Early access scheme unveiled in UK

The UK government has unveiled new scheme which will give patients with life threatening or seriously debilitating conditions access to as-yet unlicensed medicines.

Dr Ian Hudson, Chief Executive of UK medicines regulator the MHRA said: "We are delighted that the Early Access to Medicines Scheme will be launched in April 2014. The scheme is intended to enable patient access to medicines for treatment of life threatening or seriously debilitating conditions where there is an unmet need. This is a major new development in medicines policy in the UK.

"The scheme offers a way by which unlicensed medicines can be available to patients before approval of a licence to benefit public health. It will also enable companies to gain additional knowledge and experience of these medicines in clinical use."

The scheme will see the MHRA giving a scientific opinion on a new medicine or indication that has demonstrated a positive risk/benefit balance. It will begin taking applications from companies from early April 2014.

The UK regulator points out that the scheme is voluntary, and the opinion from MHRA does not replace the normal licensing procedures for medicines.

MHRA is responsible for the scientific aspects of the scheme and the scientific opinion will be provided after a two-step evaluation process:

• step 1, the promising innovative medicines (PIM) designation

• step 2, the early access to medicines scientific opinion.

Promising innovative medicines (PIM) designation

The PIM designation will give an indication that a product may be eligible for the Early Access to Medicines Scheme (based on early clinical data). The PIM designation will be issued after an MHRA scientific meeting and could be given several years before the product is licensed.

Companies who want to move to step 2 must hold a PIM designation and provide further relevant data on quality, safety and efficacy.

Early access to medicines scientific opinion

The scientific opinion will describe the benefits and risks of the medicine, based on the information submitted to MHRA by an applicant after sufficient data have been gathered from the patients who will benefit from the medicine. The opinion will support the prescriber and patient to make a decision on whether to use the medicine before its licence is approved.

Paul Catchpole, Director of Value and Access at the UK pharmaceutical industry body the ABPI, said:

"The scheme will benefit patients, the NHS and the UK clinical research community, of which our industry is a part. Most importantly, it means that patients with some life-threatening or seriously debilitating conditions, without adequate treatment options, will be able to get faster access to important innovative medicines as these will be made available before launch, following an assessment by the MHRA.

"The introduction of the Promising Innovative Medicines (PIM) designation should help make the UK more attractive for investment and growth, with similar merits to the FDA Breakthrough Therapy Designation in the US.

"The collection of additional data and evidence over the period the medicine is available before launch is very welcome. This has the potential to assist NICE and other health technology assessment bodies, in being able to recommend more medicines for use with greater certainty after they are launched and could, therefore, result in more timely routine access to licensed medicines for NHS patients.

Catchpole said the fact that the scheme did not come with any additional funds could present issues for some companies.

"This means companies have to bear the risk associated with the upfront investment that will be required to participate in the scheme," said Catchpole. "As part of our consultation response, we have called for a one year on joint review of the scheme so that we can appraise its first year and potentially review funding options."

Pete Butterfield, Chair of the ABPI Smaller Companies Forum, said: "This scheme provides an exciting opportunity for smaller companies. We have already seen a similar approach working well in the US through the FDA's Breakthrough Therapy Designation. Companies use it to secure investment and increase the credibility of the medicines in the scheme, particularly in other countries. This new scheme provides a clear signal that the UK is a leading healthcare provider with an innovative approach to securing quick and safe access to medicines for patients who need them."

However not everybody has welcomed the move. The Faculty of Public Health (FPH) opposed the changes in its response to a consultation, saying that there was "no substantive research that allowing medicines into the market place earlier" would benefit improvements in population health, and said it feared the pharma industry would exploit the reduced requirements for evidence.

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