Science and TechnologyWritten evidence submitted by Cancer Research UK

Executive Summary

Cancer Research UK1 is the largest independent funder of cancer research in Europe. Over half of all cancer research in the UK is carried out by our doctors and scientists. Cancer Research UK’s research is entirely funded by the public. In 2010–11 we spent £332 million on research, supporting the work of more than 4,000 scientists, doctors and nurses. We fund research into all aspects of cancer from exploratory biology to clinical trials of novel and existing drugs, as well as epidemiological studies and prevention research.

As the UK’s largest independent funder of cancer research, Cancer Research UK works with many partners including industry to leverage expertise and investment in order to deliver new therapeutics and understanding about existing therapies to patients. Our work with industry extends through joint working and several key programmes. Our scientists and doctors have contributed to most of the world’s top cancer drugs.2

Our full response to the inquiry into improving the commercialisation of research is detailed below, however in summary:

We urge the Government to develop a clearer vision for the Health Research Authority (HRA), outlining how it will fit in with the rest of the regulatory and governance landscape.

Further details of the measures that will be put in place to enable Clinical Commissioning Groups (CCGs), the Secretary of State for Health and the NHS Commissioning Board to uphold their duty to promote research are needed, to help position the NHS as world-leader in research.

We believe UK Governments should maintain the diversity of funding streams for biomedical research including funding to Research Councils, Funding Councils, and National Institute of Health Research funding. They should also continue to demonstrate long-term commitment to supportive funding (such as the charity support element of Quality Related funding) that enables charities to fund world class research in universities and the NHS.

We call on UK governments to lead on the development of a strong life sciences roadmap which pulls together the various commitments into one vision for UK science, and demonstrates commitment to nourish and grow the UK’s science base, to reassure researchers and investors of their long-term support.

UK Governments should better advertise opportunities for accessing EU funding, encouraging researchers to engage with all available funding mechanisms.

We welcome Government plans to consult on the use of patient data in carefully controlled research studies. Allowing patient data to be shared can be hugely beneficial to research.

We welcome the Government’s proposal to develop an early access scheme, and want to see this taken forward swiftly with sufficient engagement across the sector.

We believe the Government will need to monitor the impact of reforms to university funding to the uptake of STEM (science, technology, engineering, maths) courses to ensure that the supply of future researchers is not disrupted.

We call on the Government to ensure that immigration policy is supportive of UK science and enables recruitment of the brightest and best scientists from all over the world.

1. What are the difficulties of funding the commercialisation of research, and how can they be overcome?

Regulation

In the UK, one of the most significant difficulties of conducting research are the barriers presented by our regulatory and governance framework. Evidence outlined in the recent Academy of Medical Sciences (AMS) Review3 outlined that the system in the UK is too complex, leading to the process for getting clinical research studies up and running being unacceptably slow.4 This presents a major cost to the commercialisation of medical research. (See Appendix, Case Study 1).

Since publication of the AMS Review in early 2011 we have seen significant progress from Government in taking forward measures to streamline the regulatory environment. Cancer Research UK supports the Government’s response to recommendations from the medical research community to improve the UK’s regulatory and governance framework. These were set out in the Budget and associated Plan for Growth and included proposals to:

Set up a new health research authority (HRA) to streamline regulation and improve the cost effectiveness of clinical trials.

Make funding from National Institute for Health Research (NIHR) conditional on meeting a 70 day turnaround time for R&D permissions; from the time a Provider receives a valid research protocol to the time when that Provider recruits the first patient for that study.

Cancer Research UK was pleased to see swift progress on establishing the Health Research Authority as a Special Health Authority, with it beginning to operate at the end of 2011. However it still remains unclear what other “regulatory organisations” will be joining the NRES (National Research Ethics Service) as part of the HRA. We are reassured that progress on developing a phased approach for the HRA to take on additional regulatory roles is ongoing, however we have yet to see clear plans and timelines outlined. We urge the Government to develop a clearer vision for the Health Research Authority (HRA), outlining how it will fit in with the rest of the regulatory and governance landscape.

NHS environment

Another major difficulty of funding the commercialisation of research is the inability to capitalise on the potential the NHS offers.5 Cancer Research UK was pleased to see that this was an area addressed in the Government’s review “Innovation, Health and Wealth”, and we support the Government in its efforts to find a solution to this problem as changes could offer possible transformative benefits to the environment for medical research.

The key points we believe will lead to the NHS being more supportive of innovation are:

The NHS Commissioning Board should set out a vision for research and innovation that outlines standards and targets for all levels of the NHS structure, and should then monitor to evaluate national progress.

Clinical Commissioning Groups (CCGs) should be supported to develop their own strategies for delivering their duty to promote research in innovation, and meet nationally set targets.

Secure mechanisms that ensure safeguards are in place should be further developed to open up data stored within patient records in the NHS. This data when used in ethically approved research studies and audit can be a major driving force for innovation in the health service.

There are significant opportunities for innovation through the introduction of stratified medicine that the NHS needs to take advantage of.

The Health and Social Care Bill provides an opportunity to ensure that innovation is promoted at all levels within the NHS. If we act now we could position the NHS as world-leader in research, and make significant contributions not only to the health of the nation but also to the economy through savings and increased investment from industry.

Further details of the measures that will be put in place to enable Clinical Commissioning Groups (CCGs), the Secretary of State for Health and the NHS Commissioning Board to uphold their duty to promote research are needed, to help position the NHS as world-leader in research.

Partnerships

Despite difficulties, the UK science base has considerable strengths which offer solutions to the problems with commercialising research.6 Many of the researchers that are attracted to work in the UK choose to come here because of its outstanding reputation as a hub for cutting-edge research.7

Biomedical research, in particular, benefits from a unique combination of stakeholders. The combination of Government support, charity and industrial partners, and National Health Service, provides a breadth and diversity that is crucial to tackle many of the health-related issues currently faced as well as those that will arise in the future.8

Each funding body, as a result of their specific priorities and motivations, will cover different aspects of research funding, and will contribute to particular stages in project development. For example, while Funding Councils provide core infrastructure support for higher education institutions, charity grants for university researchers cover the direct costs of research, but not the indirect costs, such as heating and rent for the laboratory.9

In a report, commissioned by Cancer Research UK, the Office for Health Economics found substantive benefits, both financial and qualitative, from the existence of a diversity of funders for UK medical research.10 These include:

Sharing costs and pooling risks for research programmes. This enables high cost studies that might not otherwise be funded, and permits each funder to diversify risk across a wider portfolio of projects.

Providing a stable flow of financial support for medical research in the long term.

Building an environment more conducive to research in the UK, by drawing on the differing skills and know-how of funders and through the complementarity of research funding across this diverse group of funders.

Creating a competitive research environment that has the potential to increase research quality.

Whilst our research is entirely funded by the public, the work of Cancer Research UK is highly dependent on investment from a range of government agencies. Industry, charities and the Government have different but complementary roles as research funders. The synergistic nature of these relationships, and how they link with the unique resource provided by the NHS, is a vital asset to the commercialisation of UK biomedical research.

Government funding and vision

Government funding is instrumental for overcoming the difficulties in funding the commercialisation of research. It provides a framework in which all other funders can operate, both by funding specific institutions and through policies and regulations that shape the funding environment for other investors.

We believe UK Governments should maintain the diversity of funding streams for biomedical research including funding to Research Councils, Funding Councils, and National Institute of Health Research funding. They should also continue to demonstrate long-term commitment to supportive funding (such as the charity support element of Quality Related funding) that enables charities to fund world class research in universities and the NHS.11

We call on UK governments to lead on the development of a strong life sciences roadmap which pulls together the various commitments into one vision for UK science, and demonstrates commitment to nourish and grow the UK’s science base, to reassure researchers and investors of their long-term support. The Innovation and Research strategy did go some way towards developing a long term vision. However a disadvantage of the strategy was that it did not provide any information beyond 2015. While we understand that Governments are constrained by five year terms in office; the nature of biomedical research, with an average of 17 years between basic research and patient benefit, means that Governments need to establish a long-term vision by laying out the fundamental principles for the research environment in the UK.

International Funding

Increasingly researchers in the UK are looking to other countries for funding to overcome the translational gap, or so called “valley of death”, and are winning support from the private, public and charity sectors. UK research groups have benefitted from EU funding through the Framework Programmes and from the Innovative Medicines Initiative (IMI), Europe’s largest public-private initiative.

While many UK researchers have successfully negotiated these European funding mechanisms, several have been deterred by the complicated application process. UK Governments should better advertise opportunities for accessing EU funding, encouraging researchers to engage with all available funding mechanisms.12

There are Contract Research Organisations (CRO) who will take on a “no win no fee” role in helping scientists with applications for EU funding grants. Cancer Research UK believes that there is an opportunity for the Government to explore the potential of these firms to help researchers maximise their engagement with international funding opportunities.

2. Are there specific science and engineering sectors where it is particularly difficult to commercialise research? Are there common difficulties and common solutions across sectors?

Rare cancers

Kidney cancer is a rare disease, and an example of a specific science sector where is particularly difficult to commercialise research. Only a few thousand people are diagnosed with kidney cancer every year, and only one in 10 people diagnosed surviving beyond five years of diagnosis.13 In 2008 the only available treatment option was immunotherapy; if patients were not suitable for immunotherapy, or if it did not work, there were no other options.

Subnitnib, a new generation kidney cancer treatment was approved by NICE in 2009. While this drug was being developed, it took a relatively long time to run large enough trials to gather sufficient evidence for it to be approved because of the low numbers involved.

As we have already commented, if we make real progress on streamlining the regulatory environment for clinical trials in the UK, combined with advance in taking a stratified approach to medicine, should enable us to get trials to these rarer patient populations quicker.

Early Access

It takes around 17 years to get a drug from the early stages of development to patients. We believe that once we know that drugs are safe , under strict supervision and initially for conditions where there are no other treatment options, we should explore options that would bring drugs to patients earlier in the drug development process, in the hope that patients can reap the benefits of promising new drugs sooner.14

The reason that this is so important is that many cancer patients find themselves in the devastating position of having no treatment options available to them—even though there are new drugs in the development pipeline from which they might benefit.

This measure will help to bridge the “valley of death”, as it should create strong incentives for companies to develop drugs for smaller groups of patients, with the potential for an earlier return on their investment. This in turn, has the potential to encourage pharmaceutical companies to invest in the UK. We welcome the Government’s proposal to develop an early access scheme, and want to see this taken forward swiftly with sufficient engagement across the sector.

Medical Physics

Research into medical physics is a very different process to biomedical research. Feedback from the medical physics community of Cancer Research UK researchers strongly indicates that the barriers to commercialisation for medical physics are not widely acknowledged and therefore there has been less progress in this area than there has been for biomedical research in recent years (see Case Study 2). Cancer Research UK believes more needs to be done to understand the distinct pathway to commercialisation for medical physics and medical technology and to help us identify the specific difficulties in the commercialisation of this research area.

4. What evidence is there that Government and Technology Strategy Board initiatives to date have improved the commercialisation of research?

Stratified medicine

Cancer Research UK is working with AstraZeneca, Pfizer and the Government on an exciting Stratified Medicine programme which will allow the NHS to adopt new targeted therapies and make the UK a better place for research into more personalised cancer treatment.

Stratified medicine has the potential to benefit patients, the NHS, the UK economy, research and academia. Patients will get more effective treatments, with fewer side effects. The NHS will improve prescription cost-effectiveness, and the UK economy will benefit from attracting investment from the life sciences industry, improving the commercialisation of medical research.

The revenues from stratified cancer medicines could be higher than those generated through the traditional business model. Superior clinical performance should lead to rapid and wide adoption of stratified medicine; effective patent life should be longer because development times are shorter, and peripheral benefits, such as cheaper marketing costs and revenue from associated diagnostics and biomarker tests, will all serve to enhance profitability. Patient stratification would be a competitive advantage for companies seeking market access.

5. What impact will the Government’s innovation, research and growth strategies have on bridging the valley of death?

Patient data “opt out”

Government Strategy for Life Sciences plans to consult on changes to NHS constitution to allow for an “opt out” of use of patient data in research.

At Cancer Research UK we depend on patient data enormously for our lifesaving research. Analysing patients’ records has helped us understand the causes of cancer, including how to prevent the disease and to diagnose it at an earlier stage when treatment is most likely to be successful.

We therefore welcome Government plans to consult on the use of patient data in carefully controlled research studies. Allowing patient data to be shared can be hugely beneficial to research but the process must be subject to strict safeguards. It is very important that the Government gains public support for the plan, if it is to simplify the regulatory environment for the benefit of patients and researchers.

7. What other types of investment or support should the Government develop?

Charity Research Support Fund

Charities chose to fund research in universities because of the world-class research environments they provide. The dual support system for university research is a key factor underpinning this excellence. The Quality Research (QR) block grant from the Higher Education Funding Council for England (HEFCE) builds strong autonomous universities by giving them the stability, flexibility and freedom to make strategic decisions about their own research activities. The charity research support element (known as the Charity Research Support Fund) is a vital part of QR as it enables Government funding to leverage additional partnership funding from the charity sector.15

Universities and charities need to be able to plan their future funding and research strategies with the secure knowledge that the dual support system, and the important charity support element within it, will continue. A “stop-start” approach could lead to the attrition of the research base, and could disproportionately affect progress in medical research in universities, where the majority of charitable funding is focused.

Cancer Research UK calls on the government to reaffirm its commitment to ongoing partnership funding to ensure the stability of charity- funded research in universities in England.

Francis Crick Institute

The Francis Crick Institute will be Europe’s largest biomedical research facility. Cancer Research UK is a key partner, along with the Medical Research Council (MRC), University College London, the Wellcome Trust, Kings College, and Imperial College.16

We are delighted that the Government has confirmed investment for the Francis Crick Institute17 which represents a unique partnership between six of the UK’s most successful scientific institutions. It will champion an approach in which teams working in different disciplines collaborate to uncover fundamental biological mechanisms relevant to human health. Groups spanning the biological, clinical and physical sciences will share insight and techniques to capture a more complete understanding of life’s processes. Bringing specialists together to work on the greatest health challenges will increase the pace of discovery.

The commercialisation of research is one of the core principles of the institute. Technology transfer will be a fully integrated activity with equal prestige to discovery research. Researchers will work closely with the existing technology transfer bodies of the founding partners and the Institute will also have its own technology transfer staff.

Investment in People

Excellent people are the foundation for excellent science. Medical Research relies on scientists involved in basic, translational and clinical research.18

The future of UK research depends upon a continued supply of high quality researchers. The two central elements to this are the need to attract students into science, and, once trained, to support their on going development and encourage them to remain in science.

Proposals for tuition fees to be the major source of university income could mean that institutions might consider compromising their preferred course content to attract students and this could be detrimental to the quality of UK science and scientists. We believe the Government will need to monitor the impact of reforms to university funding to the uptake of STEM (science, technology, engineering, maths) courses to ensure that the supply of future researchers is not disrupted.

The UK research base depends not only on the contribution of British scientists, but also on the contribution of researchers from other countries, including those from outside the EU. The Government needs to be mindful that its immigration policy is supportive of UK science and enables recruitment of the brightest and best scientists from all over the world.

Cancer Research UK calls on the Government to ensure that immigration policy is supportive of UK science and enables recruitment of the brightest and best scientists from all over the world.

February 2012

APPENDIX

Case Study 1

Closure of CR-UK funded bladder cancer trial due to regulation and governance

The SPARE trial, launched in 2007, was a multi-centre feasibility pilot study, involving muscle invasive bladder cancer (MIBC) patients. Unfortunately, recruitment rates were not sufficient for the trial to progress to a phase III study, which would have determined standard of care in MIBC patients, and it was closed in January 2010. This is a huge disappointment to the UK and international urological oncology community, especially as the President of the British Association of Urological Surgeons had endorsed the study as one of the most important clinical questions facing UK urology.

One of the major factors identified as a reason why recruitment to the SPARE trial did not reach anticipated levels was challenging bureaucracy.

The time taken from a centre expressing interest in the trial to being ready to recruit patients was excessively long: 36 centres opened to recruitment during SPARE set-up and the average time taken from gaining first approval of the Site Specific Information Form to opening was 8.6 months (range 1.6–18.1 months). Most of the delay involved Trust R&D departments, with them often taking a long time to authorise the standard clinical trial agreement between the Trust and trial sponsor. This was exacerbated by the need to ensure that all Principal Investigators had adequate Good Clinical Practice training.

Case Study 2

Commercialisation of medical technology

In the last 45 years, only two medical technologies have been successfully commercialised in the UK. Osiris, an application for generating body contours from laser lines on a patient, was developed by Robin Wilkes and commercialised after he retired from the NHS; and Roy Bentley’s pioneering treatment planning system which was developed in the 1970s.

Over the same time period, a department of the University of Wisconsin (UW) has researched, developed and commercialised solid water; Thermoluminescence Dosimetry (TLD); Pinnacle, the world’s leading planning system; Tomotherapy, the world’s leading arc therapy treatment machine; and the compact charged particle accelerator.

Rock Mackie, UW Professor of medical physics, human oncology and nuclear engineering and the most commercially successful medical physicist in the world, is currently heading a UW department specifically focused on the commercialisation of intellectual property (IP).

The feedback from the Cancer Research community of medical physicists suggests that this success is due to the fact that all discoveries are IP protected and patented to individual researchers and inventors. Proceeds from the successful commercialisation of technologies are therefore passed onto individuals. This creates a culture of extensive IP cover and investment into research as individuals perceive they can benefit from the commercialisation process.

1 Registered charity no 1089464.

2 The “top” cancer drugs were defined using worldwide sales figures from 2005 (DataMonitor Data). Sales figures were from the US, UK, Japan, Italy, France, Germany and Spain. Examples of these drugs include temozolomide, anastrozole and herceptin.

3 Academy of Medical Sciences (2011): A new pathway for the regulation and governance of medical research.

4 Cancer Research UK submission to Office for Life Sciences Review September 2011.

5 Cancer Research UK submission to Office for Life Sciences Review September 2011.

6 Cancer Research UK submission to Office for Life Sciences Review September 2011.

7 Martina Garau, Arik Mordoh, Jon Sussex, Exploring the Interdependency between Public and Charitable Medical Research (April, 2011).

8 The Academy of Medical Sciences raised many of these issues in its submission to the Spending Review,
http://www.acmedsci.ac.uk/p48prid83.html

9 Cancer Research UK, 2011. Building the Ideal Environment for Medical Research.

10 Martina Garau, Arik Mordoh, Jon Sussex, Exploring the Interdependency between Public and Charitable Medical Research (April, 2011).

11 Cancer Research UK, 2011. Building the Ideal Environment for Medical Research.

12 Cancer Research UK, 2011. Building the Ideal Environment for Medical Research.

13 Sarah Woolnough, Bringing Science to Life, The New Statesman, January 2012, p 12–14.

14 Sarah Woolnough, Bringing Science to Life, The New Statesman, January 2012, p12–14.

15 Government support for charity funded research in universities: A joint statement from universities and charities in the UK.

16 www.crick.ac.uk/

17 Sarah Woolnough, Bringing Science to Life, The New Statesman, January 2012, p 1214.

18 Cancer Research UK, 2011. Building the Ideal Environment for Medical Research.

Prepared 11th March 2013