Phil Willis diagnoses huge potential but enduring problems.
There are very few occasions when a Parliamentary report creates an almost visionary landscape that has the potential to save millions of lives, alleviate suffering and reduce health costs for future generations.
Regenerative medicine, that is the replacement or regeneration of cells, tissues and organs to restore or establish normal function, has huge potential. This is no longer in question.
There are already over a million patients who have received cell-based treatments globally. In laboratories across the USA, Europe and Asia, over 1000 clinical trials are taking place promising breakthroughs in the treatment of chronic diseases including diabetes, coronary disease and a wide range of neurological conditions associated with ageing.
For governments, the opportunities to reduce costs of dealing with long term chronic illness are highly significant.
Some 30 per cent of UK citizens suffer from chronic conditions including diabetes, strokes, dementia, heart and kidney disease which take up 75 per cent of current NHS spend. This will continue to rise with the NHS facing a funding shortfall of between £44bn and £54bn by 2022 – not to mention the very significant economic costs where people are of working age.
Regenerative medicine has the potential to provide more effective treatments and in a number of cases a cure. This is why we need a more urgent approach to ensure the UK takes advantage of its position at the very forefront of this new landscape.
The House of Lords Report, Regenerative Medicine , acknowledges the excellence of the UK academic base in the areas of cell and gene therapies, recognises an almost unique infrastructure led by a 67 million-strong NHS patient database, a growing R&D funding base and the buy-in of Ministers in BIS, Health and crucially the Treasury where the Chancellor has given the area ‘capital priority status’.
Improve funding and regulation
The report however sends out a very stark warning that theUKis in danger of losing out on regenerative medical potential, largely because of a lack of co-ordinated leadership, a lack of appropriate funding models for high-risk low-volume treatments and an over-complex regulatory environment.
As so often in the past, brilliant science is far too slow to move from laboratory to bedside as we attempt to apply traditional models of regulation, finance and governance when new, more radical approaches are necessary.
The lack of substantial private sector investment is hardly surprising. This is a relatively new field and it has potential risks which make investors wary. What is more, the development of treatments with potentially low volumes of patients is not attractive to big pharma.
To expect the newly formed Cell Therapy Catapult to support late clinical trials with a budget of £70 million spread over five years is simply not adequate. The suggestion to look at other non-UK funding models must be an urgent priority.
The French Citizens Innovation Funds model offers companies a tax-advantaged investment and tax breaks. This is one possible solution, as is the Californian model of the creation of ‘mega funds’ or secured bonds. There are also other options.
A more readily achievable goal is to streamline the regulatory system which is robust but not responsive to this fast emerging field.
The establishment of the Health Research Authority as a non-departmental government body gives it the power and scope to make significant changes, to cut out overlap, establish a single point of entry and exit and offer a comprehensive advice service to researchers and commercial companies. The notion that ‘because the science is complex so must the regulation’ is as unhelpful as it is unnecessary.
The report sets out a number of clear and achievable objectives. They must be acted upon swiftly, before other nations overtake us.