Out-Law News 3 min. read

New fast track procedures for life saving drugs

Two new schemes designed to speed up the process of getting new life saving medicines to patients with critical or life-threatening illnesses have been announced. 

A life sciences legal expert has said that the schemes will build on existing regulatory frameworks and patient access programmes already in place in Europe and will be welcomed by the industry and patients.

"What patients want is sometimes to try medicines that may not be clinically proven to be effective but are clinically safe." health secretary Jeremy Hunt said when launching the UK's Early Access to Medicines Scheme (EAMS), which is set to launch next month. "We are streamlining the process so these medicines can be used much earlier - particularly if they have early promise - and that is something which will bring hope to a lot of patients."

Paul Ranson of Pinsent Masons, the law firm behind Out-Law.com, welcomed both EAMS and the adaptive licensing pilot announced by the European Medicines Agency (EMA) earlier this week.

"The EAMS and the EMA's adaptive licensing pilot will build on existing regulatory and patient access frameworks already in place in Europe and the UK," Ranson said. "Often, patients with critical or life-threatening illnesses don't qualify for clinical trials. Some are just too sick. Or perhaps their previous medical treatment disqualifies them from the study. Whatever the reason, for many of these patients the wait for drug approval is too long." 

Under the EAMS, drug companies will have the chance to follow a three-stage fast-track process for bringing their products to the market. They will be able to obtain a 'Promising Innovative Medicine' (PIM) designation from the MHRA to signify, during the development stage of a new drug, the potential of that product for treating patients with life threatening or seriously debilitating conditions for which there is either no cure or where existing treatments are unsatisfactory.

The companies could also obtain a scientific opinion which would allow doctors to prescribe the drug whilst still unlicensed, provided the potential benefits outweigh the risks. In addition, the companies would also be able to use a new licensing and rapid commissioning process being created by NICE and NHS England.

Drug companies would not be able to charge for the use of their drugs under the scheme until they obtain a license for the drug, although they would be able to recover costs.

"The scheme will provide a mechanism by which UK patients will be able to access the next generation of breakthrough medicines earlier than would have been the case had the company producing it waited for the granting of a marketing authorisation," the UK government said. "Any potential economic benefit of the scheme is likely to be in the area of creating a new, positive signal to companies developing medicines that their products are likely to gain traction in the NHS market. This may in turn help those companies in gaining investment."

"This will, alongside the NHS’s comprehensive R&D infrastructure, including the data resource provided by the Clinical Practice Research Datalink, put the UK in a leading position globally for health life sciences," said the government.

"Drug development companies will be interested in what the validation a PIM designation could bring to their development programme along with the possibility of having the benefit of assistance from both the MHRA and NICE in designing the remainder of the regulatory path," Ranson added.  

In another new development, drugs companies have been invited to submit applications to participate in a new adaptive licensing pilot project (8-page / 171KB PDF) being run by the EMA.

"The UK’s EAMS and the PIM designation may facilitate identification of good candidates for the EMA's adaptive licensing pilot," Helen Cline of Pinsent Masons said.

Adaptive licensing is a term used to describe a new flexible pathway for the approval of innovative medicines to treat life threatening conditions, the aim of which is to balance timely access for patients to possible life saving treatments with the need for evidence of benefit and safety. Amidst an environment of continual testing and development, this new staggered approach allows an initially restricted number of patients to benefit from new treatments before being gradually expanded for use by more individuals as more information on the drug is generated.

"With the adaptive licensing pilot project we intend to explore with real medicines in development a progressive licensing approach that would allow timely access for patients to new medicines that address serious conditions with unmet medical needs," Hans-Georg Eichler, the EMA's senior medical officer, said. "The approach seeks to maximize the positive impact of new medicines on public health by balancing timely access for patients with the need to provide adequate evolving information on their benefits and risks."

Allistair Booth of Pinsent Masons also welcomed the UK government's consultation on the Medical Innovation Bill, commonly referred to as the Saatchi Bill in recognition of the Bill's sponsorship by Lord Maurice Saatchi.

Under the proposed new Bill, doctors would be able to "depart from the existing range of accepted medical treatments for a condition" without having to fear that their actions could be deemed negligent, as long as their decision to do so is a responsible one. The circumstances that would allow doctors to take this action are where they believe the treatment they propose to proscribe is either not backed by "a responsible body of medical opinion" or it is unclear whether there has been or would be such support for the proposed treatment.

The Bill explains a further list of considerations doctors must make before prescribing new innovative treatments.

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