public support for or recommendation of a particular cause or policy.


support for, argument for, arguing for, calling for, pushing for, pressing for; defence, espousal, espousing, approval, approving, endorsement, endorsing, recommendation, recommending, advising in favour, backing, supporting, favouring, promotion, promoting, championship, championing, sanctioning, acceptance;


Advocacy is a term used by patient groups all over the world who are working to raise awareness of a cause, often to influence those who develop legislation to implement positive changes that they believe will impact society in a positive way.

Retina International and its stakeholders advocate for the implementation of policies to promote research and development, the inclusion of the patient voice in all policies that affect them and to improve access to therapies that can improve their lives.

Advocacy comes in different forms, including self-advocacy, citizen advocacy and independent advocacy.

Advocacy is key to people with unmet medical needs and disabilities who wish to access a proper diagnosis and access to the medical expertise and interventions that will help them to better understand their condition and prognosis. Often this piece of ‘advocacy’ can be in engaging the local healthcare provider (HCP) and even your personal doctor.

People living with disabilities can often be in a situation where they are not getting access to their entitlements. In this case, a person may need to engage their medical professionals but also local counsellors to advocate for their needs. In this case a person can advocate locally for services that can help them in their day to day lives, but they can also work with peers to engage a specific community to work together at a local or national level to ensure that all those with the same needs for the same support or services can use their collective voice to increase the chances of success in achieving their goals.

Advocacy is also becoming much more effective at a super-national or global level to influence decision making that has a global impact. The advent of computer technology and communications platforms has made it much easier to work globally and this is a particular objective of our organization. Since Retina International was formed, we have witnessed huge strides made by the medical and scientific community in uncovering the genetic changes that can lead to degenerative vision loss. These efforts are beginning to bear fruit with the first ocular gene therapy approved and launched onto the market in 2017. However, this stellar progress would not have been possible without strong patient advocacy. Now that treatments are within reach for some conditions and on the horizon for others, patient advocacy needs to continue in order to ensure access to the appropriate testing and access to treatments for those who need them.

An empowered patient population that is well educated in the disease area as well as in systems of research and healthcare can result in effective patient advocates that can bring about change. Appropriate training ensures that patients have the confidence and knowledge required to bring their expertise to the table in discussions on health care, research and medicines development with scientists, clinicians, pharmaceutical companies and policymakers.

But how do you get started? What do you need to consider? Who do you talk to? And how do you make your voice heard? In the delivery of your own personal healthcare, in accessing rehabilitation services for your community, how do you access a genetic test, how do you advocate to enter an appropriate clinical trial and how do you ensure that you and others with the same needs access the emerging therapies that can have a positive impact on your life?

The first step in patient advocacy can be approaching relevant patient organisations in your area. Retina International’s member organisations are a great place to start

Understanding the various systems involved in biomedical research and healthcare can seem daunting. However, there are helpful education tools available online. These include the European Patients’ Academy (EUPATI) that has a wealth of resources freely available and runs a patient education course online; EURORDIS, the ‘voice of rare disease patients in Europe’, provides training for patients, in person and freely available online, and acts as a link between trained patients and significant organisations, such as the EMA, where patient advocates can effect change in many areas. Some countries are now beginning to provide patient education courses that are specific to their own country’s systems. Examples of these include the 6-month long IPPOSI Patient Education Program in Ireland and the EUPATI UK Mini training day courses.

This section of our toolkit aims to help you to understand the areas that the Retina community is concerned with and how we can work to ensure the views of this community are considered in the development of policy that affects us today and in the future.

‘Nothing about us without us.’

Questions to Ask Your Health Care Provider

  • Communicating clearly with your Eye Care Professional (ECP) is essential for both of you to understand one another and to ensure that you are getting the best advice relevant to your individual circumstances. Asking questions and understanding your ECP’s responses is essential to good communication.
  • It is sometimes recommended that you bring a family member with you too – two pairs of ears are better than one – particularly where the family member is acting as a caregiver. Caregivers may also need to ask for advice and the best person to ask is the ECP. Additionally, while caregivers will discuss their loved one’s care with the ECP, they seldom talk about their own health, which is equally important. Building a partnership with an ECP that addresses the health needs of the individual and their caregiver, as applicable, is crucial. Ideally the responsibility for the partnership is shared between the affected individual, their caregiver, the ECP and any other healthcare professionals.
  • When meeting with your ECP it is important to be prepared to get the most out of these appointments. Make a list of your most important concerns and problems. Issues you might want to discuss are changes in symptoms, medications or general health, specific help or concerns that the caregiver has, etc. Remember the ECP only sees a moment in time; make sure you let them know of any concerns that exist in the routine daily environment. Remember also to enlist the help from all of those involved in your care, including nursing staff and pharmacists. Other organisations, such as local patient organisations and support groups can help too.
  • Make sure your appointment with your ECP meets your needs

You may feel overwhelmed by the information you receive at your appointment, so it can be useful to make a list of questions beforehand to take with you. Here are some suggestions that might be helpful;

  • What is my diagnosis?
  • How will my vision be affected?

A doctor will not be able to give you an exact answer to this question, as everyone is different, and conditions can progress at different rates in different people. They will be able to tell you which parts of your vision are most likely to be affected, for example, your central vision or peripheral vision.

  • How will it affect my home and work life?

The doctor may be able to give you an example of how the condition will affect an everyday task such as reading or driving.

  • What is the short-term and long-term prognosis for my disease or condition?

Many conditions will cause your vision to change over time. A doctor will not be able to give you an exact prognosis or time line but may able to give you an idea of what can happen in the short and long term in most cases of your condition.

  • What caused the disease or condition?

Is your condition genetic or can it be affected by lifestyle or environmental factors.

  • Are there local agencies or organisations that can help me learn more about my condition and vision loss?

There may be an organisation or support group that deals specifically with your condition or may be able to give you more information.

  • Has my vision changed since my last visit?
  • Could medication or health supplements I take have any effect on my vision? If this is something you are unsure about, bring a list of any medications or supplements to your appointment and ask your doctor about them.

Public Patient Involvement

Patient and public involvement – commonly shortened to PPI – is a framework to give lay people an effective and active role in all forms of “healthcare research”.

When using the term ‘patient and public’ we include potential patients, carers and people who use healthcare services as well as people from organisations that represent people who use these services. Whilst all of us are actual, former or indeed potential users of healthcare services, there is an important distinction to be made between the perspectives of the patient/public and the perspectives of people who have a professional role in healthcare, be they clinicians, researchers, regulators, payers or policy makers.

The cornerstone of PPI is that research is being carried out ‘with’ or ‘by’ members of the public rather than ‘to’, ‘about’ or ‘for’ them. Having patients and the public involved as partners in research should lead to the development of treatments and services that better meet people’s needs and are more likely to be put into practice.

In this context, Retina International believe that PPI should be a cornerstone of all innovative programs in the retinal space. We recommend that “healthcare” is defined as widely as possible, to include medical care, public health, and social care, and “research” covers all stage of the product/service development lifecycle, including basic science, pre-clinical and clinical development, regulatory and reimbursement processes and service delivery.

The purpose of PPI must be to align research more closely with the public’s needs and thus make it less likely to fail, more effective, and more cost-effective.

Today patients and the public already take part in research, but usually as participants in a study, where researchers collect data about them and their health. PPI should create an active partnership between the patient/public and researchers, with the voice of the patient/public contributing throughout the research process.

PPI occurs when the patient/public work in partnership with researchers in setting priorities for research, in planning and managing research studies, as well as in summarizing, distributing, sharing, and putting results into practice.

PPI is an important step in ensuring that the real life experiences of patient/public are considered when decisions are being made about what research to do, what are the most important questions to be answered, how to design studies that patients are more likely to both take part in studies and stay involved in, what constitutes a good outcome, what clinical endpoints are relevant and what value should be placed on a particular treatment or service.

Measuring “value” is a particularly contentious issue where one may be talking about gradual deterioration and/or loss of vision over decades and the burden of all of the associated implications of vision loss. As these costs are typically not borne by public healthcare systems, quite often they are ignored in any economic cost-effectiveness analysis.

What is Patient Empowerment?

We hear the term ‘Empowered Patient’ the ‘Patient Advocate’ the ‘Involved Patient’ a lot these days. Here at Retina International (RI) a key objective is to educate our patients on emerging policies that may affect their health care in the future. Regardless of where we live in the world inequalities in access to diagnosis, care and treatment are a reality. By working together to think internationally and act nationally we can draw on each other’s experiences so that as individuals and as a collective we can work towards better outcomes for people living with retinal dystrophies be they genetically inherited or age-related.

We are all advocates for our future healthcare, as are the clinicians, allied health professionals and scientists who work with us at RI.
If you are new to the area of health advocacy and patient empowerment here are some explanations that we hope will define the terminology used.
We want to hear from you so if you have any questions let us know.
Many researchers place a high value on PPI. Hear what Prof Joseph Carroll of University of Milwaukee Eye Institute, USA thinks about PPI in research.

Recently, the EMPATHIE project (“Empowering Patients in the Management of Chronic Diseases”) developed a working definition of an empowered patient. This definition in fact combines both empowerment and involvement:

“An empowered patient has control over the management of their condition in daily life. They take action to improve the quality of their life and have the necessary knowledge, skills, attitudes and self-awareness to adjust their behaviour and to work in partnership with others where necessary, to achieve optimal wellbeing. Empowerment interventions aim to equip patients with the capacity to participate in decisions related to their condition to the extent that they wish to do so to become “co-managers” of their condition in partnership with health professionals; and to develop self-confidence, self-esteem and coping skills to manage the physical, emotional and social impacts of illness in everyday life.”

However, this definition focuses only on the individual patient and its scope is limited to managing life with a chronic condition, even though it does go beyond the purely medical context. It does not include the notion of “critical awareness” and patients’ active participation with the aim of changing the system. But it also does not address the role of patient organisations as representatives of the patient community in a wider sense whose role is to advocate for the patient perspective at policy level.

The Alma Ata declaration defined civic involvement in healthcare as both a right and a duty: The people have the right and duty to participate individually and collectively in the planning and implementation of their healthcare.” (Alma Ata Declaration Principle IV1978, WHO). “Meaningful patient involvement” as part of a global patient movement, RI along with other umbrella groups belive it is based on the premise that patients have a specific expertise derived from simply being patients, which is a valuable source of experienced knowledge. The patient’s perspective is not the same as the lay/consumer perspective. The patient’s knowledge is derived from living with a condition day to day and from being in frequent contact with the healthcare system.

In addition to being a right, patient involvement is recognised as having benefits for developing therapies and healthcare services in a way that has positive impact on patients and society. RI believes that meaningful patient involvement in health policy and programmes will lead to services that provide real value for patients, as well as reducing unnecessary waste.

Health Literacy

Health literacy is a key aspect of empowerment. Although empowerment involves much more than becoming an educated/informed patients the right information and resources are fundamental tools for empowerment. Health literacy however, is more than information. It is defined variously as: The cognitive and social skills which determine the motivation and ability of individuals to gain access to, understand and use information in ways which promote and maintain good health. (WHO, The ability to make sound health decisions in the context of everyday life at home, in the community, at the workplace, the healthcare system, the market place and the political arena.

From a patient’s perspective, the knowledge and competence gained through health literacy leads to the strength and empowerment needed to manage well a disease and its impacts on quality of life.

Health literacy starts with good, easily understandable information: information is a tool towards improved health literacy. To make genuinely informed decisions about health and treatment, it is vital that patients can access all the relevant information needed in an easily understandable format. The health literate patient is then able to process, appraise and apply the information to their own personal circumstances. This is usually defined as “functional” health literacy. More advanced levels of health literacy are interactive health literacy and critical health literacy. Patients’ information needs are diverse and vary according to age, socio economic status, gender, beliefs, preferences and coping strategies, and according to their general literacy, first language, skills and abilities. Needs often change during the patient’s journey, as does the “empowerment status” of the individual patient.

Clinical Guidelines and Care Pathways

A clinical guideline is a document guiding decisions and criteria regarding diagnosis, management, and treatment in specific areas of healthcare. They are based on an examination of current evidence within the paradigm of evidence-based medicine and usually include summarized consensus statements on best practice. Typically a guideline will be published by a global learned medical organisation, after consultation with a range of care providers in the area of interest.

A care pathway pulls together all of the disparate parts of a patient’s care associated with a particular condition, pulling together relevant elements of all of the associated clinical guidelines. (As an example a care pathway for IRD may include diagnosis, monitoring, therapeutic intervention and tertiary services across a range of medical, psychological and social professionals.) It is one of the main tools used to manage the coordination and standardisation of care processes across all settings. Their implementation reduces variability in clinical practice and improves outcomes. Care pathways can range in scope from simple medication utilization to a comprehensive treatment plan. Care pathways aim for greater standardization of treatment regimens and sequencing as well as improved outcomes, from both a quality of life and a clinical outcomes perspective.

Within the eye care setting there is a need to develop appropriate care pathways addressing the complexity of the challenges that face patients and their carers. The aim of the care pathway should be to focus on the patient’s overall journey, rather than the contribution of each specialty or caring function independently. They must all be shown to be working together for the benefit of the patient. This is particularly so given the nature of the decline in vision over years and decades and the co-morbidities associated with vision loss.

You can find an easy-to-download PDF entitled ‘Guide to Essential Care for IRDs’ here.

Investing in Support

Low vision impacts on every part of a person’s life. It is associated with falls, reduced capacity to carry out everyday activities, the need for residential care and is one of the strongest risk factors for functional status decline in community living adults. Evidence suggests that low vision services significantly reduce visual disability and are associated with positive patient outcomes. Furthermore, for the relatively small costs of low vision aids, there can be huge cost saving in terms of health and social care support.

Low vision services should enable people with loss of vision to regain or maintain as much independence and autonomy as possible, and can include rehabilitation, visual aids, emotional support and advice. Low vision services that aim to improve functional ability, quality of life and psychosocial status in those with visual impairment are required.

Vision Rehabilitation

Vision rehabilitation is one of supports that ensure you have the right information, training, skills and aids to transition to living with sight loss. Your local sight loss charity will have information on local supports. Please see the member charities of Retina International for a local sight loss charity near you

Visual rehabilitation can help you achieve your personal goals that can be identified by a support assessment. Vision rehabilitation support can be quite varied. It can include helping you understand your eye disease and what it means for you, understand the changes you may need to make in your life, how to continue looking after yourself and finding new ways to do tasks.

It can include ensuring you are safe inside your home and how to care for it, for example assessing mobility needs, reducing risk of falls and advice on appropriate equipment or mobility aids if needed.

Simple adjustments such as reviewing the lighting in your home can make helpful improvements. A low vision service can advise you on the use of aids such as magnifiers and specialist lighting.

Being able to get out and about is very important to maintain independence.

Vision rehabilitation can help with travelling confidently and safely and give advice on using public transport.

Vision rehabilitation support is not just for physical adjustments but also takes into account a person’s communication needs and how they keep in touch with others. This can be aided with help with reading, writing, talking books and newspapers, telling the time, using technology such as smartphones, tablets and speech software. Most smartphones or computers have accessible features built in while there are many packages available that enable everyone to use a computer.

VIAOPTA is a recently developed suite of mobile applications that have been developed to assist visually impaired people with their daily lives. These apps allow individuals to maintain their independence by assisting with daily activities, navigating your local region and recognising people and places using image analysis technology.

There are many options for support, however it is best not to assume the first solution will be right for you. We’re all different and have different needs.

A visual impairment does not preclude an individual from education or employment. Appropriate vision rehabilitation can ensure access to training, education and learning opportunities as well as advice about disability employment.

The NEI/NIH has useful videos, including this one on ‘How Can People With Low Vision Maintain Their Independence?’

And this NEI/NIH video describing ways that can help with vision loss on a day to day basis ‘What Can I Do if I Have Low Vision?’

With the appropriate support and guidance, it is possible to work through and surmount difficulties to find new and life-enhancing meaning and purpose in living.


In order to advocate for access to and improvement of low vision services data is required to unequivocally demonstrate the value that such services bring to the individual and wider society. Where practical and appropriate, Retina International recommends that data should be gathered from existing services and new services to demonstrate the important role that provision of low vision services play in the overall healthcare of the affected individual, their family and their community.

Access to Genetic Testing and Clinical Trials

Genetic testing is of utmost importance for many IRDs due to their genetic origin. Genetic testing can aid in diagnosis and, critically, it can determine if individuals have a specific mutation that may be treatable by specific gene therapies. Access to genetic testing varies widely across the world, with some areas not having access at all.

Retina International feels very strongly that patients need access to genetic testing in order to have the best chance of accessing treatment and advancing research. We are determined to provide information necessary for patients and patient groups to advocate for access to genetic testing.

You can learn more about how genetic testing works, and what it can do, at our toolkit ‘SENDING A RED ALERT!’ intended to inform those with rare eye diseases about genetic testing services:

Clinical trials look at new ways to prevent, detect, or treat disease. Clinical trials can study new drugs or devices or combinations, new ways of doing procedures, new ways of using existing drugs or devices and new ways of changing behaviours. The goal of clinical trials is to determine if new approaches to treatment, prevention, and behaviour are safe and effective.

With no effective treatments approved for the majority of IRDs, clinical trials currently provide the best opportunity for people with IRDs to receive a novel treatment that could potentially stop or slow down the progression of their disease. It is important to note however that clinical trials may not be suitable for all people. Speak to your doctor if you are interested in participating in a clinical trial, to learn about options for participation and potential risks and benefits.

You can hear retinal researcher Prof John Flannery give his views on the importance of genetic testing in this video ‘No gene, no gene therapy’.

For further details on clinical trials, visit This site provides the largest registry of clinical trials being conducted around the world, with tens of thousands of trials and locations in more than 200 countries. The site allows users to search by medical condition or other criteria for trials for many diseases and conditions. The site offers information about a trial’s purpose, who may participate, locations, and phone numbers for more details.

Access to Treatments

Access to treatments varies across the world. There is great disparity not only between countries but often also within countries. RI has 43 member organisations with each continent represented and so, RI strives to ensure access to treatments is available across all countries.

RI works to empower and educate patient organizations to ensure they have the best tools to advocate for access to treatments in their own countries.

Once a treatment has been approved by a regulatory body such as the FDA or EMA, it is up to the individual countries to assess whether they will recommend the treatment for use in their country. Many different factors are taken into account to make this decision. A Health Technology Assessment (HTA) can carried out by a government body to determine if the new treatment would be impactful in their country and economically possible. Patient organisations can advocate for their position by making submissions to the relevant bodies in their countries. This enables patients to give relevant information on what the introduction of the treatment would mean for patients

and how it would affect the patients, their health, their other treatments, their daily living, independence and contribution to society, their carers and their families.

Even when a treatment is recommended for reimbursement (introduced into the country), access may not be equitable within that country. For example, genetic testing is required to have a genetic diagnosis in order to access the correct gene therapy. However, genetic testing is not widely available and it may not be affordable or covered by health insurance.

Please see our RED ALERT Toolkit for more information on genetic testing for IRDs.

Another example is if the treatment itself is quite expensive and is not covered by a public health system, then only those who can afford the high cost will be able to access it. Therefore, while it is important to advocate for a new treatment to be introduced into a country, many other steps may be required before it can be delivered to all those who need it. Patient organisations can advocate at each step in the process.

Impacts and Costs of IRDs

Vision loss results in significant economic costs for the patients, their carers and the rest of society. While age-related visual impairments are increasing as the global population ages, inherited forms of vision loss are a significant contributor to economic and social costs of blindness. This is because they affect babies, young children and young adults and therefore can affect the entire life of a person. Vision loss affects not just the individual. It impacts the caregivers, family and community.

This can result in a significant cost burden. The quality of life and emotional distress of severely visually impaired people has been shown to be similar or worse than that of other serious chronic illnesses such as stroke, which translates to a high cost. In terms of economic burden on the health system, the overall cost of visual disorders in Australia, for example, was found to be seventh of all diseases, ahead of well-known diseases such as heart disease, diabetes, depression and stroke.

Determining the economic impact of visual impairment and relevant interventions is highly important as demand on health systems continue to increase. Therefore, understanding the broad range of direct and indirect costs and real effects on people is vital as almost all treatments aim to prevent or reduce these costs. They can be measured as an ‘incremental cost effectiveness ratio’ (ICER). As resources in health systems are finite and under increasing demand, a clear understanding of the economic impact of any disease is necessary for these finite resources to be prioritised to be most effective.

Costs can come under many different categories but can mainly fall into three: direct costs, indirect costs and intangible costs. Direct costs can be thought of as expenses directly related to an illness. These include medical costs, which are the cost of the resources needed to treat the illness, and non-medical costs which are the costs caused by the disease but are not the medical costs. An example of a non-medical direct cost would be supporting services, home care or travel expenses.

Indirect costs, however, are defined as ‘the value of lost output caused by reduced productivity due to illness or disability’. These include financial supports for income, accommodation, disability benefits; productivity losses such as loss of work, part-time work and absence from work for health reasons; years of life lost, and so-called ‘dead-weight losses’. In economics, dead-weight losses are due to market inefficiency but in relation to health issues, they can be described as excess financial burdens on society.

The burden of the illness on affected individuals, their caregivers and families is referred to as ‘intangible costs’. Intangible costs include loss of wellbeing and loss of quality of life and can be recorded using questionnaires completed by patients and their caregivers. They can be difficult to quantify and so are not usually given a monetary value. They can be described by disability adjusted life years (DALYs), a summary measure used to give an indication of overall burden of disease. The loss of the equivalent of one year of full health is represented by one DALY. The burden of diseases that cause premature death, but little disability, can be compared to the burden of diseases that do not cause death but do cause disability, such as blindness.

The greatest costs due to vision loss are caused by productivity losses in patients as well as their carers. Recurrent hospitalisations and the use of medical or supportive services contributed to significant economic costs. Surprisingly, a significant amount of the directs costs are not due to eye-related medical care, but are related to falls, a reduced ability to manage other illnesses and psychological issues related to vision loss. While intangible effects are difficult to quantify, the small number of studies that have researched them showed a large economic impact due to effects such as loss of independence, quality of life and excess morbidity. All types of costs that arise due to vision impairment need to be taken into account in economic evaluations of the impact of vision loss

and of interventions to alleviate these costs both on the individual and on society at large.

To date, most studies on the impacts and costs of blindness have encompassed all forms of blindness, including those related to aging. The specific impacts and costs of inherited retinal conditions, such as LCA, that can have effects from birth are not known. This is a significant gap in the information required to adequately address the needs of individuals affected by IRDs at social and healthcare levels currently. With new treatments coming onto the market that are considered to be expensive, the complete picture of the extent of medical, economic and social effects of IRDs is vital to ensure appropriate access.