The government says it has decided not to progress its proposed Earlier Access to Medicines Scheme for the time being, "given the many other ongoing initiatives in the NHS."
This decision does not affect other existing mechanisms for earlier access to unlicensed medicines, such as through clinical trials and named-patient use, the Medicines and Healthcare products Regulatory Agency (MHRA) said last Friday (May 6).
The idea for introducing a UK scheme to make certain new and promising medicines available to patients before they are formally licensed was first put forward in 2009 by the Ministerial Industry Strategy Group (MISG), an initiative that brings together relevant UK government ministers and pharmaceutical industry chief executives. The MISG then asked the MHRA and the Association of the British Pharmaceutical Industry (ABPI) to explore the feasibility and desirability of introducing such a scheme.
A working group co-chaired by the MHRA and ABPI - which also sought views from the public and healthcare professionals - developed a framework for an earlier access scheme to operate within the existing regulatory regime, and this was reviewed and approved by the MISG in late November 2009.
Under the proposals, new medicines aimed at treating, diagnosing or preventing life-threatening, chronic or seriously debilitating conditions without adequate treatment options would be eligible for the scheme if they had completed Phase III trials. In exceptional circumstances, an earlier authorisation might be possible based on Phase II trial data.
A maximum 75-day review period for such products was proposed, and the approval would last for one year, renewable if necessary. The MHRA would charge a fee to cover costs of the review and publish a list of medicines approved on its website, together with the assessment report. The Agency would also publish information about any medicines not approved.
The earlier-access scheme would not involve any appraisal by the National Institute for Health and Clinical Excellence (NICE). Manufacturers would have freedom of pricing for medicines supplied under the scheme, but there would be no central mandate for funding as applies to NICE-approved products, or any additional funding for earlier-access medicines.
Primary Care Trusts (PCTs) would therefore be responsible for considering whether the funding of early-access medicines was cost-effective, and for finding such funding from existing budgets. The MISG acknowledged that this could lead to continued variation in uptake and NHS availability around the country, but pointed out that the fact that these medicines had been subject to an approval process and would be made available on the same basis across the country should help reduce such variability.
The MHRA said last week that while it has prepared an outline on how the Earlier Access to Medicines scheme might work in practice, no decision has yet been made on whether it will go ahead. If however the concept of the scheme is approved, it will then be subject to a 12-week public consultation, it added.