The Government has published its response to the Early Access to Medicines Scheme consultation outlining how the Scheme will work when it is launched in April. It is hoped that not only will it make the UK a more attractive place for research but will also allow faster access to promising new drugs for patients with life-threatening and severely debilitating conditions whose symptoms cannot currently be treated.
The government's 2011 Strategy for UK Life Sciences set out to make the UK a more attractive place for research. As part of the Strategy the Government agreed to address barriers to drug development and access by exploring an early access scheme.
In the autumn of 2012 the Medicines and Healthcare Regulatory Authority (MHRA) consulted on a scheme which would allow early access to drugs for serious conditions which have proved promising in trials but before they have completed all the stages needed to be licensed. We responded to the consultation welcoming the introduction of an early access scheme as an important first step towards allowing patients access to drugs more quickly. However, we also explained why an early access scheme on its own is not enough and outlined why the Government needs to do more to explore additional adaptive approaches to licensing new drugs.
What has happened now?
The Government has published its response to the consultation and announced that the Early Access to Medicines Scheme will launch in April 2014. The Scheme, which goes further than the original proposals put forward by MHRA, looks very promising.
How will the scheme work?
Stage 1: ‘Promising Innovative Medicine’ Designation
Similar to the ‘Breakthrough Therapy’ designation used by the US Food and Drug Administration (FDA), a Promising Innovative Medicines (PIM) designation will be granted by MHRA for treatments in areas of unmet clinical need that show early promising signs in Phase I and II clinical trials. It may be for conditions for which there are no treatments available or where there is an identifiable subset of patients who do not respond to the current treatment. Importantly, the designation can be given not only to new biological or chemical entities but also for re-purposed or recently approved drugs licensed for other conditions.
Products with a PIM designation will be subject to early review and early clinical data will be available to the MHRA and NICE for them to assess the benefits and risks of the treatment.
Stage 2: An ‘Early Access to Medicines’ Scientific Opinion
Using the data collected during Stage 1, the MHRA will issue treatments a benefit:risk scientific opinion. A positive opinion will allow prescribers to offer the medicine to those patients with no therapeutic alternatives. At this stage the medicine will be made available to patients free of charge by the sponsoring company.
If, as the Government hopes this new benefit:risk scientific opinion is issued by MHRA at the end of phase II clinical studies rather than as currently after phase III clinical studies this could speed up the development of a drug by several months or even years.
Stage 3: Licensing and Rapid Commissioning
A co-ordinated NICE technology appraisal and NHS England Commissioning process will be introduced which will use data collected in the earlier stages to fast-track the assessment of medicines developed through the Early Access Scheme. The new process will also allow manufacturers to make use of Pharmaceutical Price Regulation Scheme provisions for Flexible Pricing.
The medicines will typically be commissioned by NHS England through its specialised commissioning processes meaning they will be available nationally. Currently there are no plans for the Scheme to receive Government funding. It’s essential therefore that the Government invests in the specialised commissioning budget, which is under increasing pressure, to ensure the NHS can afford to provide those drugs which make it to Stage 3 to patients.
What does this mean for patients and medical research charities?
This is good news for people suffering from some of the most life-threatening or debilitating conditions without effective treatments. It might mean new treatments reach them sooner. But importantly it also preserves the important role of the MHRA as the expert in assessing safety and risks of new products for parients.
Traditional clinical trials are the main way severely ill patients get access to the newest treatments, but many are excluded from trials because they have complicating factors, such as multiple pre-existing conditions. For those people this scheme could make a real difference. Some have said that the scheme will also cut the time lag between early clinical research and widespread use to as little as five years, compared with the current industry average of 10 to 15 years. Time will tell whether this is overly optimistic or not and whether the Scheme will significantly increase the number of drugs available for the most in need patients.
Commenting on the Early Access to Medicines Scheme, Sharmila Nebhrajani, Chief Executive of AMRC, said:
We welcome the Early Access to Medicines Scheme as an important initiative that will allow patients to access potentially life-saving drugs more quickly. However the Scheme is just one part of a much larger picture. There are problems at every stage of the pathway from bench to bedside and adoption of innovation by the NHS is low and slow. The Government must do everything it can to help researchers develop new and better treatments and ensure they reach patients who desperately need them.
This scheme is part of a wider movement to speed up innovation and adoption. Sharmila recently blogged about other initiatives underway including the NICE Implementation Collaborative, which examines what stops the adoption of new treatments by the NHS where NICE guidance has found that a particular treatment is both clinical and cost effective and the Centre for Sustainable Medical Innovation’s adaptive licensing pilots. In a report published last year, the MHRA co-ordinated Expert Group on Innovation in the Regulation of Healthcare recommended that existing flexibility in the current regulatory system should be better exploited.
For all of these to succeed for patients there needs to be a concerted and joined up approach across government and the NHS, one that involves patient groups, medical research charities, and industry. For example, we need streamlined and co-ordinated regulation to help researchers conduct early clinical research, we need to be anticipating the next medical innovation and the challenges it may bring, and we need world-leading IT systems to track the effectiveness of treatments made available through Early Access or other schemes.
We will be raising these and other issues in our response to the Government consultation on the Medical Innovation Bill (also known as the Saatchi Bill).
On 28th April BIA will be holding an explainer event about the Scheme- if you would like to come along to find out more about how it will all work please contact Martin Turner.