11 March 2015
UK Health Secretary Jeremy Hunt and Life Sciences Minister George Freeman are due to announce new investment and actions intended to maintain the country’s standing as a leader in medical innovations and accelerate their uptake within the National Health Service (NHS).
The announcement is to incorporate several important developments, including the first novel diagnoses from the 100,000 Genomes Project and the first drug approval through the Early Access to Medicine Scheme (EAMS), as well as plans to build two cutting-edge proton beam therapy cancer treatment centres in London and Manchester to open in 2018, and a new review into accelerated uptake of medical innovations into the NHS.
First patient diagnoses from 100,000 Genomes Project
Two families are reported to have benefited from clinical whole genome sequencing at Newcastle Hospitals and University in the form of specific diagnosis of rare genetic disorders, said to allow personalised treatment and care for the patients and families.
The first of these case studies identified a rare form of inherited kidney disease in one family; diagnosis of one patient with kidney failure and a family history of similar problems will now allow testing of the rest of the family and careful monitoring and control of blood pressure and kidney function in those who have inherited the causative genetic mutation. The second case involves diagnosis of two adult brothers with a new mutation causing peripheral neuropathy; it is hoped that new treatments currently in development may allow other family members who have inherited the genetic mutation to avoid developing the same condition.
Genomics England has also announced today that is has received ethical approval from a Health Research Authority Research Ethics Committee (REC) for the main phase of the 100,000 Genomes Project. This means that the nominated NHS Genomic Medicine Centres can start inviting patients and their families to join the project and receive whole genome sequencing and analysis; previous participants have been part of early pilot phases projects with individual research ethics approval.
Developments in precise and personalised cancer treatments
The first drug approved via the fast-track EAMS is reportedly pembrolizumab, a treatment for advanced melanoma. It is in fact not technically a drug but a biologic, an engineered antibody-based agent that acts to stimulate anti-tumour responses from a patient’s own immune system.
The new proton-beam therapy centres will use this extra-precise radiotherapy technique for selected forms of cancer that can minimise the harmful side-effects of treatment on healthy tissues surrounding tumours, including the enhanced risk of subsequent secondary cancers. The technique featured prominently in the UK news last year when the parents of five-year old brain tumour patient Ashya King faced arrest for removing him from an NHS hospital and taking him to Europe for proton beam therapy against medical advice.
Speeding up access to biomedical innovations
Underlining the government’s commitment to promoting both health and wealth for the UK by building on the successful life sciences sector, the announcement will also name Sir Hugh Taylor, Chair of Guy’s and St Thomas’ NHS Foundation Trust, as the chair of a special review examining ways to ‘revolutionise and dramatically increase the speed at which 21st century innovations such as precision medicines, digital devices, apps, diagnostics and new therapeutic technologies get to patients and their families’.
Supported by the Wellcome Trust and an external advisory group chaired by Professor Sir John Bell, Regius Professor of Medicine at Oxford University, the Innovative Medicines and Medical Technology Review will examine the regulation and reimbursement systems for biomedical innovations and how these and other factors affect the speed of adoption in the NHS.
The primary focus is on treatments, but medical devices and diagnostics will also be important. For example, the rapidly evolving methods for testing complex genomic and other forms of biomarkers, for analysing genomic and other forms of biomedical big data, and for linked diagnostics and therapeutics (companion diagnostics), which identify whether or not a given patient will benefit from the associated targeted treatment.
The review will involve consideration of the roles of NICE, the MHRA and the NIHR , including in the light of technological convergence that is ‘breaking down traditional barriers between regulator categories’; the possibilities for integrated specialist commissioning systems; how the NHS can work to generate evidence of the utility and cost effectiveness of innovative drugs, devices and diagnostics; and take into account the impact of emerging technologies such as stratified medicines and digital health technologies.
The chance to make an enduring difference for health
Life Sciences Minister George Freeman said: "The explosion of biomedical innovation - whether in genomics, regenerative medicine or digital health is transforming 21st century medicine. But in recent years too many NHS patients have had to wait too long to access new treatments and slow uptake also threatens life science industry investment”.
Plans to include clinical perspectives in the review are promising, since views from all major stakeholders often provide critical insights in identifying the practical barriers to NHS uptake, allowing the creation of robust policies to circumvent them. True acceleration has to address the whole process of adoption and not merely enhance translational research; developing a supportive regulatory and public policy environment is crucial.
Equally important is a receptive NHS, preparing clinicians for knowledgeable leadership in their own services, (whether these are in teaching hospitals or elsewhere), working with them to understand how innovations can improve and streamline clinical pathways and to develop new guidelines for effective practice. Clinicians, health service funders, regulators, managers and patients must work together to take the science and technological innovation from research into practice. But, vitally, it has to be recognized that these changes do not 'just happen' - they need comprehensive 'knowledge brokering' and focused strategic development - an approach that PHG Foundation has pioneered for more than 15 years.
The PHG Foundation strongly welcomes the government announcement, and will be launching its own innovation manifesto’ to address many of the same issues and ensure that scientific innovations are made to work efficiently for health later this month.