By the beginning of September, we should know whether the UK government supports new parliamentary recommendations to make the country a serious global contender in the field of regenerative medicine. The government has two months to respond to a report published yesterday by the House of Lords Science and Technology Committee that calls for, among other things, major changes in the regulatory structure, the clinical trials environment and the funding set-up for regenerative medicine.
This field involves replacing or regenerating cells, tissues or organs in the human body to restore or establish normal function. It includes cell therapy, gene therapy, tissue engineering and other methods, and it has enormous potential to treat and cure diseases.
The 112-page report is the outcome of an inquiry aimed at pinpointing the UK’s strengths in regenerative medicine, identifying barriers to translation and commercialisation, and recommending solutions.
Despite having a robust regulatory system, the UK still faces challenges, the committee says. These primarily involve improving perceptions and creating a more streamlined framework.
To this end, the report calls on the Health Regulatory Authority (HRA) to immediately establish a regulatory advice service that builds on the expertise of the Office for Life Sciences toolkit, the newly established Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Office and the experience of regulators.
Over the longer-term, the committee wants to see:
- the HRA commission an independent advisory group – made up of national and international experts in regulation – to develop a “designed-for-purpose”, modern and efficient regulatory system, rather than the one “that has evolved in a haphazard, piecemeal way”
- proposals from the advisory group, within 18 months of its establishment, to simplify the regulatory route so that the development of regenerative medicine and other innovative therapies is not hindered
The government should also promote global harmonisation of regulatory requirements, the report says.
Making the UK a more attractive venue for clinical trials is another key element. Among other things, the committee calls on the National Institute for Health Research to establish a regenerative medicine “stream” of its clinical research network to:
- support researchers in addressing the specific needs of regenerative medicine clinical trial design
- help overcome difficulties in identifying patients
- ensure that doctors interested in such trials can be easily identified
- facilitate dialogue with regulators on future regulatory needs and issues encountered with regulation
Filling the funding gap
At present there is insufficient private funding in this field due to the high risk of failure to translate scientific discoveries into widely used treatments. The report says that it would be unrealistic to depend exclusively upon additional funding coming from venture capitalist or “big pharma” investment.
To fill this gap, the committee recommends that the Economic and Social Research Council and the Technology Strategy Board commission an evaluation of innovative funding models, which spread risk and most likely will contain a degree of government-matched funding or be underpinned by government guarantees. The evaluation would also include proposals for how additional funding could be provided for late-stage clinical development in this field.
The committee’s recommendations also address: the evaluation and pricing of treatments by the National Institute for Health and Care Excellence; the delivery of treatments to the National Health Service and the capacity to manufacture therapies on a large scale; intellectual property considerations; and regenerative medicine tourism.
After the government issues its response, there will be a debate in the House of Lords, with a follow-up report issued in 2-3 years, a committee spokesperson confirmed.