The Medicines and Healthcare products Regulatory Agency (MHRA) will this month begin a public consultation on proposals to make certain new drugs available to patients before they are formally licensed, with a view to having such a scheme up and running later this year.

The proposed Earlier Access Scheme would enable patients with serious conditions for which there are no (or very limited) treatment options to access potentially life-saving medicines in development around one year earlier than is currently the case, says the MHRA. However, it cautions that the initiative could still result in “a certain amount of variation” in such treatments’ uptake and availability, as funding will have to be found from existing Primary Care Trust (PCT) budgets.

The Scheme would operate within the existing regulatory regime and not amount to “conditional licensing,” and should be seen in the overall context of other initiatives aimed at improving access to new medicines, especially unlicensed treatments, says the Agency. These currently tend to be “locally-based and ad-hoc in nature,” but local variations in uptake could be reduced by a rapid, UK-wide approval mechanism, as provided by the proposed Scheme, it suggests, although it also acknowledges that, given National Health Service (NHS) funding mechanisms, “this cannot be fully managed away.”

The MHRA and the Association of the British Pharmaceutical Industry (ABPI) were originally asked by the Ministerial Industry Strategy Group (MISG) - an initiative which brings together government ministers and pharmaceutical industry chief executives - to examine the feasibility and desirability of an Earlier Access Scheme, and in December the MISG approved the framework for such a scheme, proposed by an MHRA/ABPI working group.

Under the group’s proposals, the Scheme will be available for medicines that have completed Phase III trials, although in exceptional circumstances an earlier authorisation may be possible, based on Phase II data.

It will be for the medicine’s developer to decide whether to apply for inclusion in the Scheme. The MHRA will review the treatment, charging a fee for its costs and taking a maximum of 75 days overall - consisting of a 30-day initial assessment period with a possible single round of questions to the applicant, 15 days for a response plus a further 30 days to review.

Companies will be free to set the prices of medicines supplied under the Scheme, and approval will last for one year, renewable if necessary. No National Institute for Health and Clinical Excellence (NICE) appraisal will be involved, and there will be no central mandate for funding – as applies to NICE-approved products – nor any additional funding for medicines supplied under the Scheme. A UK-wide exit strategy which is specific to each medicine – for example, funding of patients receiving the drug under the Scheme once the product has been licensed – must be stated upfront, the proposal notes.

The MHRA/ABPI working party says it was keen to test the views of the public and patients on the additional risk, or at least uncertainty, of treatment with an unlicensed medicine alongside its potential benefit. Its consultation with these groups produced a “fairly strong “ message that patients and the public understands the risk trade-offs which are at stake and that, as long as all available information is provided and there are strong consent arrangements, people should not be prevented from choosing such treatments. “It was interesting that the views of doctors in the research were rather more sceptical as to the ability of patients to take such decisions,” the group comments.

- The public consultation is set to run for 12 weeks, says the MHRA, which adds that this, and further work to finalise practical aspects of the scheme, could be taken forward “relatively quickly, with a view to launching a formal scheme during 2010.”