The US Food and Drug Administration (FDA) has proposed new rules that would make experimental drugs for serious or life threatening diseases more widely available to patients who lack other therapeutic options. The draft regulations were issued on 11 December and public comments will be accepted for 90 days.
Currently, access to experimental drugs has varied, depending on the disease and the section of the FDA with oversight of that pharmaceutical product. The proposed rules would clarify and standardise that process, explained Richard Klein of the FDA's office of special health projects.
The proposal creates a new “small group” category. This would fall between the existing system that is commonly used to give early access to new drugs to large numbers of people (such as with HIV drugs) and single person compassionate use, which is how many cancer patients gain access to experimental drugs.
The proposed regulations also establish standards for data that must be gathered in each category and define better the risk-benefit for patients and providers, Mr Klein said.
The FDA has been caught between various public advocates who focus on either access or safety. The Abigail Alliance has pushed for virtually unfettered access to experimental drugs for people who are terminally ill. In 2006 it won a landmark federal court decision affirming a constitutional right to such access. That decision is under review.
“Taking current policy and putting it into regulation does nothing to help many cancer patients and others with serious life threatening illnesses that could be helped,” said the alliance's founder, Frank Burroughs.
But Sidney Wolfe of the Public Citizen Health Research Group emphasises safety. He saw the changes as potentially “unleashing some floodgates that could do more harm than good for a number of people.”
The new proposal would also allow companies to charge certain fees for providing these drugs. The FDA has long allowed companies to charge patients for some costs associated with access to an unapproved drug, but not to make a profit. Very few large drug companies have charged for such access.
But as drug development has changed, with an increasing share of innovation coming from small start-up biotech companies with limited capital and infrastructure to support such programmes, there has been a growing sense that this has restricted access to experimental drugs through compassionate use programmes.
The proposed regulations say that charges are permissible in a clinical trial only to facilitate development of drugs that promise significant advantages over existing treatments and might not otherwise be developed because of their cost. It is meant to encourage access to drugs that otherwise might not be made available unless the manufacturer could recover the costs.
The proposals are available at www.fda.gov/cder/regulatory/applications/IND_PR.htm.
