The government recognizes the destructive effects of motor neuron disease. Providing NHS patients with access to the most effective and innovative drugs and treatments is a top priority. 

We are very sorry to hear that someone has been diagnosed with motor neuron disease (MND). We recognize the tremendous challenges faced by those who suffer from this truly devastating and incurable disease. 

The government understands the importance of patients having access to the most effective medicines and treatments. We make the NHS one of the most pro-innovative healthcare systems in the world. The NHS multi-year plan and industry agreements for life sciences provide a clear vision of a health system that prioritizes innovation and faster patient access to life-changing products. The Accelerated Access Collaborative (AAC) brings together government, NHS and industry leaders to address the barriers to innovation development and adoption in the UK. 

All medicines must have a marketing authorization (permit) before they can be placed on the market and given to patients outside of a regulated clinical trial. Licenses are granted on the basis of a positive benefit / risk assessment by a regulatory authority, taking into account the results of animal studies and clinical trial data designed to demonstrate efficacy and safety in humans. 

To conduct a clinical trial, a company will submit 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 that clinical trials are conducted correctly. The company that produces NurOwn can choose where the clinical trials will be conducted and in this case the company has not conducted any in the UK. 

There are several schemes under which UK patients may have access to medicines before they are licensed. The Early Access to Medicines Scheme (EAMS) aims to provide patients with life-threatening or severely disabling conditions with access to medicines that are not yet licensed. Companies can choose whether to participate in the scheme, which runs parallel to the normal licensing procedure. To date, no EAMS application has been received by the NurOwn manufacturer. British patients may also be able to access medicines UK patients may also be able to access unlicensed medicines that have been specially manufactured or imported on behalf of a physician for the treatment of individual patients. UK law provides an exception to the need for a marketing authorization for a medicine that is issued on the order of a doctor and is intended for use by a patient for whose treatment that person is directly responsible. It is important that we have a system for making fact-based decisions about whether new drugs should be routinely funded by the NHS. In England, the National Institute for Health and Care Excellence (NICE) makes evidence-based recommendations to the NHS on whether new licensed drugs are a clinical and cost-effective use of resources. Where NICE recommends a drug, NHS commissioners are required by law to make routine funding available so that clinicians can offer it to their patients. Since NurOwn is not yet licensed in this country, NICE is unable to rate this drug. 

More generally, we invest over £ 1 billion a year through the National Institute for Health Research (NIHR), to fund research and 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 collaboration in the life sciences industry and contract research across the entire translation process, from early translational (experimental medicine) research, through clinical research to applied health research, including the generation of high-quality evidence to support the development and licensing of new medicines in the UK and elsewhere. 

As for the drug inclisiran, (a new cholesterol-lowering drug), we announced the innovative partnership in public health with Novartis in January 2020. This is a world-leading example of British clinical trial innovation and technical expertise. It includes supporting a clinical trial with inclisiran, guiding the path towards implementing a new approach to care, protecting more people from cardiovascular disease, and laying the foundation for more efficient production of new medicines . While the UK has always been open to industry partnerships for innovative medicines that would benefit the UK population, any medicine would be subject to MHRA and NICE approval processes. By extension, inclisiran is fully subject to approval and assessment by MHRA and NICE and the same approval standard as any other medicine.