ABPI welcomes improvements to rare disease drugs access


The ABPI has welcomed a DH proposal to improve access to medicines for rare diseases – but cautioned against an increase in the use of unlicensed drugs.

The new initiative plans to commission expert assessments of off-label and unlicensed medication to inform doctors and patients before prescribing them – focusing in particular on hard-to-treat conditions such as rarer cancers and autoimmune diseases.

Stephen Whitehead, Chief Executive of the ABPI, said that the ABPI recognises the lack of licensed medicines for rare diseases, and that there can be important clinical reasons why people should be prescribed an unlicensed or off-label drug.

But he said that it is always “preferable for a medicine to be licensed for the purposes for which it is used and has been subject to the rigorous scrutiny of the regulatory authorities”.

“The regulatory and licensing system in the UK exists to protect the public, so the circumstances when an unlicensed or off-label medicine is prescribed should continue to be strictly limited to occasions where there is no suitable licensed alternative available and use is in the best interests of the patient concerned.

“We look forward to engaging with all government and healthcare stakeholders in the months to come to help shape the implementation of these proposals, and ultimately, improve patient access to new, innovative medicines”, he said.

On average, 1,000 specific requests for off-label drugs are made to NHS commissioners every year.