancy.
The US introduced a mandatory pre-approval process for pharmaceutical drugs after over 100 people were killed by an untested drug formulation. Today, each drug must go through a series of strictly controlled trials, including Phase 1 tests on healthy volunteers, followed by Phases 2 and 3 which test the drug and dosages on smaller and then larger patient groups. The process is estimated to cost $500 million per drug and to take 8 – 12 years.
The process is designed to ensure the efficacy of drugs has been scientifically demonstrated to a very degree of confidence, and to ensure that patient safety is sufficiently protected. In many ways it has been a triumph of science and regulation.
But it has been a failure for one small group of patients: those with rare, imminently lethal diseases, for whom there are no existing good treatments. more…