Science and Technology CommitteeWritten evidence submitted by Dr Mark Edwards FRCA FSB

Introduction

1.1 As a staunch advocate for the UK life sciences, these are my personal views on the work of the Health Research Authority (HRA) and what it could achieve for the UK with appropriate resource.

1.2 Since its inception, the HRA has presented itself as a dynamic champion of change, with a determination to review every aspect of clinical trial regulation, to remove processes that do not work or really make sense, and to develop and implement revised procedures that do.

1.3 I believe the HRA has the potential to evolve as the single, hugely credible, unifying port of entry to do clinical research in the UK. While recognising that it must be able to walk before it can run, its scope of influence is potentially enormous. It also has the right people in place to achieve this vision, most notably Janet Wisely, who, with a common-sense approach, is an inspirational leader of the first order.

The HRA Approvals Process

2.1 Via the National Research Ethics Service (NRES), the HRA has established a process that works well for the ethics approval of clinical trials and I rarely now hear about this being an issue for companies. Similarly, the process for obtaining MHRA approval for clinical trials seems to work well overall.

2.2 So, the remaining challenge for the HRA in the “approvals” space is to achieve a single NHS Research & Development (R&D) approval for multicentre trials. This is a huge challenge, but one that, if successful, could alone make a significant impact on the attractiveness of the UK as a place to do clinical research ie for the UK to be able to offer industry the “holy trinity” of a single approval for ethics, regulatory and NHS R&D matters.

2.3 Personally, having worked with the Office for Strategic Co-ordination of Health Research (OSCHR) and then the National Institute of Health Research (NIHR) to help establish the Translational Research Partnerships (TRPs), which are predicated on the fact that a commercial partner can access all of a TRP’s clinical academic expertise through a single contractual signature, I believe the achievement of a single NHS R&D approval for clinical trials is completely doable. It does however, require a collective mind-set to be established in which people understand the importance of “waking up and smelling the coffee”. In this instance this means the development of a common understanding that collaboration between Trusts will achieve more than each Trust continuing to operate in its own silo. One idea for the HRA might be to bring together selected Trust CEOs (and their R&D office personnel) who have demonstrated a willingness to “do things differently” to develop a framework for a single NHS R&D sign-off.

2.4 This now brings me to the overall “time, cost, quality, reliability” (TCQR) quartet that are the primary industry considerations for placement of certainly later phase clinical trials. The HRA has to date, quite rightly, focussed on improvements to the “time” element. The single NHS R&D approval is a key component of this.

2.5 The costs of doing clinical research in the UK present a mixed bag of opinion from industry. Most agree that paying a reasonable premium for clinical research in the UK is acceptable, as long as time, reliability and quality factors are addressed such that the “end product” justifies the premium price. The key bugbears seem to be 1) instances where the UK is cited to be a “high end” outlier compared to other countries and where no explanation for the exorbitant costs are supplied and 2) a general concern that overhead charges are just too high and cannot be justified. Companies end up feeling ripped off. At the very least, there should be efforts made to standardise overhead costs across the NHS and provide greater transparency as to how they are derived. The HRA could play a pivotal role in achieving this. Indeed, a parallel could be drawn here with the way in which Trusts have Health Resource Groups (HRG) under Payment By Results (PBR) whereby they group procedures into HRGs and cost them accordingly. This leads to a consistent charge across the NHS.

2.6 This leaves quality and reliability factors. I do not sense that lack of quality in UK clinical research is routinely a major issue with companies. Reliability issues are, however, a concern. Some companies have told me that this is indeed their most significant issue with clinical research in the UK ie “they do not deliver what they said they would deliver”. Most late phase clinical research is conducted on a global scale, which, within industry requires a great deal of planning to allocate the right degree of resource (and associated costs and budgetary management) to the various countries where the research has been placed. Under-delivery therefore, not only delays the timelines for delivery of the research results (and therefore potentially the approval of a new medicine), but can also increase overall development costs by forcing companies to revise their clinical operations strategies in “mid-flow”. Such over-promising and under-delivering is therefore a serious issue for any territory which hopes to attract commercial R&D investment. Maybe there is a role for the HRA here, which in the first instance could be to understand its root cause?

2.7 What is certain however is that for the HRA to be successful in improving all TCQR factors, it needs the wider and more open engagement of industry to share its experiences. These need to include helping the HRA and its networks to understand how the UK is benchmarked against other countries in these key parameters. In turn, this requires industry to provide truly comparable data ie data from like-for-like studies are needed. The UK offices of the large global contract research organisations (CROs) may be in one of the best positions to help here and I would recommend discussions between them and the HRA are commenced soonest.

Recommendations for Future Areas of Focus

3.1 Looking further to the future of possibilities for the HRA, I now suggest a few areas for consideration where I sense it could have a greater role to play, or at the very least needs to keep an eye on. They are based on what I perceive is the HRA’s ability to act as an “honest broker” and to adapt to changing circumstances ie to ensure that health research regulation keeps pace with evolving science and safeguards patients, but in a manner that enables the UK to be world-leading;

Patients and adherence with medicines; helping industry engage constructively with patients/patient groups to enable it to play a full part in the clinical research patient engagement, involvement and participation agendas (it should be remembered that industry scientists and physicians probably know the in’s and out’s of their medicines in development better than anyone else). In turn, this “better engagement” would play its part to assist the enormous issue we have with medicines adherence. We are aware that this is a “hot topic” within Dept. Health under the Medicines Optimisation agenda led by Dr Keith Ridge (Chief Pharmaceutical Officer) and Clare Howard (Deputy Chief Pharmaceutical Officer). This requires pan-stakeholder action and the HRA may be well-placed to play its part.

Adaptive licensing; a great chance for the UK to be world-leading should pilot projects be successful. The emphasis that this will have on effectiveness data collection puts the UK at a potential advantage over elsewhere because of the relative maturity of health informatics systems here. What role could the HRA play to ensure an appropriate level of regulation for adaptive licensing projects?

Stratified medicine; the Technology Strategy Board has issued a “roadmap” for stratified medicine which appears, rightly, to be embrace policy, as well as scientific issues. This will build on the UK’s translational research expertise and will have emergent health research regulation considerations.

Open access; helping all stakeholders to understand that, with the right processes in place, it’s “OK” to share your toys, because you’ll get access to a whole load more for the benefit of all.

Conclusion

4.1 I wish the HRA sustained success, influence and growth. I call on the Government to ensure that it receives the right level of resource to achieve its full potential as a key driver of clinical research investment into the UK.

February 2013

Prepared 16th September 2013