Health: Innovative Medicine

Baroness Thomas of Winchester Excerpts

Tuesday 1st April 2014 Lords Chamber

Asked by

[Baroness Thomas of Winchester]

To ask Her Majesty’s Government what steps they are taking to ensure early access to innovative medicine for life-threatening conditions.

[Baroness Thomas of Winchester (LD)]

My Lords, I am grateful to have attracted such a stellar cast for the important matters that we will be talking about in this short debate this evening. I hope that it might build on the interesting debate on 13 March on regenerative medicine, in which my noble friend Lord Willis of Knaresborough said:

“The King’s Fund estimates that by 2070, 20% of the UK’s GDP will be spent managing long-term conditions”.—[Official Report, 13/3/14; col. 1944.]

Such a situation would be unsustainable and unaffordable, and we must ensure that new treatments and technologies are found so that the quality of life of those with long-term conditions is as good as possible.

The very next day after that debate last month, the Government announced the early access to medicines scheme, which offers a way by which unlicensed medicines can be made available to patients before approval of a licence to benefit public health. The scheme has been warmly welcomed by many of us who are involved with rare-disease patient groups and their families. Indeed, with the support of the Muscular Dystrophy Campaign, the All-Party Parliamentary Group for Muscular Dystrophy, in its report last year, highlighted the need for such a scheme. Ground-breaking research for potential treatments for rare diseases is set to grow and this scheme should ensure that patients will, in future, be able to get the treatments that they need at a much earlier stage of the process.

Professor Dame Kay Davies of the department of physiology, anatomy and genetics at Oxford University is leading the development of a potential treatment for Duchenne muscular dystrophy, which is currently in early clinical trials. She said:

“The introduction of a ‘fast-track’ system offers an exciting opportunity to intervene in a safe way and ensure effective medicines reach the people who need them as early as possible. It is good news for families affected by Duchenne muscular dystrophy, who are deeply anxious about the speed at which future potential therapies will reach their children. Several potential treatments are in clinical trials and further laboratory research projects are underway—the prospect of accelerating the progress of approaches that show particular promise is a very welcome one”.

This view is supported by many families of boys with Duchenne, who say that every second counts and time is not a luxury that they have.

Professor Dame Sally Davies, the Chief Medical Officer, said that the scheme would allow drug-makers to demonstrate the value of unlicensed medicines, improving their chances of eventual approval by regulators and NICE. Obviously, allowing patients early access to medicines is not without some risks, which is why the Government were right to be clear that sufficient data must be available to demonstrate safety before a drug can be considered under the early access scheme.

The Minister will know that I was bound to mention the dismay of many of us in this field at the fact that AGNSS—the Advisory Group for National Specialised Services—was being disbanded, along with NHS Specialised Services. AGNSS was responsible for the appraisal of very rare drugs, while NHS Specialised Services had responsibility for the commissioning of services for very rare diseases, as well as a ring-fenced fund to subsidise treatments for the drugs to treat these diseases. AGNSS’s duties have transferred to NICE—the National Institute for Health and Care Excellence—while NHS England has taken over the NHS Specialised Services role. However, little clarity was given on what the new appraisal and commissioning processes will look like and there was widespread concern that NICE’s “cost per quality-adjusted life year” approach to the appraising of new drugs will effectively exclude, on the grounds of high costs, treatments for small patient populations.

NICE has now established its highly specialised technology programme, responsible for the appraisal of orphan drugs. Unfortunately, early indications of its outlook on high-cost treatments do not appear promising, although it is still at an interim stage. A current example is the approval that NICE is considering for Soliris, a treatment for atypical haemolytic uraemic syndrome, a rare blood disorder that may be inherited. Before Soliris, there was no treatment available to prevent death or organ damage and up to 25% of patients would die following their first attack. Soliris was recommended for approval by AGNSS, based on its effectiveness in halting the progress of the disease and its low cost per quality-adjusted life year. It was assumed that, as a result of this recommendation, the drug would be available to patients from October 2012. However, in January 2013 the Government announced that Soliris would be subjected to a second assessment, under the new system for specialised services within NICE, and Alexion Pharmaceuticals, which developed the drug, has been asked to explain the high cost of Soliris.

NICE has also asked for advice from NHS England on what considerations relating to the management of its specialised commissioning budget it considers should be taken into account in formulating a recommendation. I hope that the fears that many people voiced when AGNSS was disbanded that treatments for rare disorders might be denied on grounds of high costs are not going to be realised. After all, patients with rare and life-threatening conditions deserve access to treatments just as much as those with more prevalent conditions.

The next matter that I wish to raise is the clinical trials process, which must be speeded up. One way of achieving this could be to authorise a process of study approval whereby various stages of clinical trials can be conducted in parallel with one another. This would avoid a lengthy sequential process. For example, in the case of exon-skipping technologies for Duchenne, each drug or molecular patch will treat only certain specific mutations causing the condition and, under current procedures, future molecular patches would have to go through the same lengthy requirements. Will my noble friend the Minister urge the Medicines and Healthcare products Regulatory Agency and NHS England to consider such a study approval process?

Another important consideration is the clinical trial infrastructure, including additional specialist centres to enable more patients to participate in clinical trials. During the APPG inquiry, we were concerned to hear about the cutting of administrative support by some hospital trusts. It is a false economy to cut back on this infrastructure, such as patient registries. At present, many patient registries are charity-funded, with little or no long-term funding security. With limited back-office support, some centres are finding that there is no one available to input patient data. Consequently, such cuts can seriously damage the ability of centres to carry out large-scale clinical trials. Will my noble friend say what steps the Government are taking to ensure that centres and clinics across the UK have the resources required to manage patient registries and for clinical trials to be carried out?

Finally, in last month’s debate my noble friend said that consideration of the Health Research Authority’s business case to bring together and streamline NHS approvals and local ethics approvals should be completed shortly. Will he update us on that? Will he also endorse the need to streamline NHS approvals so that unnecessary regulation and delays are avoided? We must always look forward with great optimism in the search for treatments for intractable conditions. The early access to medicines scheme is a very welcome initiative, which must not be allowed to fail because of bureaucratic obstacles in its path.

[Baroness Jolly (LD)]

I remind noble Lords that we are tight for time this evening. Six minutes is the limit and, as soon as the clock reaches six, your time is up.