The Parliamentary Under-Secretary of State, Department of Health (Earl Howe) (Con)
My Lords, I congratulate my noble friend Lady Thomas on securing this debate and I am grateful to her for providing the opportunity to update your Lordships on, in particular, the early access to medicines scheme. It is just one way in which the Government are supporting improving patients’ access to new medicines.
I begin by making it clear that our priority is to ensure that patients, including those with rare and life-threatening or life-limiting conditions, have access to new and effective treatments on terms that represent value to the NHS and the taxpayer. I agree with the noble Baroness, Lady Masham, that it is essential that people get the medicines and treatment that they need. That is why we have set up the cancer drugs fund and why we have NICE to give evidence-based advice to clinicians and the NHS.
On 5 December 2011, the Prime Minister announced a new strategy for UK life sciences. One of its commitments was that,
“early in 2012 the MHRA will bring forward for consultation proposals for an ‘Early Access Scheme’”.
I am pleased to be able to say that, following public consultation co-ordinated by the Medicines and Healthcare products Regulatory Agency—known as the MHRA—and engagement with patient groups and industry, we announced the early access to medicines scheme on 14 March. The purpose of the early access to medicines scheme is to support access in the UK to promising new, unlicensed or off-label medicines in areas of unmet medical need. Under the scheme, the Secretary of State for Health, acting through the MHRA, will provide a scientific opinion on such medicines to treat, diagnose or prevent life-threatening or seriously debilitating conditions that do not have adequate treatment options. This could include patients with advanced cancer or children with Duchenne muscular dystrophy.
MHRA is responsible for managing the scientific aspects of the scheme, which will follow a two-step process. Step one involves giving a new medicine a promising innovative medicines designation, known as a PIM designation, which will provide an early indication that a product may be a possible candidate for entry into the early access to medicines scheme, based on the available clinical data. Companies that wish to move to step two must hold a PIM designation and provide further relevant data on their product’s quality, safety and efficacy. At step two, the MHRA will produce a scientific opinion describing the benefits and risks of the medicine, based on information submitted by the applicant after sufficient data have been gathered from the patients who will benefit from the medicine.
The scientific opinion will be made available on the MHRA’s website to assist clinicians and patients in making treatment decisions and to support informed consent by patients by informing them of the risks and benefits of the product. The scheme will be launched and ready to receive applications from Monday of next week, 7 April 2014. I understand that full details, together with guidance, will be published on the MHRA website at this time.
The noble Lords, Lord Kakkar and Lord Hunt, asked about equal access for patients under the scheme. EAMS medicines will be provided for free by the company concerned. The scientific opinion will be available on the MHRA website, as I have mentioned, to allow doctors and patients to make treatment decisions. That will provide an equitable platform for patient access.
The noble Lord, Lord Kakkar, also asked about academic science input into the scheme. The scheme is open to academics, industry and charities, provided that the criteria are met. Step one, the PIM designation, would also be open to academic research units such as at UCL. As the noble Lord mentioned, academic health science networks could well have a role in promoting the scheme. All AHSNs are now up and running and their funding is assured for the immediate future.
It is important to recognise that the early access to medicines scheme is a UK-only scheme that relates to unlicensed medicines en route to market. It is always better for a patient to receive a licensed medicine where possible and for companies to have the legal certainties that come with a marketing authorisation. For this reason, the MHRA continues to engage at both European and global level to explore how the medicines licensing process can become more efficient. We welcome the European Medicines Agency’s launch of its adaptive licensing pilot on 19 March 2014, as the MHRA has had a leading role in the preparation of the pilot and accompanying guidance.
Adaptive licensing is a prospectively planned, adaptive approach to bringing drugs to the market. It seeks to maximise the positive impact of new drugs on public health by balancing timely access for patients with the need to provide adequate evolving information on benefits and harms. Adaptive licensing uses the regulatory processes and flexibilities within the existing EU legal framework, such as conditional marketing authorisation. The pilot will explore the strengths and weaknesses of all options for development, assessment, licensing, reimbursement, monitoring and utilisation pathways in a confidential manner and without commitment from regulator or company. I plan to meet relevant government and industry partners later this month to ensure that the UK can capitalise on the pilot. I hope that UK-based companies will be at the forefront of those presenting products to the EMA.
The noble Lord, Lord Kakkar, asked about the draft Medical Innovation Bill, which aims to encourage responsible innovation in certain circumstances and to discourage irresponsible innovation. I echo the noble Lord’s thanks to my noble friend Lord Saatchi for putting these concepts before your Lordships’ House previously. We are currently consulting on this draft Bill. The consultation paper was published on 27 February and the consultation runs until 25 April. For that reason, I am afraid that I am not yet in a position to make a definitive pronouncement on the Bill’s provisions.
The noble Lord, Lord Kakkar, asked about equality of access to specialised services. NHS England, as he knows, is now responsible for commissioning prescribed specialised services across England, so patients should know what services they can expect. The Government remain totally committed to making the UK a world-leading place for life sciences investment. The noble Lord was right to say that trialling drugs is an expensive business, but good progress is being made through the life sciences strategy to build a better life science ecosystem to attract and develop talent, to reward innovation and to overcome barriers to innovation. The Strategy for UK Life Sciences states:
“It has become increasingly challenging for life sciences companies, particularly SMEs, to discover, develop and commercialise medical innovation”.
AHSNs, as the noble Lord rightly said, have therefore been set up as a key response to these challenges, acting as the link between the NHS and industry. I think that the universal coverage of AHSNs has had multiple benefits in terms of potential UK growth.
The noble Lord, Lord Walton, and my noble friend Lady Thomas mentioned ultra-orphan drugs. We are aware of the challenges posed by treatments for the very rare conditions, with small patient populations, for which these drugs are made. My noble friend expressed particular worry about eculizumab, or Soliris. From April 2013, NICE has been responsible for the evaluation of selected highly specialised health technologies. It issued draft guidance on 27 February which does not recommend the drug’s use. Stakeholders had until 25 March to submit comments to NICE. I must stress that NICE has not yet issued its final guidance on eculizumab to the NHS and I understand that that is expected in July. While NICE undertakes its evaluation, NHS England has developed an interim commissioning policy to enable patients with aHUS to receive eculizumab. To clarify, AGNSS found that eculizumab was clinically effective but that further information was needed to demonstrate its cost-effectiveness. That is why we tasked NICE with evaluating it.
All candidate medicines have the potential to induce toxicity. I will address the interesting speech of my noble friend Lord Colwyn by reminding noble Lords that medicines must undergo a series of rigorous assessments, progressing from small cohorts of patients in rigorously controlled conditions to larger and more diverse groups of patients, thus ensuring quality, safety and efficacy. We have three phases of clinical trials to ensure that those things are assured.
It is possible to run adaptive licensing design studies that allow for modifications as the trial progresses; for example, the numbers of different treatment arms can be tried out. Such designs have the potential to speed up clinical development and can use resources more efficiently. There is regulatory guidance on adaptive designs. I respond to my noble friend Lady Thomas by welcoming many features of the new clinical trials regulation, which provides for a more streamlined approach, with the introduction of combined clinical trial and ethical approval and a single portal for all EU applications.
I share the enthusiasm and excitement of the noble Lord, Lord Walton, about the developments in genetic medicine. That is why we have established Genomics England, a development that has put us firmly at the head of the field.
The noble Lord, Lord Hunt, asked what we are doing to ensure the rapid uptake of NICE-recommended drugs in the NHS. Innovation, Health and Wealth, a document that we published in 2011, set out a range of measures to support the rapid uptake of NICE-approved medicines in the NHS, including the establishment of the NICE Implementation Collaborative, the automatic incorporation of NICE-recommended drugs into local formularies and the introduction of an innovation scorecard to compare local uptake. Good progress has been made in delivering those commitments, but we recognise that more can and should be done. We are committed to seeing Innovation, Health and Wealth progress.
There are variations in drug usage among CCGs. As the report points out, there can be many reasons for variation. Different areas may have different health needs and it is right that the treatments used should be decided by doctors and patients. As I have said to the noble Lord before, we are committed to tackling unjustified variation in the usage of medicines and we encourage NHS organisations to consider the findings of the report in the context of the needs of their populations.
As my time is running out, I will have to write to noble Lords to cover those issues to which I have not yet managed to reply. However, in conclusion, I was pleased to announce yesterday my approval of the business case presented by the Health Research Authority and the funding that goes with that to enable it to fulfil its remit. The HRA will provide a single approval for research in the NHS to radically streamline and simplify how studies are set up. I believe that the UK’s approach of allowing patients access to promising but as yet unlicensed medicines while encouraging greater use of European licensing flexibilities will provide much earlier access to a number of innovative new medicines, in particular in areas of unmet need. We can be proud of the leading role that we play in ensuring that the UK remains one of the leading countries in which to develop medicines and to see them reach the patient’s bedside in clinical use.