In November, I had decided to challenge the impasse over the availability of an effective, but unlicensed, medication for Lambert Eaton Myasthenic syndrome by writing an FP10 for the cheaper unlicensed DAP on Channel 4 news. So what happened next?
The community pharmacist didn’t quite know what to do with the perhaps one of the most highly publicised prescriptions in the West Midlands, but then life got easier. Biomarin, the manufacturer of the more expensive licensed treatment, amifampridine (Firdapse) announced it was “voluntarily” cutting its price by 10%, although its share price went up. However this was not enough. The UK commissioners network did not recommend funding of Firdapse, to quote their legal advice: “There is no reason in principle why the NHS should be required to prescribe a more expensive licensed drug when a pharmacologically identical drug is unlicensed for the treatment in question.”
The government may have not known what to do, but it was particularly gratifying when my patient turned up to clinic last week, she had managed to get her medication, having been unable to get anything for almost 6 months. I congratulated her on her advocacy efforts which undoubtedly had made a massive difference to many patients across the UK. As one neurological colleague in Scotland commented to me, she was being told that it was “illegal to use an unlicensed drug, so patients were being held in limbo” but the BMJ articles have helped move the debate on massively.
What will happen next? The MHRA or Biomarin could decide to challenge legally the widespread prescription of unlicensed 3,4 DAP as a “special” for LEMS by UK neurologists. This would be an interesting legal exercise, but potentially very embarrassing for both the MHRA or Biomarin. Alternatively a patient could challenge their PCT legally for not being able to get the expensive licensed drug.
What have I learnt personally? The only colleague I have found since our BMJ letter who would not have been willing to sign it, were constricted by confidentiality agreements with Biomarin which would have put my colleague in a potentially difficult position. I have been involved with many clinical trials and normally do not give confidentiality agreements a nanoseconds thought, but this has really made me rethink signing such statements without having my employers legal department say-so first for a “whistleblowing” clause.
Patient groups and charities need to very careful in their relationship with BigPharma, in this instance, both the Myasthenia Gravis Association (MGA) and Muscular Dystrophy Association (MDA)were exemplary and acted for their patient members. The former was a co-signatory on the BMJ letter, and the latter lobbied for an Early Day Motion in Parliament over the costing of Orphan Drugs.
The NHS needs to completely rethink its drug pricing negotiations. In short, the NHS should think more like Tesco. Tesco gets criticised for its hard bargaining with suppliers, yet the NHS is a much bigger monopoly than the supermarket giant. It just seems remarkable to me, that government, in a time of austerity, takes such a relaxed view over drug pricing. The orphan drug industry is, I estimate, worth between £100-500 million per year in the UK. So my message to Jannett (and anyone else) is- we (with the help of the BMJ) won the battle, but there is a much a bigger fight over drug pricing that has not even begun.
David Nicholl is a consultant neurologist at City Hospital, Birmingham