Science and TechnologyWritten evidence submitted by the Association of Medical Research Charities

1. We welcome the opportunity to contribute to this inquiry.

2. The Association of Medical Research Charities (AMRC) is a membership organisation of the leading medical and health research charities in the UK.

3. Charities are part of the UK’s diverse funding landscape. In 2011–12, AMRC’s 124 medical research charities invested over £1 billion in health research.1 In the same year, over 3,000 clinical studies were conducted in the NHS; 37% were funded by AMRC member charities. This investment contributes to the UK’s strength in biomedical science, bringing economic benefits. All our members are focused on benefiting patients and share a commitment to funding the highest quality research and many have a strong patient group allied to them.

4. In this response we use case studies of current charity practice to demonstrate the role of medical research charities in the UK’s research funding ecosystem. Responding to your questions we demonstrate some of the key blockages and how charities could play a greater role working with others in industry and universities to accelerate the commercialisation of research.

5. Key Points

Supporting medical research is the most popular reason for people to donate to charities in the UK. Therefore charities have a strong mandate to ensure their investments deliver better healthcare for patients. Successful commercialisation of research is key to achieving this.

Therefore charity and industry priorities to commercialise research overlap.

Government must focus on providing the infrastructure to underpin collaboration and successfully drive commercialisation. This includes measures underway to streamline regulation and create a research-friendly NHS, and also proactive work to increase market certainty, focus funding to increase research capacity in key areas and boosting academic engagement in commercialisation.

Charities are hubs of expertise playing a unique role as an honest broker between patients, academia and the commercial sector.

Charities pull innovation through the system by identifying and championing investment opportunities and developing valuable market data. As experts in their disease area they can identify gaps in research and capacity and proactively drive exploration of opportunities with researchers, universities and commercial investors.

Charities de-risk investments by providing vital funding for early stage research. Charities of all sizes can work constructively with private investors to de-risk their investment.

Charities provide a patient focus. This can improve design of research studies, recruitment of patients and uptake of commercial products, combating market uncertainty.

The Life Sciences Advisory Board membership should include charities who can represent the patient voice. The third sector offers valuable expertise in developing innovative funding strategies focused around patient need.

6. The Role of Charities in Funding Medical Research in the UK

The UK is relatively unique in having a strong medical research charity funding sector. Medical research is the most popular cause people choose to support in the UK with over 11 million people giving on a monthly basis.2 AMRC has 124 members with a range of sizes:

7. Together AMRC member charities invested over £1 billion in health research in 2010–11.3 These charities are part of a diverse funding landscape which helps create a stable research environment. This makes the UK an attractive place to invest.

8. An Office for Health Economics analysis commissioned by Cancer Research UK4 shows the value of this interaction and the interdependency of funding, creating economies of scale which attract further investment. This finding built on previous work looking at US models which demonstrated that charitable investment can attract industry investment:

“A £1 increase in UK government or charity spending on medical research could lead to an increase in private research spending from the pharmaceutical industry of between £2.20 and £5.10.”5

9. Charities are hubs of expertise. Many have direct links with people with medical conditions and work with leading clinicians in the field, bringing together research knowledge and patient experience. They can play a unique role as an honest broker between patients, academia and the commercial sector.

10. Charities can pull innovation through the system by identifying and championing investment opportunities and developing valuable market data. As experts in their disease area they can identify gaps in research and capacity and proactively drive exploration of opportunities with researchers, universities and commercial investors.

Case Study 1—The Ministerial Advisory Group on Dementia Research

The Ministerial Advisory Group on Dementia Research includes many AMRC member charities—Alzheimer’s Society, Alzheimer’s Research UK, Age UK, Parkinson’s UK, The Stroke Association and the Wellcome Trust. This group has ensured dementia research is a priority on the political agenda and have produced a route map for dementia research.

Alzheimer’s Research UK recently published Defeating Dementia 20126 to push for the development of a long-term national dementia research strategy. This conducts analysis of UK dementia research, noting the areas of excellence and the capacity building needed to maximise this. Initiatives driven by this group will maintain/attract industry investment in dementia research in the UK.

Case Study 2—Investing in Infrastructure for Research

Breast Cancer Campaign conducted a wide scale involving over 50 of the world’s leading breast cancer researchers. This highlighted that the one of the main barriers to progress was a shortage of good quality tissue—the raw material for research.

In response to this, Breast Cancer Campaign have established a unique collaboration with four leading research institutions to create a vital resource of breast cancer tissue for researchers across the UK and Ireland—the UK’s first ever national cancer tissue bank.

Tissue samples donated by patients from across the UK will be safely and consistently stored. These samples will then be available to scientists to study how and why breast cancer develops and spreads, and to devise the best possible treatments. There is currently no other large source of breast tissue available to scientists and doctors, like this one, anywhere in the world.

Further information available here: [accessed 22 September 2011]

Case Study 3—Action on Hearing Loss Translational Research Initiative for Hearing (TRIH)

Action on Hearing Loss has established a Translational Research Initiative for Hearing (TRIH) which is focused on supporting translational research to advance medicines to protect and restore hearing, and silence tinnitus.

The initiative is designed to support translational research:

Through a new funding scheme.

By facilitating partnerships between industry, academics, investors, philanthropists and others.

By engaging patients to support, and to demand, new treatments and cures for their hearing loss and tinnitus.

Action on Hearing Loss has developed tailored portals for different partners in the process:

TRIH Funding Scheme—supporting translational research in hearing loss and tinnitus.

TRIH Partnerships—a platform by which industry can enter hearing research in a low-risk way.

TRIH Research Hub—a service to assist companies in easily identifying partners (academic groups & contract research organisations) to carry out specific tests or assays.

Clinical Trials Database—a means of engaging patient support for, and participation in, clinical trials for hearing loss and tinnitus globally.

Further information available here: [accessed 31 August 2011].

Case Study 4—Translation Awards

The Wellcome Trust provides translation awards with the primary purpose of securing a healthcare benefit from the development of new technologies/products. Projects must address an unmet need in healthcare or in applied medical research, offer a potential new solution, and have a realistic expectation that the innovation will be developed further by the market. However, given the uncertainty inherent in early stage research, awards are made with no expectation of a financial return.7

11. Charities can de-risk investments by providing vital funding for early stage research. Charities of all sizes can work constructively with private investors to de-risk their investment and release greater investment in research.

Case Study 5—Exploitation—Seeing an Opportunity—Yorkshire Cancer Research

Approximately six years ago, trustees of Yorkshire Cancer Research developed a new funding stream to allow them to fund a spin-out company founded between one of the charity’s programme award holders and his university. They were subsequently able to support a researcher with a promising translational project to enter into negotiations with the company, developing non-disclosure agreements to allow these to go ahead. The researcher is now applying for a Commercial Development Award from Yorkshire Cancer Research to undertake proof of concept research. If successful, the company, with Yorkshire Cancer Research’s support, will be interested in a much larger scale plan for commercialisation. Without the charity’s input the connection between the researcher and the spin-out company is unlikely to have been made.

Case Study 6—Cancer Research UK Clinical Development Partnerships

Cancer Research UK—clinical development partnerships ensure oncology agents that might not be worth industry investing in can come to market.

“Our clinical development partnerships serve this function, by taking molecules which have been shelved for strategic reasons within pharma or biotech into drug development in Cancer Research UK, based on an option arrangements whereby we move it forward, then afterwards the company we’re dealing with gets to look at the data and, if they like what they see, take it back. But if they don’t, then we get to take it forward.”

Keith Blundy, CEO of Cancer Research Technology.8

12. Charities provide a patient focus—improving design of research studies, recruitment of patients and uptake of commercial products, combating market uncertainty.

Case Study 7—UK DUETS

The UK Database of Uncertainties about the Effects of Treatments (UK DUETS) works with patients, carers and clinicians to identify where more research needs to be done into the effects of treatments—in effect flagging up opportunities for further research to improve treatments, some of which could entail small changes which make a dramatic difference. A number of medical research charities have taken part in this process, with the “top 10 questions” becoming an integral part of their research funding strategy.

Further information available here: [accessed 31 August 2011]

Case Study 8—Arthritis Research UK User Committee

Arthritis Research UK has developed a Stakeholders Research Strategy Review (USER) committee with the remit to provide a user perspective on research that the charity may fund. USER comprises front-line healthcare professionals, who are not research-active, and informed lay members. USER members consider: strategic importance; potential of the research to lead to clinical benefit; issues of practicality. USER provides a brief report which is circulated to the relevant funding committee to inform the peer review process. These views ensure that the best science with the best chance of practical uptake is funded.9

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

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

13. Commercialising life sciences research takes a long time and there is a high failure rate which can be a disincentive for many investors. The development of a new drug or therapy, including gaining regulatory approval and optimising complex manufacturing processes, can take 10–15 years before it is ready to go on the market and become available to treat patients. Investment is needed throughout this process. However, after this time period, this investment does offer significant returns.

“For each pound invested by the taxpayer or charity donor into cardiovascular disease and mental health research, a stream of benefits is produced equivalent to earning 39 pence and 37 pence respectively each year ‘in perpetuity’”.10

14. Creating a system to incentivise investment up to this point and share the risk is necessary if we are to offer an attractive proposition for investors.

15. There are difficulties specific to commercialising life sciences research:


Market certainty.

Developing research capacity and supporting skilled individuals to engage with translation.


16. Clinical trials are a vital stage in the development of effective new treatments for patients. There is currently a lack of proportionality in the regulation of clinical trials and considerable administrative burden which leads to increased costs and delays and is hampering the conduct of clinical trials in the UK.11 The UK’s share of patient enrolment declined from 2004 to 2008 from 2% to 1.4% of both phase II and phase III trials. There is concern that if this trend continues and less trials are based in the UK, our capacity and expertise to conduct these trials will erode.12

17. Research must often take place across multiple NHS sites. The process for obtaining research permissions across multiple sites can introduce delays and duplication.

Case Study 9—Time Taken to Navigate Complex Research Approval Processes

A recent analysis from Cancer Research UK showed that after its funding for a study has been agreed, it now takes an average of 621 days to recruit the first patient.13

18. Access to patient data offers a considerable resource for health research and is valuable to the commercialisation of research. However further work is needed to create a safe and secure system which simplifies access to data, ensures that patients can be offered relevant opportunities to be involved in research. AMRC polling found that there is considerable public appetite for these opportunities to be made available:

72% would like to be offered opportunities to be involved in trials of new medicines or treatments if they have a health condition that affects their day-to-day life.

80% would like to consider allowing a researcher confidential access to their medical records.

88% would be happy to be asked to talk to researchers about their family history or give a sample of their blood to be tested in a laboratory.

19. Despite ongoing work to improve recruitment including the creation of the UK Clinical Trials Gateway14 which allows people to search for clinical trials of interest to them and greater links with patient networks, there can still be considerable delays in recruiting patients to clinical trials. UKCRC funded work exploring patient and GP understanding and acceptance of the use of patient data in research highlighted significant barriers at GP level to facilitating data-sharing.15

20. Greater recognition of the value of patient and public involvement in research is needed. This will require support for health professionals to engage with patients effectively about research projects.

Market Certainty

21. To attract investment into commercialisation of research there needs to be an end market. Our members have expressed frustration with the products of their research both failing to receive NICE approval or receiving NICE approval and subsequently not being adopted throughout the NHS. Strict commissioning guidelines are needed to ensure approved innovations are rapidly taken up across the NHS.

Case Study 10—NICE Decision to Not Approve Abiraterone

In February 2012, NICE ruled that Abiraterone would not be funded on the NHS.. Abiraterone, a drug that blocks the production of male hormones in all tissues has proven successful in reducing symptoms and increasing the quality of life of men with advanced prostate cancer, but has been turned down by NICE on the grounds of cost. Abiraterone has been one of the most requested treatments from the Cancer Drugs Fund since it became available in the UK, with over 500 men receiving the drug. The Cancer Drugs Fund is only available in England and is set to expire in 2014.

The drug was developed with at the Institute of Cancer Research, following extensive support of charities including Cancer Research UK. It has been approved for use in the USA, but will not be available to men in the UK.16

22. It is worrying that promising therapies with no clear market value may not be commercialised.

Case Study 11—Difficulties Securing Funding for Off-Patent Drug Commercialisation

Research funded by Breast Cancer Campaign and conducted in mice found that a regimented six week course of two established anti-cancer drugs was effective in reducing breast cancer growth and secondary tumours in bone. However, as a result of one of the drugs being out of patent and the other reaching the end of its patent, the researchers were unable to secure funding for a full clinical trial to demonstrate efficacy in patients. There has been no further work conducted on this potential new treatment.

23. The advent of stratified medicine, which could lead to the development of treatments for smaller sub-groups of patients within a disease area, raises future challenges to uptake, including the need to develop systems to identify those that may benefit and develop local expertise to administer treatments.

Case Study 12—Cancer Research UK Investment in Stratified Medicine

Recognising the direction of cancer research and the potential of stratified medicine—treating different groups of patients differently based on the genetic nature of their disease—to improve treatment, Cancer Research UK are building a partnership between patients, the research community, the NHS, the pharmaceutical and diagnostics industry, and the government to make genetic testing routine practice and link this to research, creating a national database of tumour genetic information, treatments and outcomes as a research resource to help deliver better treatments in the future.

Developing research capacity and supporting skilled individuals to engage with translation

Universities must be proactive to attract industry investment, developing models for early collaboration. Recognising that many researchers do not consider commercialisation opportunities, charities are already building relationships with researchers and university technology transfer offices to exploit opportunities for commercialisation. These models of best practice could be applied across the UK.

Case Study 13—Identifying Opportunities—Yorkshire Cancer Research

Yorkshire Cancer Research meets every year with each of its programme award holders to discuss patient benefit, IP and commercial issues. The meetings involve its research liaison officer, commercial and clinical development officer, and an experienced life-sciences patent agent (who provides this assistance for free). University technology transfer representatives sometimes attend or the charity communicates any positive outcomes it discovers to the university. This allows the charity to put its own expert slant onto the opportunity from the outset.

There isn’t a set format for the meetings. Some programme award holders will involve some or all members of their teams and the charity is happy to encourage this approach. It fosters good relationships with all programme team members and lets them know Yorkshire Cancer Research is interested in the commercialisation agenda. Without exception, the charity has come away from the meetings knowing things it did not know before. In the majority of cases, there has been something in the way of a possible patent application to explore or a commercial issue to follow up.

Case Study 14—Three-Way Meetings between Principal Investigators, Technology Transfer Office and Charity—Breakthrough Breast Cancer

As Breakthrough Breast Cancer jointly owns IP arising from its funded research, it needs to be proactive and keep track of new and potential opportunities arising from research. To do this, it has established three-way meetings where representatives from Breakthrough Breast Cancer’s research management directorate meet with the principal investigators and the Institute of Cancer Research’s enterprise unit. The meetings take place twice a year and are used to update and inform all parties on all aspects of IP arising from Breakthrough Breast Cancer-funded laboratories.

The IP portfolio is discussed, including patents, licensing agreements, exclusivity rights and research that may have future commercial value. Decisions are made on all aspects of IP management from exploitation through to allowing patents to lapse. The principal investigators and charity benefit from the insight and expertise of the enterprise unit and the process allows the enterprise unit and charity to keep track of potential new opportunities raised by the principal investigators.

What, if any, examples are there of UK-based research having to be transferred outside the UK for commercialisation? Why did this occur?

25. AMRC members’ priority is to fund high quality research to develop new treatments for patients. The majority of this funding is invested within the UK, however in some cases, charities choose to fund abroad. In 2011–12, 6.1% of AMRC members research funding was invested outside the UK. This is a slight increase on the previous year where 5.4% was invested internationally. However there is no clear long-term trend.17 Interestingly, alongside this slight increase in international funding, AMRC member spending on UK research programs is down slightly on the year before. This is in contrast to a long-term trend of year on year increase.

26. This may be because research capacity is not present in the UK.

Case Study 15—Action on Hearing Loss Funding Outside the UK

Action on Hearing Loss funds £1 million of hearing research each year but a lack of hearing research capacity in the UK means that they are often forced to make investments outside the UK. For the period 2009–10 to 2011–12, 44% of their research budget has been spent overseas.

27. There are also concerns that delays negotiating UK research regulation and poor levels of patient recruitment may drive research abroad, which over time may erode our capacity and expertise to conduct research trials.18

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

28. The Technology Strategy Board has a strong business focus which contributes a valuable diversity of approaches to research commercialisation in the UK. The Board’s remit is broad but it has limited engagement with medical research charities. The creation of a Catapult Centre focused on cell therapy is valuable but to play an effective role in supporting UK-based commercialisation, this should provide enabling infrastructure to underpin investment. There are valuable opportunities to involve third sector investors focused on developing research capacity in their disease area.

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

29. The recognition of healthcare and the life sciences as a potential growth area for the UK has brought multiple initiatives.

30. Plan for Growth

The Plan for Growth published in March 201119 heralded work to streamline the regulation of health research and establish a Health Research Authority. This work is welcome but it is early to evaluate the impact on commercialisation. There should be ongoing evaluation of the impact of these measures with the power to recommend further action if needed.

31. Strategy for UK Life Sciences

The Strategy for UK Life Sciences20 and recommendations to improve the uptake on innovations within the NHS, Innovation, Health and Wealth: Accelerating adoption and diffusion in the NHS21 launched on 5 December 2012 outline further steps to support the commercialisation of health research in the UK.

32. R&D tax credits

R&D tax credits and support targeted at small and medium enterprises (SMEs) is welcome to enable these investors to enter UK public markets. These investors are able to enter into long-term commercial collaborations with charities and exploit market opportunities.

33. Biomedical catalyst scheme

Universities and small or medium enterprises (SMEs) will be able to apply to this £180 million investment in a Biomedical Catalyst scheme to support the development of promising early-stage drugs into new treatments. These applicants should be supported to work with charities who can bring expertise and insights into opportunities that could valuably be targeted.

34. Early Access Scheme

The plan to consult on an Early Access Scheme to identify where new treatments could potentially be given conditional authorisation, have their assessment accelerated or be licensed early to speed commercialisation is welcome. The government should consider other novel approaches of early adoption and living trials which may be more effective in accelerating commercialisation. This could be explored by the Life Sciences Advisory Board and will need the close engagement of patient communities.

35. Life sciences advisory board

The new Life Sciences Advisory Board comprising of representatives from industry, academia, NHS, MRC, TSB, NIHR and government departments and led by two expert Life Sciences Champions, Professor Sir John Bell and Chris Brinsmead, alongside the appointment of a Life Sciences Adviser to the Minister for Science and Universities, George Freeman MP, is welcome. The third sector offers valuable expertise in developing innovative funding strategies focused around patient need. The Life Sciences Advisory Board membership should reflect this.

36. Safe and secure access to patient data for research

The steps announced to develop a safe and secure system which opens up patient data for research and ensures patients are offered opportunities to be involved in research relevant to them are valuable. As the largest health system in the world, the data resource the NHS can offer researchers is considerable.

37. It is important that this system is developed with public confidence. Raising public awareness of the value of their data and providing detail of how it will be used safely and securely by all research funders is central to developing a system that has public support and is attractive for investors in research.

38. Government should consider the impacts on the use of patient data for research when engaging with the revision of the Data Protection framework currently underway in Europe.

39. A research-friendly NHS

Key to delivering the Strategy for UK Life Sciences is a research-friendly NHS—a National Health and Research Service which welcomes and supports research and will quickly adopt new innovations when commercialised. 97% of the public believe that it is important the NHS should support research into new treatments22. As the new bodies proposed in the Health & Social Care Bill are established, the NHS should work closely with the life sciences sector including medical research charities and learn from successful initiatives pioneered by the NIHR clinical research networks to support research, commercialisation and uptake of new innovations.

40. Investing in a skilled workforce for the future

To be an attractive location to commercialise research, the UK needs individuals with the necessary skills and expertise. Continued support for the development of STEM skills and to foster research capacity is vital. The government should recognise the international nature of science and industry and ensure our immigration laws facilitate the movement of skilled individuals in and out and of the UK, sharing expertise globally to remain competitive.

Should the UK seek to encourage more private equity investment (including venture capital and angel investment) into science and engineering sectors and if so, how can this be achieved?

What other types of investment or support should the government develop?

41. A stable funding environment requires a broad range of investors with different risk profiles. This includes private equity investment.

42. Charities of all sizes give their funding more reach and maximise the potential of their research by collaborating with other not-for-profit organisations, government departments, research councils and the pharmaceutical industry.23 This benefits all partners, catalysing novel research funding partnerships in areas of need and reducing risks for individual investors.

43. As outlined in this response, charities play an important role in creating innovation-pull and identifying markets. As such they are a unique asset to the UK research environment. To capitalise on this, the government should focus on ensuring the infrastructure and supply-side is available to support the development of innovative partnerships between diversely different funders. This includes developing clear pathways to take up innovations providing market certainty for investors, focusing funding to increase research capacity in key areas and encouraging academic engagement in commercialisation including sharing models of best practice.

February 2012

1 £1,189 million including capital expenditure—Data from AMRC research expenditure database, collected 2011-12.

2 Charities Aid Foundation and NCVO, UK Giving 2011, 2012
[accessed 17 February 2012]

3 Data from AMRC research expenditure database. [accessed 17 February 2012].

4 Cancer Research UK, Exploring the Interdependency between Public and Charitable Medical research, April 2011 [accessed 22 September 2011]

5 Alzheimer’s Research Trust, Forward Together: Complementarity of public and charitable research with respect to private research spending, 2009 [accessed 22 September 2011]

6 The Ministerial Group on Dementia Research – headline report,
[accessed 17 February 2012]
Defeating Dementia 2012,
Dementia_-_Building_capacity_to_capitalise_on_the_UKs_research_strengths.pdf [accessed 17 February 2012]

7 [accessed 23 September 2011].

8 Dow Jones Newswires, 08:35 GMT DJ INTERVIEW: UK Cancer Research Technology Offers Unique R&D Access, 19 Sep 2011.

9 [accessed 31 August 2011].

10 Medical Research: What's it worth? UK Evaluation Forum; 2008.

11 Revision of the EU Clinical Trials Directive: A joint statement from non-commercial research organisations in the UK, 2011 [accessed 17 February 2012].

12 Nesta, All together now: Improving cross-sector collaboration in the UK biomedical industry, 2011 [accessed 17 February 2012].

13 Average time from 25 studies approved by Cancer Research UK’s Clinical Trials Awards and Advisory Committee during the period November 2006 to July 2007.

14 [accessed 17 February 2012].

15 UKCRC Public Awareness Sub-Group on Patient Data, Attitudes and awareness amongst general practitioners (GPs) and patients about the use of patient data in research, 2010.


17 Data from AMRC research expenditure database, collected 2011-12

18 Nesta, All together now: Improving cross-sector collaboration in the UK biomedical industry, 2011 [accessed 17 February 2012].

19 [accessed 17 February 2012].

20 [accessed 17 February 2012].

21 [accessed 17 February 2012].

22 AMRC, Breast Cancer Campaign, British Heart Foundation MORI poll 2011 | [accessed 17 February 2010].

23 AMRC, Ways & means: How to make research collaboration work, 2010 [accessed 22 September 2011].

Prepared 11th March 2013