Drug companies ‘exploiting law for profit’

In an open letter to the Prime Minister and the Health Secretary Andrew Lansley, and published online in the British Medical Journal, the neurologists and a patient group, say they are deeply concerned about the unintended consequences of the European Union’s regulations on orphan drugs.

They say that pharmaceutical companies are buying up existing, unlicensed drugs, obtaining a licence and then massively hiking the price. They point to one drug, called 3,4-DAP, which has been used for 20 years to treat Lambert Eaton Myasthenic Syndrome (LEMS) and congenital myasthenic syndromes, both of which cause serious and disabling muscle weakness. It cost between £800 and £1,250 per patient per year.

Earlier this year a pharmaceutical company, BioMarin, bought a slightly different version of the drug, one which had a stabilising compound, and put it on the market under the new name Firdapse. All its safety data was gained from the unlicensed drug, the letter says. The cost of prescribing the licensed BioMarin drug is between £40,000 and £70,000.

But the clinicians said that this is “simply a modified version that meets regulatory standards.” They said the drug company had been “allowed to set the price at an exorbitant level with no clinically relevant advantage”. In other words, there is little difference between the two drugs except for the price.

Funding

Some Primary Care Trusts (PCTs) are refusing to fund the new drug, called Firdapse. But in the meantime, the Medicines and Healthcare Regulatory Authority (MHRA) have told the manufacturers of the unlicensed 3,4-DAP to stop producing it. They have also said that now it is licensed, clinicians are obliged to use it.

BioMarin, itself, has written to number of suppliers of 3,4-DAP to say that now they have marketing authorisation for Firdapse, it would now be a breach of regulations for them to supply hospitals with 3,4-DAP.

BioMarin would not be interviewed, but in a statement said there were no safeguards in place with the unlicensed drug. Now, they said, “patients can be confident that they are taking a medicine which is required to conform to specified quality controls and which undergoes continuous safety monitoring.”

They also said they have completed a trial on the effect of taking the drug with food and were planning three more trials.

Channel 4 News spoke to Jannett Grandison, 49, who has LEMS. She said: “I went to bed one night and woke up the next morning in a wheelchair.”

Following her diagnosis she was prescribed 3,4-DAP by her neurologist David Nicholl, at City Hospital, Birmingham. But that stopped in May when the supply began to dry up.

Mrs Grandison said the drug had been working, that she had felt more strength coming back into her limbs.

Dr Nicholl said a block had been put on prescribing 3,4-DAP from September although, even earlier, by July, the drug did not seem to be available.

However, The West Midlands Specialised Commissioning Group, which advises primary care trusts on what drugs to pay for, has taken legal advice and now believes they are within their right to agree to the purchase of 3,4-DAP.

Licensed drug choice

David Lock, a barrister specialising in healthcare issues, said that if there is a choice between a licensed and unlicensed drug, doctors are strongly encouraged by the General Medical Council code of practice to prescribe the licensed drug because, in general, this will represent a lower level of patient risk and a greater chance of effective treatment.

But, Mr Lock said the code is silent as how the doctor should prescribe where the licensed product is not available on the NHS.

In this case, some PCTS are saying they will not agree to the purchase of Firdapse because it is too expensive. Therefore, it is not available. Dr Daphne Austin, who sought the legal advice, said she was disgusted by the price increase. She said that if the NHS paid the £40,000 per patient per year, other patients would suffer. Off the back of Mr Lock’s advice, she said she now believes the unlicensed drug can legally be prescribed.

It is estimated that if all PCTS in England agreed to the purchase of Firdapse, it would cost the NHS more than £10 million – the equivalent of 360 patients on dialysis.

In their letter, the consultants say that in “the present economic situation it seems vital to ensure systems are set in place to prevent excessive commercial profits being made that the expense of patients and public spending.”

In short, they want the loophole closed.