Valuing treatments – the regenerative medicine challenge

Speaking at a conference in Osaka, Japan, this week, Nick Crabb, NICE’s programme director for scientific affairs, described recent developments in NICE’s thinking on how we can evaluate new treatments such as regenerative medicine products.

Nick Crabb

Once the realm of science fiction, the ability to regenerate human cells, tissues or organs to restore or establish bodily functions is a rapidly expanding area of science and medicine.

Regenerative medicine’s star players, stem cells, get a lot of media interest, but are just one part of an exciting field showing it is possible to help the body by replacing worn or damaged parts – from new windpipes to artificial blood.

In 2013 a House of Lords Science and Technology Committee report described the huge potential of regenerative medicine in many chronic diseases such as diabetes and heart disease – conditions that take an ever-increasing share of NHS funds. Investing in such treatments could be a major benefit to the UK public purse as well as provide health benefits. 

But the committee’s report cautioned that problems in our regulatory arrangements and a lack of co-ordinated leadership on the issue were holding back the chance to deliver improvements to the quality of people’s lives.

The UK has considerable strengths in regenerative medicine – from the world-class research that takes place in our universities and hospitals to the access the NHS provides to hundreds of thousands of patients in a unified healthcare system.

The path from scientific research to treatments in the clinic is often long and tortuous; from clinical trials to regulatory approval and licensing, to overcoming the barriers to their use in the clinic.

The NHS, like any healthcare system, is not going to provide these treatments just because they are new. They need to know that regenerative medicine products – indeed any new technology – provides additional value for the extra NHS resources they eat up, and more importantly that other, more effective healthcare, is not squeezed out as a result.

For over 14 years NICE has been responsible for finding the balance between the need for innovative new treatments and the finite resources of the NHS. Measuring the ‘promise’ of a new technology – and providing a clear message to the NHS about that promise – is what we do.

Balancing the need to act as a gatekeeper for the NHS’s resources with the enabling role we play in encouraging adoption of new products keeps our independent appraisal committees busy.

Rising to the challenge

Regenerative medicine presents a challenge: their mode of action and method of application is quite different from simply taking a tablet. They may provide a cure rather than simply alleviate symptoms but as a result come with very high up-front costs.

These developments in regenerative medicine mirror in many ways what is going on in the wider pharmaceutical sector. For some years the mantra that ‘the age of the blockbuster drug has gone’ has led companies to look to new ways of discovering and developing new drugs. The science of genomics and personalised medicine has shaken up the sector, and schemes such as the NEWDIGS initiative, of which NICE is a member, use systems engineering and other techniques to speed up the development chain.

As drug discovery changes, the regulatory system is changing too. The Early Access to Medicines Scheme, where drugs are made available to patients before they have gained a full licence, is one such example. A similar scheme is adaptive licensing – where drugs are initially approved for a small, defined group of patients, with their use then expanding to larger populations.

The benefits of the schemes are clear: an early sign from regulators about the potential of a new medicine; better interaction with bodies such as NICE and NHS England who are part of these projects; and gaining additional data and experience of using these medicines in the clinic. All result in a smoother path to market once the licence is gained.

There are other challenges in appraising regenerative medicine treatments. To understand long-term outcomes, we often have to extrapolate data from short-term trials, which is risky, particularly for treatments that offer a cure. There may also be unknown long-term side effects. The speeding up of the regulatory process means NICE is being asked to evaluate treatments much earlier on in their development. This means dealing with uncertainty around the clinical and cost-effectiveness of these technologies when they come to market.

NICE's role

In our response to recent proposals to change our methods to incorporate value-based pricing of technologies, we said that there needs to be agreement on how the risk of this uncertainty can be shared more productively between companies and the NHS. Is there a way to recommend treatments based on manufacturers’ proposals to mitigate these risks? Can we help to develop more imaginative patient access schemes which allow patients to be prescribed drugs in a cost effective way, with the aim of progressively reflecting the value of new treatments as our knowledge of what they can offer to patients increases? These are all areas that we are actively exploring at NICE.

There are many other ways in which we can help the NHS and manufacturers negotiate the complex world of evaluation and market access. In 2009 we created our Medical Technologies Evaluation Programme to identify and select promising technologies. It specifically allows technologies which may have a sparse evidence base, both published and unpublished, to be quickly assessed to reflect the rapid pace of development.

We are also working with NHS England on how we use ‘real-world’ clinical evidence, rather than only clinical trial data, on a scheme called Commissioning through Evaluation, a new approach to evaluating promising specialised treatments for which there is insufficient evidence to support routine commissioning.

Talking to both companies and industry groups is important. NICE Scientific Advice also runs seminars that enable manufacturers to understand the evaluation process so they can build this into their plans from an early stage.

NICE’s methods for evaluating new technologies have always evolved – and it is clear they need to continue to do so to gain the full benefits these new technologies can bring.

Stem cell