Petition Explore options for making NurOwn available to treat Motor Neurone Disease (MND)
No effective treatment exists for 5,000 sufferers of MND in the UK. NurOwn is in the final months of Phase 3 of a US FDA regulated clinical trial. Phase 2 data indicated NurOwn was safe and demonstrated signs of efficacy at low doses.
The Government should explore options for making NurOwn available to treat MND. Either early availability / approval of is sought or access to treatment that, at the latest, coincides with FDA approval. An Accelerated Access Review (AAR), Accelerated Assessment (MHRA), Accelerated Access Collaborative (NICE), a clinical trial and collaboration between the NHS and drug company are different means of expediting availability and access to NurOwn for the treatment of MND in the UK.
Inclisiran is a drug that in January 2020 had not yet been approved in the UK or EU. In January the Secretary of State for Health and Social Care approved one of the world's largest drug trials in a collaboration between Novartis and the NHS, with the latter funding and overseeing treatment.
This response was given on 7 May 2020
The Government recognises the devastating effects of motor neurone disease. It is a key priority to provide access to the most effective and innovative medicines and treatments for NHS patients.
Read the response in full
We are very sorry to learn about anyone who has been diagnosed with motor neurone disease (MND). We recognise the immense challenges faced by those who are suffering with this truly devastating and incurable disease.
The Government understands how important it is for patients to have access to the most effective medicines and treatments. We are making the NHS one of the most pro-innovation healthcare systems in the world. The NHS Long-Term Plan and the Life Sciences Sector Deals set out a clear vision of a health system which prioritises innovation and delivers faster patient access to life-changing products. The Accelerated Access Collaborative (AAC), brings together the leaders from Government, the NHS and industry to tackle the barriers to the development and adoption of innovation in the UK.
All medicinal products are required to have a marketing authorisation (a licence) before they can be put on the market and given to patients outside of a regulated clinical trial. Licences are given based on a positive benefit/risk assessment by a regulatory authority considering the results of animal studies and data from clinical trials which are designed to show efficacy and safety in humans.
To run a clinical trial, a company will make an application to a regulatory authority (in the UK this is the Medicines and Healthcare products Regulatory Agency), and a research ethics committee, to ensure the clinical trials are conducted appropriately. The company that manufactures NurOwn can choose where to conduct their clinical trials and in this case the company has not conducted one in the UK.
There are various schemes by which UK patients may be able to access medicines before they are licensed. The early access to medicines scheme (EAMS) aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation. Companies can choose whether to participate in the scheme, which runs in parallel with the normal licensing procedure. To date no EAMS application has been received by the manufacturer of NurOwn. UK patients may also be able to get access to unlicensed medicinal products which have been specially manufactured or imported to the order of a doctor for the treatment of individual patients. UK legislation provides an exemption from the need for a marketing authorisation for a medicinal product which is supplied in response to an order from a doctor and is intended for use by a patient for whose treatment that person is directly responsible for.
It is important that we have a system in place for making evidence-based decisions on whether new medicines should be routinely funded by the NHS. In England the National Institute for Health and Care Excellence (NICE), makes evidence-based recommendations for the NHS on whether new licensed medicines represent a clinical and cost-effective use of resources. Where NICE recommends a medicine, NHS commissioners are legally required to make routine funding available so that clinicians can offer it to their patients. As NurOwn is not yet licensed in this country NICE is not in a position to appraise this drug.
More widely we are investing over £1bn per year through the National Institute for Health Research (NIHR), to fund research, and to provide the skills and facilities to enable high quality research in our health and care system. The NIHR provides the best possible environment for collaboration between the life sciences industry, charities, academia and the NHS. It supports, facilitates and enables life sciences industry collaborative and contract research, across the translational pathway from early translational (experimental medicine) research, through clinical research, to applied health research, including generation of high-quality evidence to support development and licencing of new medicines in the UK and elsewhere.
With regard to the drug inclisiran, (a new cholesterol lowering drug), in January 2020 we announced the innovative population health collaboration with Novartis. This is a world-leading example of the UK’s clinical trial innovation and technical expertise. It includes supporting a clinical trial of inclisiran, guiding the way to implementation of a new approach to care so that more people are protected against cardiovascular disease, and laying the foundations for more efficient manufacturing of new drugs. While the UK has always been open to collaborations with industry on innovative medicines that would benefit the UK population, any medicine would be subject MHRA and NICE approval processes. In line with this, inclisiran is completely subject to MHRA and NICE approval and assessment, and to the same standard of approval as any other medicine.
Department of Health and Social Care
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