Eleanor Wheeler

Patient advocates: vital voices in shaping health policy and care

5 September 2024

Patient advocacy can wield significant power to inform and influence health policy. But more needs to be done to make sure patients’ experiences are not overlooked.

The World Health Organization constitution states that ‘informed opinion and active co-operation on the part of the public are of the utmost importance’ in improving population health. It might therefore seem obvious that insights from people affected by a condition should be viewed as integral to the decisions that govern how healthcare is provided and health is improved. Yet their voices and perspectives are often left out of the evidence, decision frameworks, governance and processes that shape our health systems and the policies that govern them.

Clinical trials, for example, are often not designed with the needs of their target populations in mind. The result is that people who participate in them often do not fully represent the populations affected by a condition, and the outcomes measured are not the ones that really matter to patients. Similarly, at the policy level, lack of engagement in regulatory decisions means that the patient perspective is often omitted from important decisions, which may contribute to disparities in access or uptake of meaningful innovations.

Patient organisations act as an important channel to bring patients’ voices into discussions informing health policy.

What is patient advocacy and why is it important?

Patient advocates might be people with lived experience of a disease/condition themselves, or carers. They could also be members of the public who represent the voices of people affected by a disease. They may self-organise as part of a patient advocacy group or join an established organisation.

Patient organisations are core sources of information and support to those affected by a condition – and this role is essential for many people and their families. Such organisations help them obtain accessible information, engage with others with the same condition, and discover services, often filling a huge gap in the kind of support provided by the health system itself. But their role extends far beyond this; these organisations also act as an important channel to bring patients’ voices into discussions informing health policy.

As illustrated by the phrase ‘nothing about us, without us’, decisions about people should not be made without suitable representation of their views and perspectives. This requires a rethink of how patients’ voices can be integrated into the way we conduct and design clinical research and health services, decide which innovations should be prioritised and funded, and determine which overarching policies should govern our health systems.

How patient engagement adds value: It enhances the diversity and representativeness of data collected for research or other purposes, adding the voices of underserved communities. Sharing personal stories conveys the human impact of a condition. It ensures that patient perspectives are reflected in research, such as informing recruitment strategies, trial design and the outcomes to be measured. It highlights gaps in clinical services and drives person-centred care approaches.

What role does patient advocacy play in shaping policy?

Strong patient advocacy can enable policy – and the evidence that informs it – to be viewed through the lens of lived experience, drawing on expertise from populations for whom the policy has a direct impact. When done effectively, this advocacy work can change the nature of discussions about the disease or condition.

Advocacy can take many forms but, simply put, it can be defined as ‘intentional activities initiated to influence the policymaking process.’ These activities can include building relationships with decision-makers or formal institutions (states, courts, political parties etc); acting as a contributing stakeholder to policy decisions in the form of consultations or working groups; or having a formal role on key decision-making bodies. For example, the National Institute for Health and Care Excellence in the UK has an explicit mandate to include patients and members of the public in its appraisal process to ensure that their experiences are considered alongside population-based evidence on both clinical and cost effectiveness of health interventions such as medicines.

Patient stories are also a critical complement to scientific evidence, and are particularly powerful for illustrating the impact of a health condition or disease on people’s daily lives, ability to work, and mental and physical wellbeing.

Patient advocacy has driven tangible policy change

Patient advocacy can shape significant policy changes and dramatically impact people’s lives and wider society. Two examples of this are AIDS Coalition to Unleash Power (ACT UP) and the ‘right to be forgotten’ movement in cancer.

Example: ACT UP

ACT UP was formed in the late 1980s in response to a lack of government action to address the HIV and AIDS crisis. The group has had a global-level impact on how HIV/AIDS is perceived, treated and even clinically defined. Using the power of large-group advocacy (at its peak, ACT UP had over 140 chapters worldwide), the group’s actions led to:

the expansion of the definition of HIV/AIDS to better reflect the symptoms women experienced, which enabled better understanding of the impact of HIV/AIDS on women in the US and, importantly, made it easier for them to access clinical trials and effective treatments
a reduction in the price of the only drug approved to prevent and treat HIV/AIDS, which was unaffordable for the majority of patients, by directly targeting the pharmaceutical company that sold the drug. The group achieved a price drop from $10,000 to $6,400 per patient per year
the removal of HIV as a pre-existing condition for private insurance, expanding insurance availability and protecting people from potentially catastrophic health costs
the legalisation of needle exchange programmes to reduce the risk of transmission among people who use intravenous drugs.

ACT UP has had a global-level impact on how HIV/AIDS is perceived, treated and even clinically defined.

Example: The ‘right to be forgotten’

The European Initiative on Ending Discrimination against Cancer Survivors has been actively driving the ‘right to be forgotten’ (RTBF) movement. RTBF refers to the right of people who have had cancer – after a certain number of years has passed since their treatment – not to be required to disclose their former medical condition.

One in five people worldwide develop cancer. Improvements in early detection and treatment for many types of cancer have translated into better rates of survival; globally, the number of people living with cancer five years after diagnosis is estimated to be 50.6 million.

However, while these improvements in survival rates are positive, people who have had cancer are often required to disclose their medical history when applying for life, health or travel insurance, and they often experience discrimination related to their diagnosis. In many instances, people’s applications for insurance, bank loans and mortgages are rejected or, if approved, subject to significantly increased premiums (up to 300%).

France adopted RTBF into its national cancer plan in 2014. The advocacy work on RTBF has also led to the adoption of RTBF legislation in eight other EU Member States, which have taken legal measures to address financial discrimination against people living beyond cancer. As a result of this legislation, people diagnosed with cancer before the age of 18 are now exempt from disclosing it to insurers five years after their treatment has ended. For those diagnosed as adults, their medical history would be ‘forgotten’ after a defined period of time without any relapse or recurrence (this ranges from five to ten years, depending on the country). The RTBF movement has had a global impact, with other countries following suit.

Addressing barriers to patient advocacy

Despite these promising examples of success, it is important to recognise that patient advocacy exists to a varying degree in different countries and cultures. Its effectiveness in influencing policy often depends heavily on political, social and cultural norms as well as the power dynamics between stakeholders. People may also face physical or financial barriers to becoming involved in advocacy – including poor health, a lack of time, or limited availability to participate in meetings. Many of these barriers mirror those seen in the context of patient and public involvement in research, and in health and social care

Also, people may not feel equipped or confident enough to become patient advocates. Some organisations have made considerable efforts to address barriers by providing training, support and guidance; the European Patients’ Forum has developed the European Patients’ Academy of Therapeutic Innovation to provide training to the general public on many aspects of clinical research and regulatory frameworks. And the Workgroup of European Cancer Patient Advocacy Networks has developed the Evidence-Based Advocacy programme to help patients better understand evidence to support their advocacy needs.

Several organisations have carved out a role for patient advocacy to shape policy. Cancer Research UK, for example, supports a dedicated role for Campaigns Ambassadors. The European Society of Cardiology (ESC) established a Patient Forum in 2018 in recognition of the important role patients play in sharing practice. The Forum was recently involved in developing the ESC strategic plan, and many of its members also engage with policymakers at the EU level and locally.

A role for us all

Strengthening the voice of patient advocacy is everyone’s responsibility. At the most basic level, we can scrutinise the evidence we engage with to determine whether people with lived experience of a given condition have been able to contribute their experiences. Furthermore, we should review and adapt our research methodologies and policy-shaping activities to ensure that patient voices are purposefully and meaningfully included.

Patient advocates: vital voices in shaping health policy and care

Patient advocacy can wield significant power to inform and influence health policy. But more needs to be done to make sure patients’ experiences are not overlooked.

Recent Insights

What is patient advocacy and why is it important?

What role does patient advocacy play in shaping policy?

Patient advocacy has driven tangible policy change

Example: ACT UP

Example: The ‘right to be forgotten’

Addressing barriers to patient advocacy

A role for us all