Many tough questions. No easy answers.
From the latest edition of the British Medical Journal ...
Speeding up access to new drugs
The prime minister promised at the end of last year to give some patients quicker access to new drugs. Was this just an empty aspiration?
No. A lot of thought has been given to a faster access scheme since David Cooksey recommended it in his review of UK health research funding in December 2006. A working group from industry and government produced a plan by November 2009. It sat on a shelf until resurrected two years later.
How would it work?
Medicines that have completed phase III trials (or in exceptional circumstances phase II) and that will treat or prevent life threatening, chronic, or seriously debilitating conditions that lack adequate existing treatments would qualify. Manufacturers would have to apply, and a decision would be promised in 75 days.
How would that be any quicker than licensing, if phase III trials have already been completed?
It usually takes a year or more to get licensing approval after a successful phase III trial. The new process is expected to get these medicines to patients a year earlier than otherwise would be the case.
But doesn't it mean that proper risk assessment will be skimped?
There's a danger of that. The burden of risk will be shifted towards the doctor and the patient and away from the manufacturer. Good information will be vital; patients will need to be fully informed and give active consent. Legally, the position will be the same as that for any unlicensed medicine, and the working group believes-but cannot guarantee-that primary care trusts or clinical commissioning groups will not be liable should anything go wrong. And it says that if the decision by a doctor to treat a patient was reasonable in all the circumstances and all relevant information was provided, a successful claim for negligence is unlikely.
If unlicensed medicines can be marketed and sold, what's the point of licensing?
The medicines under this scheme would be exceptional and few in number, perhaps only one or two a year. The NHS already uses unlicensed or off-label medicines in some cases, on the authority of the prescribing doctor (for example, bevacizumab for age related macular degeneration). Patients consulted by the working group were confident that they were competent to make a proper assessment of the risks. Doctors were not so sure.
What happens when the medicine is licensed?
The approval will last a year and can be renewed if necessary. When the medicine gets a licence it will become part of the normal process and the National Institute for Health and Clinical Excellence (NICE) will examine its cost effectiveness. Arrangements for continued funding of early access patients will need to be agreed for each medicine in advance.
So these fast access medicines will bypass NICE?
Yes. NICE deals with licensed medicines; these are unlicensed, so NICE does not have a role.
So how can we tell if manufacturers are overcharging?
We can't. They will set the price. It will be up to commissioning organisations, without NICE input, to decide if it's a price they want to pay.
Where's the money coming from?
An important question, the answer to which could yet put a spanner in the works. Since these medicines will not be approved by NICE, there will be no obligation for primary care trusts or clinical commissioning groups to pay for them. There will be no additional money available, unless the Department of Health or the drug companies provide it. Commissioning bodies will have to fund the drugs out of their normal allocations and in hard times may be reluctant to do so. This could lead to "postcode prescribing," as the working group conceded. But it hoped that if the process were applied only to medicines providing "significant clinical benefit in areas of current unmet need" the variation could be minimised.
Has anybody else attempted a similar scheme?
Yes, the French Temporary Authorisation for Use (ATU) scheme is quite similar and has operated for 15 years. To qualify for this, the medicine must have no satisfactory alternative and patients cannot access the scheme if they could become part of a clinical trial of the same medicine. The scheme is restricted to hospital specialists, and companies must agree to submit an application for a full licence, usually within a year, of the temporary authorisation being granted.
Experience shows that companies usually set high prices, but if the price fixed after market authorisation is lower they can be asked to refund the difference.
What about surveillance of side effects?
Applications for faster access would have to include plans for drug surveillance. "Collecting safety data is essential to protecting patients receiving the medicine," the working group concluded and is also an important way of developing a better understanding of the medicine. But the demands should not be so burdensome as to discourage companies from applying; nor should data gathering be seen as a clinical trial.
The Medicines and Healthcare Products Regulatory Agency plans to launch a consultation on the scheme at the end of March. It will allow 12 weeks for responses.