14 Mar 2014 Posted in Opinion By Research, Medical and Innovation
This was followed by a key work stream led by the Ministerial Industry Strategy Group (MISG)², and co-chaired by the Medicines and Healthcare Products Regulatory Agency (MHRA)³ and the ABPI, which developed the concept of the scheme.
Given how closely involved the ABPI has been in the development of this scheme, we are pleased to be near to the launch of what we believe is an important new mechanism that will support and benefit patients, the NHS and the UK clinical research community, of which our industry is a part.
Bringing new medicines to patients is a lengthy and risky process. It typically takes over 12 years to research and develop - via a three phase clinical trial process - just one new medicine from thousands of candidates to ensure that it meets the required quality, efficacy and safety standards for a regulatory agency to grant it a license for use in patients.
The EAMS will make it possible for some medicines being developed for life threatening or seriously debilitating conditions without adequate treatment options, to be available to patients earlier without compromising on safety. In some cases, patients could get access to the medicines up to a year before they would be expected to have obtained a license. It is also hoped that the NHS will be able to more confidently prescribe early access medicines because medicines made available under the scheme will be subject to a prior ‘benefit-risk scientific opinion’ by the MHRA. This prior regulatory assessment of the safety and efficacy of the medicine should give confidence to prescribers to make these medicines available to relevant patients.
As with any new scheme, the proof will be in the delivery, and the ABPI will be a close observer and contributor as the scheme develops. Of course, patient safety is always paramount, and patients taking early access medicines will be closely monitored which may provide us with additional important insights. For example, data collected while a medicine is in the scheme has the potential to improve the efficiency of the health technology appraisal process currently in place to ensure medicines are reimbursed within the NHS and achieve cost-effectiveness. In the long run, this scheme could give us important insights that will inform the medicines development process for all medicines.
The scheme will need close review in the first year and a robust end of year appraisal, so that we can ensure it is functioning optimally and we can learn the lessons needed for ongoing improvement. Part of this review should also take into account funding options as the scheme is currently unfunded, meaning companies have to currently take a significant risk in terms of upfront investment to participate in the scheme. Today’s commitment to the launch of the scheme is an important and significant step but we need to ensure that we can build on this so that patients can truly benefit.
Head of Regulatory Affairs