Science and TechnologyWritten evidence submitted by Action on Hearing Loss

About Us

Action on Hearing Loss is the new name for RNID. We are the UK’s largest membership charity supporting people who are deaf or hard of hearing. We’re experts in providing support for people with hearing loss and tinnitus and provide day-to-day care for people who are deaf and have additional needs. We supply communication services and training whilst offering practical advice to help people protect their hearing. We campaign to change public policy around hearing loss issues and support research into an eventual cure for hearing loss and tinnitus.

Introduction

1. Hearing loss is a major public health issue affecting over 10 million people in the UK—one in six of the population. As our population ages this number is set to grow and by 2031 there will be more than 14.5 million people with hearing loss in the UK. It is estimated that 10% of the population suffer from tinnitus. Hearing loss has high social costs and can lead to early departure from the workforce, isolation from social networks and an increased risk of depression and dementia. Hearing loss is also a significant economic burden; the cost of unemployment in the UK because of unaided hearing loss is estimated to be £13.5 billion and the market for hearing aids is estimated to be worth US$3.2 billion globally.1 , 2

2. At present there is no way to halt the progressive loss of hearing associated with age, nor treatments to lessen the damaging effects of noise. There is no way to restore natural hearing or silence tinnitus. For people with some residual hearing, amplification of sound via hearing aids or cochlear implants can be of benefit, but 97% of our members still experience difficulty hearing when using these.3

3. To respond to this urgent need, Action on Hearing Loss funds world-class biomedical research and training for scientists, and promotes hearing loss to the pharmaceutical and biotech industries and private investors as a commercially attractive area in which to invest. As a result, we have been responsible for major scientific discoveries, such as identifying biological pathways that can be targeted in preclinical models to prevent noise-induced hearing loss, and have positioned hearing loss as a tractable market for industry. For example, we recently helped Autifony, a new start-up focussed on developing drugs for hearing problems that target ion channels, to secure £10 million worth of venture funding and to form research collaborations with leading UK academic groups.

4. Despite this progress the development of biomedical cures and therapies for hearing loss and tinnitus is being held back by a significant underinvestment in hearing research and a lack of translational research. We warmly welcome the inquiry into bridging the “valley of death”. We identified this problem in 2010 and this has led to the creation of the Translational Research Initiative for Hearing (TRIH), which supports translational research with a new funding scheme and by facilitating investment partnerships. This is an excellent example of best practice.

Summary

5. We would like to see:

A change to the current reward system so that academic researchers and institutions are not penalised for pursuing translational research.

Incentives put in place to encourage investment in therapeutic areas (eg hearing loss) that have not traditionally been a focus of commercial activity.

A greater involvement of the voluntary sector in the commercialisation of research.

The Government follow through on its commitment to streamline the regulation of clinical research.

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

6. In the area of hearing research rapid progress is being made towards understanding the biological causes of hearing loss and developing strategies to both protect and restore hearing and silence tinnitus, but a lack of translational research is stalling the development and commercialisation of new treatments leading to lost opportunities to improve quality of life and generate wealth.

7. The commercialisation of research involves many stakeholders with different drivers, facing very different challenges. This makes it difficult to develop innovative ideas emerging from basic curiosity-driven research into potential new treatments, able to attract the commercial investment needed to bring them to market.

Academic Institutions

8. Academic research institutes are driven by the need to produce high quality and high impact research publications to compete for public and charitable funding. Research is also led by individual investigators driven by their own personal interests. This system serves basic research well and succeeds in generating innovative ideas and increasing our knowledge base. However, it tends not to support or encourage the type of research needed to translate the outcomes of basic research into potential treatments.

9. This type of research—translational research—tends not to generate high quality publications and is often not intellectually appealing to many academic investigators. This can deter individual investigators and academic institutes from investing their time and resources in translational research, making it difficult to develop a potential new treatment to a point where it can be taken forward by the commercial sector.

Commercial sector

10. Pharmaceutical and biotechnology companies are driven by the need to generate a profit for their shareholders and investors, so will only invest in research that has a good chance of leading to a profitable product. Many potential treatments will fail during preclinical development with only 1 in 50 drugs actually reaching clinical trials.4 Given this high rate of attrition it is not surprising that the commercial sector is becoming more reluctant to invest in translational research and demands strong evidence that a treatment is likely to be successful before investing—ideally, having reached phase II clinical trials. This has created a funding gap that is stalling the development of new treatments. Furthermore, the pharmaceutical sector prefers to invest in familiar tried and tested therapeutic areas as this eases the passage of a potential new medicine through the regulatory pathway to market; lack of experience in hearing loss as a therapeutic area thus presents another specific barrier to developing treatments for this condition.

Overcoming the difficulties of commercialising research

11. A number of funders (eg MRC and Wellcome Trust) have established funding schemes to support translational research and last year as part of our Translational Research Initiative for Hearing (TRIH) we launched our own translational research funding scheme dedicated to hearing loss. But these incentives are not enough on their own; there also needs to be a culture change within the academic research community, so that our very best investigators want to pursue translational research and are rewarded for doing so. The Government needs to publicly recognise and showcase the work of translational research leaders to provide a clear incentive for others to pursue work in this area. We also recommend that changes are made to the current reward system so that institutes and the careers of investigators working on translational research projects are not penalised for doing so. Greater weight should be placed on patents filed, industrial partnerships formed and commercialisation activities when assessing the impact of an institute or individual.

12. To encourage commercial investment in translational research we need innovative ways that companies can share the risks outlined above. Action on Hearing Loss’s Translational Research Initiative for Hearing (TRIH) is an example of how the voluntary sector can help to dilute the risk which can deter potential investors. TRIH was set up to accelerate the development of therapeutics in hearing loss and tinnitus by bringing academics, industry, investors and patients together. It provides a platform from which industry can easily identify the most promising lines of research to invest in and the best research groups to work with. Through these partnerships academic groups benefit from the industrial partner’s focus and experience of commercialising research.5

13. Companies need further incentives to encourage the investment that is needed in translational research; such incentives could take the form of tax breaks for research and development activities such as those recently announced by the Chancellor6 and access to funding schemes designed specifically to support translational research such as the recently announced Biomedical Catalyst Fund.

14. Access to specialised equipment and expertise can often be costly, making it difficult for small companies lacking in-house capabilities to commercialise research. We welcome the Government’s plans to establish innovation centres (Catapult centres) that will give businesses access to generic technologies, but it is vital that centres focussed on the life sciences are prioritised.

15. Finally, the voluntary sector plays a significant role in funding medical research in the UK, investing £1.2 million in medical research in 2010–11.7 In common with the commercial sector, medical research charities make investment decisions based on their vision to see new treatments brought to market, but unlike the commercial sector are not driven by the need to return a profit. This allows research charities to play a unique and important role in supporting the earlier more high-risk stages of the commercialisation process. Charities are also in a strong position to identify areas where there is a high level of clinical need. We would welcome clarification from the Government as to whether the voluntary sector will have a role to play in the distribution of the Biomedical Catalyst Fund, announced as part of the Government’s Strategy for UK Life Sciences.

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

16. It is particularly difficult to commercialise research in areas were research capacity is limited. Hearing loss is one such case. It has not traditionally been recognised as a major public health issue, despite being the third most common chronic condition in older people after arthritis and high blood pressure. Significant steps have been taken over the last few years to bring hearing loss onto the mainstream public health agenda, but hearing research still attracts a disproportionately low amount of funding in relation to the level of clinical need.

17. In 2007–08, less than 1% of the collective spend on research by the Medical Research Council, Biotechnology and Biological Sciences Research Council and the UK’s medical research charities was directed at hearing research, despite hearing loss affecting more than one in six of the population. That is equivalent to only £1.34 spent on hearing research for every person with hearing loss. This figure compares with £14.21 spend on vision research and £21.31 spent on diabetes for every person affected, both also long-term conditions.8

18. Most hearing research is supported through competitive responsive-mode programmes. Competition for this funding is high and with a relatively small number of hearing researchers competing against larger numbers of scientists working in high-profile areas such as cancer, cardiovascular disease and neurological conditions, hearing research attracts a disproportionately low amount of funding relative to the scale of the problem. This limits the field’s ability to commercialise research.

19. We are calling for the Government to take urgent action now to build hearing research capacity to bring research spending in line with the current and projected size and impact of hearing loss.

20. Action on Hearing Loss offers a range of funding schemes to help build research capacity. Our summer studentship grant and our PhD studentship schemes aim to encourage the best students to become involved in hearing, deafness and tinnitus research in the UK and our TRIH Funding Scheme offers researchers an alternative means of obtaining funding to kick start proposals with high commercial potential.

21. Companies are often deterred from investing in areas where there is no existing therapeutic on the market. A lack of market intelligence, understanding of patient needs and uncertainties around what would be needed to gain approval to market a treatment and how that treatment would be adopted by healthcare providers all deter investment. With a large membership base, the capacity to build market intelligence and links to key stakeholders in the field of hearing loss, Action on Hearing Loss is in a unique position to be able to provide expert advice to commercial investors and reduce these risks. We are able to articulate the needs of our beneficiaries, highlight the commercial opportunities that exist for hearing and tinnitus therapeutics through our acclaimed series of market research reports and by participating in trade conferences. We also provide market intelligence to companies and investors and links to key opinion leaders, to help them better understand the hearing market and how their technology could be developed to address clinical need. Our example demonstrates how medical research charities with their unique market insight can play an important role in helping to commercialise research in areas that have not traditionally been a focus of commercial activity.

22. We would therefore like to see:

(a)Support to better enable voluntary organisations to share valuable market data and patient need with companies.

(b)Areas lacking research capacity but with large unmet clinical need to be prioritised in government initiatives to support translational research.

(c)Incentives for companies developing therapeutics for new indications. These could follow those currently given to companies under the US FDA Orphan Drug Act (1983) under which companies developing drugs for rare conditions benefit from extended patent life, tax incentives, research design support and regulatory fee waivers.

Case Study

Developing medicines for middle ear disorders

The first research team to benefit from our innovative Translational Research Initiative for Hearing (TRIH) is led by Professor Allen Ryan at the University of California, San Diego who are developing a new approach to treating middle ear conditions, such as glue ear in children.

Middle ear infections are extremely common in children, with 90% experiencing an infection by the age of two years. This can often lead to glue ear caused by chronic inflammation and a build-up of fluid in the middle ear. It causes pain and discomfort and, in some cases, damage to hearing. Infections are treated with oral antibiotics and glue ear by surgically fitting grommets (small ventilation tubes) in the ear drum. But it is unclear how effective these approaches are. What is needed is a safe and effective way of delivering medication to the middle ear without the need for young children to undergo surgery.

Professor Ryan’s group have discovered a series of small peptides that are actively transported across the ear drum and believe that they could be used to “carry” medication into the middle ear. This would revolutionise the treatment of middle ear conditions. We are funding a three year project to optimise the structure of the peptides to maximise the rate at which they cross the ear drum and to generate evidence to show that the peptides can indeed carry drugs across the ear drum. The research we will fund will de-risk and add value to their technology making it more commercially attractive to future investors.

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

23. Action on Hearing Loss funds £1 million of hearing research each year but a lack of research capacity in the UK means that we are often forced to make investments outside the UK. For the period 2009–10 to 2011–12, 44% of our research budget has been spent overseas. Only around 10% of funding proposals submitted to the Translational Research Initiative for Hearing’s Funding Scheme were from UK research groups and the first project to be supported by this initiative will be conducted at the University of California.

24. Hearing research that does take place in the UK can be undermined by an overly complex regulatory and governance environment. An Action on Hearing Loss-funded project, investigating genetic predisposition to hearing loss caused by a specific class of antibiotic often used to treat life threatening infections in premature babies, has been held up for over two years due to the complicated bureaucracy involved in conducting clinical research at multiple sites in the UK and the lack of support to help researchers navigate the regulatory process. It is essential that the new Health Research Regulatory Agency announced in last year’s budget delivers on its objective to streamline the regulation of clinical research.

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

25. In the field of hearing research the Government and Technology Strategy Board (TSB) initiatives to date have made very little impact on the commercialisation of research. This is in part due to the fact that the TSB is tasked with supporting business-led innovation across a broad range of technology sectors and yet initiatives tend to be very focussed on a particular challenge or opportunity. This limits the opportunities for companies working in very specific areas from fully benefiting from the work of the TSB. For example, of the Innovation Centres planned, only one (Cell Therapy Catapult) will support the development of new medical treatments.

26. We recommend that the TSB works more closely with the voluntary sector to identify neglected areas of clinical need and shape future priorities accordingly.

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

27. The measures announced in the Government’s Strategy for UK Life Sciences to provide financial incentives to encourage the commercialisation of research, to make it easier for patients to get involved in clinical research, to make NHS data more accessible for research purposes and to streamline regulation will all go a long way to improving the commercialisation of research.

28. In particular, the £180 million committed to a Biomedical Catalyst Fund to support translational research is welcomed, but it will be important to work with all stakeholders including medical research charities to ensure that these funds are targeted to where the need and opportunities are greatest.

29. The formation of an independent Life Science Advisory Board made up of representatives from industry, academia, NHS, Medical Research Council, Technology Strategy Board, National Institute for Health Research and Government departments is welcomed, but as a major funder of medical research it is important that the voluntary sector is also represented on this board.

30. As part of our TRIH initiative we developed a web-based clinical trials database to make it easier for patients to identify clinical trials they may like to take part in. The UK Clinical Trials Gateway planned to be re-launched in March by the NIHR will be a great resource, making it easier to recruit patients to clinical trials and engage patients with the research process.

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?

31. We strongly support the encouragement of more private equity investment into the life sciences sector, but as the development of medical treatments can take many years it must also be balanced with more long-term financing.

32. We believe that there is a role for the Government to incentivise private equity investors to invest in emerging or neglected areas of research. A central privately-backed fund could be created, from which all investors would be rewarded for any returns. This would help minimise the risk of investing in less well-established fields of research and would help to ensure a more consistent stream of funding for researchers. Charities are well placed to help assess disease burden and we would therefore expect the third sector to play a role in the distribution of any such funding.

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

33. It is vital that the Government provides investment and support for voluntary organisations which fund £1.2 million of medical research annually and are in a unique position to help bridge the “valley of death”. In particular, it is essential that the Government maintains its support of the Charity Research Support Fund provided to universities through the Higher Education Funding Council to England (HEFCE) to cover non-direct costs associated with conducting charity-supported research.

34. TRIH is an example of a successful voluntary sector initiative which helps to reduce the risk of investments and supports the development of new treatments. Initiatives such as this would benefit from Government support in the form of direct grants or co-funding through existing Government-funded translational research programmes.

February 2012

1 Hear-it AISBL (2006) Evaluation of social and economic costs of hearing impairment.

2 Goldman Sachs (2007) Hearing aids: sounding out industry growth.

3 Action on Hearing Loss (2010) Annual Survey of Members.

4 PhRMA (2009) Pharmaceutical Profile.

5 Please see www.trih.org/download/TRIH_Brochure.pdf for further information about TRIH.

6 HM Treasury (2011) Autumn Statement.

7 Association of Medical Research Charities (2011) Annual Review.

8 Action on Hearing Loss (2010) Analysis of Medical Research Funding into Hearing Loss in the UK.

Prepared 11th March 2013