Accelerated Access Review

Ben Howlett Excerpts

Tuesday 13th December 2016 Westminster Hall

[Ben Howlett (Bath) (Con)]

I am pleased to speak in today’s debate and join others in congratulating the hon. Member for Dudley North (Ian Austin) on securing it. I join him in congratulating the Cystic Fibrosis Trust on its work, as well as other cystic fibrosis organisations and charities across the rest of the UK on theirs. As chairman of the all-party parliamentary group on rare, genetic and undiagnosed conditions, I take a particular interest in this subject and in particular how the approach taken by NICE when deciding on funding for drugs is unsuitable for drugs aimed at rare or genetic conditions.

As other Members have said, Orkambi is a particularly interesting development and is part of a new wave of gene-specific precision medicine. It tackles the underlying causes of cystic fibrosis—in this case, the defective CFTR gene—rather than simply treating the symptoms. We should be embracing this new technology and creating appropriate ways for these drugs to be approved and funded, so as not to discriminate against those with a rare or genetic disease. There may be fewer people who suffer from a rare or genetic condition, but I urge the Minister to do all he can to improve their chances. I know that he is a passionate advocate for that particular case.

The NICE process of recommendation understandably relies on data to commission the use of drugs on the NHS. However, the process is currently very rigid, which works against new, often life-changing drugs that only have trial data as evidence. That is exactly what happened with Orkambi, which only had data from a 24-week clinical trial when making its application—similar to a range of other drugs available on the market at the moment—yet evidence shows that it brings significant clinical benefits, as well as wider benefits to society, for people with cystic fibrosis and their carers.

It is not just the lack of available data that discriminates against drugs aimed at genetic and rare conditions. The NICE single technology appraisal process does not adequately reflect the potential benefits of the medicine in protecting future health deterioration or the wider holistic and societal benefits. Its thresholds for cost-effectiveness also work against those with a rare disease. Fundamentally, the diseases are rare by nature and therefore there are only a small number of eligible patients. That should not be a barrier, and we all agree that we need a system that can help those patients.

In short, the accelerated access review, which was brought about after the hard work of my hon. Friend the Member for Mid Norfolk (George Freeman), potentially holds the answers to the problems that currently beset the NICE system. It recognises that the innovative nature of new medicines means that they are unlikely to be approved through the current methods and proposes new guidelines. The new approach will help to ensure that the UK sets itself up as the best possible place to develop new drugs and, I hope, for Orkambi. The Minister might not be able to give me the answer now, but I would like him to write to me on the predicted result of the reduction of the drug spend through NHS England—as we recently heard in a series of evidence sessions held by the APPG for rare diseases—from 7% to about 3.5%, and on how the accelerated access review budget will be increased to compensate.

The Government are set to respond to the recommendations shortly. I hope that their proposal will benefit drugs such as Orkambi that are at the forefront of life science innovation because they treat the underlying causes of the disease, not just the symptoms, thus resulting in a lifetime of health and wellbeing benefits and savings.

[Fiona Mactaggart (Slough) (Lab)]

The hon. Gentleman is right to focus on the development of these drugs. My concern is not just that patients are denied access to life-changing drugs, but that our pharmaceutical industry finds this a frustrating country in which to develop new drugs and to ensure that they are available to people such as the constituents of my hon. Friend the Member for Dudley North (Ian Austin).

[Ben Howlett]

The right hon. Lady is quite right that pharmaceutical companies, in the rare diseases space in particular, find this country a very frustrating place to come to. The message that we are going to support the industry to bring drugs to market here is not loud and clear, and there have been a range of delays and process errors. I know that the Minister and previous Ministers have tried to address this issue, but it has been a very slow, difficult and arduous process, because the message has not been heard loudly and clearly enough.

The difficulty that Orkambi is currently facing in getting funded perfectly displays the problems faced by many other innovative drugs that aim to treat rare or genetic diseases. As chair of the APPG, I get contacted by many people across the country who are desperate to see potentially life-changing drugs approved by NICE. There is a clear deficiency in the process for this type of drug, so I hope that the Minister can today announce a pilot process to show that the UK is committed to leading in this field and providing hope for all those sufferers of rare and genetic diseases.

[Sir Alan Meale (in the Chair)]

Before we proceed, I say to Members that we are running very close to the line now. A number of Members have taken the trouble to write in and I need to try to call them all, so I ask each Member to restrict whatever they bring up to a maximum of five minutes, or hopefully less.