Last week the UK regulator announced plans for a new scheme to allow fast access to new drugs for patients with serious diseases.
The scheme is intended to ‘give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation’. This could include patients with severe forms of rare genetic disease, as well as advanced forms of cancer.
Health Secretary Jeremy Hunt said: "What patients want is sometimes to try medicines that may not be clinically proven to be effective but are clinically safe”. The scheme will allow carefully controlled access to unlicensed drugs, which would need to have demonstrable safety and efficacy in order to gain a ‘promising innovative medicines’ (PIM) designation. This would allow access to the drug in certain circumstances whilst normal Phase III clinical trials go ahead and the full licensing process continues.
The Department of Health officials said that the scheme will benefit pharmaceutical companies by allowing them to learn from NHS application ahead of licensing approval. Costs would have to be borne by the drug developer, a clever move to save precious health service resources. The industry has broadly welcomed the scheme but remain concerned over the lack of funding (i.e. reimbursement) for PIM drugs, believing that it may result in low uptake of the scheme by ‘undermining its attractiveness to UK SMEs and to global corporations choosing the UK as a location for their clinical trials’.
Patient groups have expressed support for the scheme, but the Faculty of Public Health (FPH) has voiced concerns over the vulnerability of seriously ill patients to receiving unlicensed medicines that may not be effective and could even harm their health.