United Kingdom embraces new paradigm of patient-oriented research

Priorities established by patients, carers and clinicians within the Sight Loss and Vision Priority Setting Partnership have become reference points for research projects and research funding bodies in the United Kingdom, according to monitoring reviews conducted by Fight for Sight at 1 year after completion of the project.

Since the results were published, Fight for Sight has awarded more than £3 million to 46 projects that directly address priorities identified in 10 of the 12 eye condition categories.

“Other funders, including the government, are looking at the priorities when they are making funding decisions. We are delighted to see that researchers are now using those priorities when applying for grants,” Michele Acton, chief executive at Fight for Sight, said.

Michael Bowen, director of research at the College of Optometrists, confirmed that research proposals are now looked at for how they relate to the Sight Loss and Vision Priority Setting Partnership (SLV-PSP).

“It is starting to settle in and become a guiding framework for all our research projects,” he said.

Image: Acton M

Administered by Fight for Sight, the U.K.’s main eye research charity, and led by Fight for Sight and the College of Optometrists, the SLV-PSP was carried out with support from the College of Optometrists, the Royal College of Ophthalmologists, the NIHR Moorfields Biomedical Research Centre, the Royal National Institute of Blind People, Vision 2020 U.K., U.K. Vision Strategy and the Cochrane Eyes and Vision Group.

It was a unique prioritization exercise aimed at ranking the top 10 unanswered questions for each of the major causes of vision loss.

“[It was] the end result of a long-standing attempt to establish a set of research priorities within the context of the Vision 2020 U.K. initiative,” Richard Wormald, MD, consultant at Moorfields Eye Hospital and coordinating editor of the Cochrane Eyes and Vision Group, said.

The SLV-PSP introduced the new paradigm that priorities for research and research grants were established by and represent the needs and views of the people who are potentially the beneficiaries of research, he explained.

The methodology and framework were provided by the James Lind Alliance, a nonprofit initiative supported by the National Institute for Health Research that brings together patients, carers and clinicians to identify and prioritize health care-related research questions.

A large national survey was launched to collect unanswered questions about the prevention, diagnosis and treatment of sight loss and eye conditions. Through a series of consecutive prioritization workshops, the 4,461 submitted questions — regarding more than 100 different eye diseases and conditions — were first pared down to the 686 unanswered questions that were unique and within scope. These were then analyzed, shortlisted and categorized into 12 disease-specific groups until the top 10 questions for each group were identified.

According to Acton, this was the largest and most ambitious priority-setting partnership ever undertaken within the James Lind Alliance framework.

“Not only treatment was involved, and not just one eye disease, but also prevention and diagnosis and the whole range of vision-threatening diseases and conditions,” she said.

Prioritized research questions

Many of the questions raised were not new or surprising, but what determined the strength of the argument was the consensus of all the parties involved.

“The project brought organizations and individuals together. It merged the process for generating priorities. Previously many research priorities were set by researchers and clinicians, whereas now we have an integrated research party, which is a fantastic achievement,” Fiona Rowe, PhD, senior lecturer in orthoptics at the University of Liverpool and director of research for the British and Irish Orthoptic Society, said.

According to Wormald, many of the issues that are obvious as research priorities often do not get to be the subject of research because the choices “are driven by a whole set of other types of agendas that have nothing to do with the needs of the patients and public, but are the interest of specialists and pharmaceutical companies.”

For instance, one of the issues many patients raised in relation to glaucoma therapy was the need for easier ways to administer eye drops.

“A bit of lateral thinking because nobody has thought much about it except putting in an eye drop, and now people are considering alternatives as we know it matters to people,” Wormald said.

Also ranked high among the priorities was the question: “Why is glaucoma more aggressive in people of certain ethnic groups, such as those of West African origin?”

“Though quite apparent, it has never been properly addressed because research is so often market-driven, and there is little interest in solving the problems of the poorest areas of the world,” he said.

In the refractive error and ocular motility section, understanding the factors that influence the development of refractive error emerged as the top priority.

“Myopia has rapidly increased to concerning levels globally. Understanding the factors that are driving these changes in order to control and manage them is a central issue and a major concern in our profession. As in all sections, the interest in causative factors was very high amongst patients,” Bowen said.

“This justifies continuation of research into etiology,” Wormald said.

All questions were matched with answers available in the literature, and only the questions that could not be answered by evidence presented in a systematic review were included in the prioritization process. Bowen explained that the James Lind Alliance protocol uses a high standard for what counts as an evidence-based answer to a given question.

“When you look thorough the priorities, it is not that research has never been done, but rather that no conclusive evidence has been produced,” he said.

The perspective of patients

The key strength of the process is that it has involved patients and carers. Their views were given equal merit to those of professionals, Rowe said.

“Those with direct experience of conditions will now be able to influence the research agenda,” Acton said.

Tina Roberts came into contact with Fight for Sight when her 10-year-old daughter, Rose, was diagnosed with Stargardt’s 3 years ago.

“When Fight for Sight offered to me the opportunity to join the workshop on inherited retinal diseases, I did not realize how big and how important this was going to become. It was a very broad spectrum group of professionals, parents and patients with a lot to learn from each other. Knowledge is power, and I hope the knowledge gained through the partnership will be used to change lives,” she said.

Through her work within the SLV-PSP, Roberts learned what could be realistically expected, addressed and advocated for the disease.

“At first many of us thought that eye transplant might be an option, and this was the top of my list. Hearing professionals talking, I realized that this is still far away, but stem cells and gene therapy are closer, and it is worth putting money in resources that are closer,” she said.

On the other hand, she became conscious of the importance of the point of view and advocacy of the patients and their families.

“With the support from patients as well as from the medical profession, fundraising associations can be stronger,” she said.

As Wormald said, “The perspective of patients and carers is a powerful fundraising argument.”

Patient-oriented research, fundraising

Lack of funding has been a long-standing problem of research in eye care, Rowe said.

“This becomes really obvious when looking at the proportion of funding attracted by other medical sectors, such as cancer or cardiovascular care. And yet vision is one of our five senses, and loss of vision has a great impact on a person’s life,” she said.

The hope is that the results of such a large partnership will attract attention, raise awareness and eventually result in more investments in eye research.

“Grants are more confidently given when applicants can say with some authority that a certain question matters to patients,” Wormald said.

“The key objective is that funding bodies for eye and vision research make funding decisions based on these priorities. This will ensure that eye research funding is being targeted to what matters more to people who have experience in eye conditions. We are already seeing this happen,” Acton said.

Fight for Sight recently launched the Vision Impact Survey to monitor the effect of the SLV-PSP on current research and, eventually, on people.

“Our aim is to find out how closely new clinical research trials, tests and treatments match the SLV-PSP priorities by surveying SLV-PSP participants annually,” Acton said.

Rowe hopes that research and clinical experience based on SLV-PSP objectives will be published by individual specialists or groups of specialists and be released to the specific audiences so they are aware of the results.

“Hopefully, prioritized questions for research that’s relevant to patients and public will generate the good quality evidence we need to support the decisions of policymakers,” Wormald said. “In the longer term, we should also be able to see to what extent the research driven by our agenda has produced changes in clinical practice.”

Bowen sees the SLV-PSP as “a great example of something the U.K. does quietly very well.”

“I am not aware of any similar system to the James Lind Alliance system, based on such a strong involvement of clinicians, patients and public in the process,” he said.

“Although it has been done from a U.K. perspective, we have set priorities that are of global interest and certainly the framework , the model, the process through which we have achieved this result could be an example for other parts of the world,” he said.

“We have had a number of approaches from people in other countries interested in undertaking a similar exercise,” Acton said. – by Michela Cimberle

• References:
• Rowe F, et al. BMJ Open. 2014;doi:10.1136/bmjopen-2014-004905.
• Sight Loss and Vision Priority Setting Partnership – Setting Priorities for Eye Research – Final report. fightforsight.org.uk/tl_files/Documents/Sight-Loss-PSP/SLV-PSP_Final_Report_WEB.pdf. Published October 2013.
• Tackling the unanswered questions in sight loss and vision research. fightforsight.org.uk/press-release/items/tackling-the-unanswered-questions-in-sight-loss-and-vision-research. Published Oct. 27, 2014.
• For more information:
• Michele Acton can be reached at Fight for Sight, 5th Floor, 9-13 Fenchurch Buildings, Fenchurch Street, London EC3M 5HR UK; +44-20-7264-3900; email: michele@fightforsight.org.uk.
• Michael Bowen can be reached at the College of Optometrists, 42 Craven St., London WC2N 5NG UK; +44-20-7766-4373; email: michael.bowen@college-optometrists.org.
• Tina Roberts can be reached at research@fightforsight.org.uk.
• Fiona Rowe, PhD, can be reached at Department of Health Services Research, Whelan Building, University of Liverpool, Brownlow Hill, Liverpool L69 3GB UK; +44-151-7944956; email: rowef@liverpool.ac.uk.
• Richard Wormald, MD, can be reached at Moorfields Eye Hospital, 162 City Road, London EC1V 2PD UK; +44-20-7253-3411; email: richard.wormald@gmail.com.

Disclosure: The sources have no relevant financial disclosures.

Are patient associations sufficiently involved in the decision-making processes affecting health care?

Patient associations increasingly involved but still under-heard

Recently there has been a lot of emphasis on patient involvement and patient-centered health care in the U.K. Several initiatives have been implemented within the National Health Service to consult and involve communities and patients in decisions about care. Shared decision making, “there is no decision about me without me,” is said to be the norm. As one of the largest charities in the U.K., the Royal National Institute of Blind People is involved with the NHS and the National Institute of Health and Care Excellence in all major consultations and takes part in all the round tables and focus groups in which health care policies are discussed. However, the feeling is that our opinions are assigned a somewhat lesser weight as compared with those of health care professionals, despite the fact that we have a wealth of knowledge from talking to patients every day. Health services are still a lot more clinically oriented than patient oriented. A lot is designed around what clinicians want and what is easy for clinicians to deliver. Our task, as patient associations, is to be there to keep reminding policymakers that patients need to be more involved and to constantly put patients back at the center of government plans and policies. We have to make sure that our pleas are noted on official records and that we are not there just to satisfy the formal need for including patients as a tick-box exercise. An additional problem with our specific disease area is that eye care never had the priority it deserves, and this is not only in the U.K. This is why it is vital that we work tirelessly to make sure that the voice of blind and partially sighted people is heard loud and clear and that we continue to influence national and local health policies.

Clara Eaglen, is a Policy Campaign Manager, the Royal National Institute of Blind People, London.

Patient associations have been drivers of key achievements

I would have to say no, organizations such as Prevent Blindness are not sufficiently involved in all such processes. However, we do play a role, and I think a valuable one. Because vision and eye health is a relatively underrepresented focus on the health care landscape of our country, patient associations are necessary in order to continually bang the drum for adequate resources and attention to eye health. Our efforts over the years have been instrumental in guiding federal programming at both the Centers for Disease Control and Prevention and the Health Resources and Services Administration. In both cases, it has been our own advocacy efforts that caused these agencies to even address vision in the first place. Additionally, our organization was a key driver in establishing the Congressional Vision Caucus, which continues today as a venue to keep eye health issues in front of leading members of Congress and their staffs. Certainly, when talking about health care policy, there are louder voices out there (provider groups, pharmaceutical companies, insurance providers, etc.), yet no one can bring the same level of passion that an association speaking on behalf of the patient and the public can. So while I think we are far from being as involved in decision making related to health care as we should, I do think that patient associations continue to serve an essential role in the process.

Jeff Todd, is Chief Operating Officer, Prevent Blindness America, Chicago, U.S.A.