Research and evaluation
NICE’s assessment process is stifling pharmaceutical innovation
The way new medicines are assessed should be reformed to provide patients with the best treatments and stimulate research and innovation in the UK.
The UK has long been a world leader when it comes to the life sciences. For more than 40 years, its pharmaceutical industry has been one of the most successful globally. It has consistently provided healthcare professionals and patients with the latest medicines to battle disease. One-eighth of the world’s most frequently prescribed medicines were developed in the UK and exciting, innovative and specialised medicines continue to come through the pipeline.
The UK government recognises the nation’s position as a global leader for innovation in life sciences and the positive effect this has on the health and wealth of society is to be commended. The 2011 ‘Strategy for UK life sciences’ pledged that the UK would help support pioneering researchers and clinicians “to bring innovation to market earlier and more easily, making the UK the location of choice for investment”.
In a few short years since 2011, however, there are concerns among many in the pharmaceutical industry that the UK is losing its hard-earned place at the top of the innovation table. The National Institute for Health and Care Excellence (NICE), which appraises medicines and health technology and makes recommendations on whether something should be funded by the NHS, needs to adapt to the ever evolving pharmaceutical research and development model.
Time to evolve
NICE has an important job to do and has approved many crucial and life-saving medicines through its appraisals process. But by the same token, a number of arguably vital drugs have not been approved, including ovarian, breast and prostate cancer drugs, along with medicines for rare diseases. The Association of the British Pharmaceutical Industry (ABPI) reiterates that NICE’s appraisal system — in its current form — needs urgent reform.
What is apparent is that, when it comes to assessing new medicines, NICE’s current one-size-fits-all approach is no longer fit for purpose. For a start, the regulatory process has evolved and there are now new routes to market and ways of licensing promising new medicines, such as the Medicines and Healthcare Products Regulatory Agency’s (MHRA) early access to medicines scheme, launched in April 2014, and the European Medicines Agency’s (EMA) adaptive pathways scheme, launched in March 2014, which aim to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need. These schemes are to be welcomed, proving that the regulatory process is adapting to a pharmaceutical pipeline that is becoming increasingly specialised, targeted at smaller patient populations and associated at times with greater levels of uncertainty in the data at launch.
Most importantly, these types of schemes are a huge first step towards helping patients get quicker access to new medicines. But this will only happen if NICE, in turn, adapts to the needs of evolving science and is equally responsive to the new medicines that are being developed by the pharmaceutical industry.
Value of treatments
One way that NICE can adapt to the changing R&D environment is to recognise more broadly the value of new treatments. For a start, the changing nature of the innovative medicines pipeline means that the evidence base is changing. Innovative medicines are new by nature and therefore may not have the volume of trial data that is available for other medicines. NICE, therefore, needs to adapt its current methodologies to assess medicines with an immature or incomplete evidence base to permit earlier access to NHS patients. This could be achieved, for example, by implementing fully flexible managed entry or commercial access agreements, including those which involve the collection of further clinical evidence. Specific consideration and substantial weighting needs to be given to innovation so that novel, valued medicines can be approved more regularly for NHS patients.
The ABPI has proposed that, in future, NICE could perform a type of scope-and-triage process when a new medicine needs to be appraised in order to decide the appropriate method for any subsequent assessment. We have long held the view that the assessment process needs to be streamlined and proportional to the potential impact of the medicine on NHS patients and the healthcare system. We have also voiced our view that there is an over-reliance on the quality-adjusted life year (QALY) as the dominant metric – QALYs are only one part of decision making in the NHS and are not applied across the NHS to assess the value of other forms of care and treatment.
The ABPI and its industry partners are keen to see a more flexible approach within the decision-making framework so that other factors are taken into account, for example, the impact a medicine might have on a government health priority area or unmet clinical need, disease characteristics, societal value and uncertainty.
More routes to access
In order for innovation to thrive in the UK and for patients to benefit from the latest cutting-edge medicines, we need to create an end-to-end system that is joined up across discovery, development, regulation, pricing, value assessment and medicine use. In other words, the government needs to ensure the healthcare system — the MHRA, NICE and NHS England — work together so that the latest medicines are available for clinicians to prescribe, as they deem appropriate, for patients. In particular, NICE, in its role as evaluator, and NHS England, in its role as commissioner, need to work together to create more routes to access.
The ABPI looks forward to working with the government on the Accelerated Access Review, which aims to speed up access to innovative drugs, devices and diagnostics for NHS patients, and the unique opportunity it presents to deliver widespread access to innovative medicines.
Reform is needed now
Without these substantial reforms to NICE’s appraisals process, we run the risk of stifling pharmaceutical innovation in the UK. From an industry point of view, this would be highly detrimental for a number of obvious reasons, not least of all the health of patients. The UK’s position at the vanguard of medicine discovery and development is threatened and the UK will become a less attractive place to conduct clinical trials. Improving access to medicines for NHS patients must be a major objective if the UK is going to attract global R&D investment because medicines in clinical development need to be compared against the standard treatment for regulatory purposes. More importantly, the effects would be equally damaging for patients. When a drug is approved and prescribed by healthcare professionals, standards are established on which the pharmaceutical industry can improve and build — leading to the best clinical outcomes for patients.
But if a drug is not approved, then innovation is effectively stymied. Innovations are critical to advance healthcare for patients — consider the examples of HIV and AIDS, and heart disease, where major advances have been made. In cancer, new and improved therapies mean that half of people diagnosed now survive their disease for at least ten years.
Medical innovation has the potential to save lives and improve many more. NICE needs to create a technology appraisal system that is fit for purpose and responsive to the life-changing and innovative new medicines that are currently being developed by the pharmaceutical industry. Above all, we need NICE to create a system that truly rewards innovation and allows the UK to be at the forefront of delivering world-class patient outcomes.
Alison Clough is the acting CEO and executive director commercial UK of the Association of the British Pharmaceutical Industry.
Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2015.20068953
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