Healthcare leaders in the Asia-Pacific region are keen to address the perception gap that exists around real-world evidence (RWE). While they recognise the significant advantages of RWE, patients are not aware of its actual or potential use. To bridge the current gap, the article offers two perspectives on the relevance of RWE to patient communities and strategies to foster greater multidisciplinary participation in RWE collection. How patient organisations use RWE to gain disease awareness traction and shape health policy and the importance of collaboration among multiple stakeholders are discussed in the article.
Hearing the diagnosis from the doctor that your child has spinal muscular atrophy (SMA), a severe genetic condition that leads to muscle wastage and often life changing complications, is something no parent should have to experience. Hearing those words with a second child goes beyond the unthinkable. Eighteen years ago, this was my reality. That is when I vowed that what was ultimately a terminal diagnosis for my two children, wouldn’t be for many others who would come after.
One in 35 people in Australia unknowingly carry the faulty SMA gene which can be passed down to newborns. The disease is cruel and can often lead to the most basic everyday actions that many of us take for granted, becoming an impossible challenge without the help of external care. Despite its brutal implications, treatment options and wider support services were rarely available in Australia at the time. As such, I recognised that the only path to meaningful change was if we as a patient community could demonstrate the burden of the disease, and the urgent need for radical improvements in the care available. Thus, I unknowingly began collecting what would turn out to be the largest set of privately held SMA Real World Evidence (RWE) records in Australia, and in parallel, started a patient organisation named SMA Australia, to enhance the quality of life for anyone associated with this unforgiving condition. Today, RWE is being utilised across a variety of conditions and it is my hope that other patient organisations can make use of this approach to help raise their voice in advocacy opportunities.
RWE are the clinical insights that are derived from a patient’s practical health status. RWE can help us to understand how patient individuality and habits impact health outcomes — thus helping predict the progression of a disease, a patient’s responses to a therapy, or the risk of adverse events. Use of RWE is by no means restricted to SMA — this approach can be applicable to a variety of conditions where patient organisations want to highlight the lived experience of their members. Unlike clinical trials, whose cohorts and conditions are often very restrictive, RWE can be sourced from a large number of individuals living in the population, sometimes only restricted by the number of patients living with a condition. By gathering wider context around how someone lives, we are able to understand the more personal and deeper consequences of a condition on both patients, as well as their families.
RWE is not a new phenomenon but with the advent of new digital and advanced analytics, the opportunities for enhanced utilisation across a variety of diseases is improving. Presently, the information plays a critical role in the development of treatments, the creation of health policy, and in understanding the overall lived experience for patients with a number of conditions. While advances in modern medicine have helped us understand the components of many diseases, the implications on a patient’s day-to-day experience still needs to be derived from them. The expansion of online patient organisations and communities has provided a platform for this evidence to be collected and shared, meaning that we now have a much clearer picture of the impact on quality of life across multiple populations. At the outset of my research, my data was focused largely on the epidemiology of the condition: How old was the individual? What SMA condition type did they have? Were they male or female? While this was hugely valuable for mapping the condition across Australia, many of the unique findings around quality of life came much later.
Despite securing some valuable SMA data, the perennial battle that I, and many other patient organisations face, is being included in the meaningful discussions that will ultimately shape your members’ lives, and the lives their families. Throughout my 18 years of being involved in the SMA community, I have often encountered shut doors where my opinions and data have been disregarded. The reality of engaging in a medical field is that it can be a tightly knit group that is often difficult to participate in if you don’t have the relevant experience. Well, my experience was that I had two children who lived with SMA and had been collecting data across the country for years, and in my mind, that made me and others like me, qualified to join the discussion.
However, I am pleased to say that the tide is changing, through platforms like The Alliance & Partnerships for Patient Innovation & Solutions (APPIS), for which I am a 2023 Council Member and I was also a speaker at the recent APPIS 2023 Summit. APPIS is a platform that is co-created by the APPIS Council, a panel of patient and healthcare leaders in Asia Pacific, Middle East and Africa, and organised and funded by Novartis. Since 2021, the platform has convened patient advocates and healthcare stakeholders from more than 60 countries to share their experience of a variety of conditions alongside a network of experts including: leading clinicians, healthcare providers, health policy experts, health tech companies and industry representatives, and more. APPIS provides opportunities to exchange ideas and contribute towards key discussions around the themes of health literacy, healthcare policy shaping and digital health and communications. The goal of APPIS is to help accelerate access for patients by bringing together the members of the health community to work together to create solutions. The APPIS platform and community helps empower and inspire patient organisations to think that they too can achieve progress using RWE in their countries by arming them with guidance reports and the right tools they need to expand their impact for the community.
The broader recognition for the power of RWE data has coincided with new collection processes. What used to take three to four years to collect can now be gathered in 12-to-18 months. RWE can help to provide quick analysis that can complement the existing scientific research and potentially address the unknown surrounding patient experience. For SMA Australia, much of our value was centred around highlighting the level of carer assistance often required by patients. This sort of detail is often outside the remit of clinical trials or health policy research, but coordination of these services is a significant part of the lived experience of patients and their families. By understanding this logistical challenge, it gave us a strong platform to call for improved access to treatments that could help to provide patients with greater personal autonomy.
Increasing support is being given to the notion that we cannot expect policy initiatives to have positive outcomes if we haven’t consulted with the group that it directly relates to. For SMA Australia, this was one of our key messages and when a platform was provided to us, we could accurately demonstrate the unmet need we had, and work with all stakeholders to push towards practical solutions.
While partnerships with all stakeholders are essential for securing long-term policy change, there are some actions that patient organisations can take to positively position themselves to highlight the unmet needs of their communities. Here are some of the lessons I’ve learnt from nearly two decades in this space:
When I first started SMA Australia, it felt like an uphill battle. It was a one-person band where I was responsible for bookkeeping, marketing, copywriting, and all the rest. However, it was during this time that I started to engage with the community, to understand the hopes and trials of patients and families, and what more could be done to help them. At this critical juncture, treatment access and policy change felt like a lifetime away. However, with each connection, the ball starts to roll and our voice gets louder and louder. Soon you start to get traction and people sit up and listen. Ultimately, I knew that if the data was of value for me, it had the potential to be valuable for other stakeholders, which is why I encourage other patient organisations to collect early and broadly.
I encourage patient groups to gather data early, and to focus on the daily lived experience of the patients. While the epidemiology data is useful, there are now large datasets available within government public health organisations. However, they do not always have access to the day-to-day practical insights that truly indicate the quality of life of a patient. For example, when thinking about how the treatment will help the patient, it’s important to think about enhancing the quality of patients’ everyday lives. It’s up to patient groups to communicate patients’ unmet needs to stakeholders, while communicating to patients about treatment and care options. This is where patient groups have significant value and by grouping our voices, we can provide robust reasoning for why we need advancements in access. Further, when engaging with the community, try to provide regular updates, as this is when people become frustrated, when they feel that stakeholders are unresponsive.
It is easy to assume that governments want to protect budgets and are not interested in hearing about the need for enhanced resources for patients. However, through my time working at SMA Australia, we approached a number of policymakers directly and personably, and managed to secure advocates who became champions for our cause. In many cases, they found the work to be deeply rewarding and one of the key reasons for engaging in public service. Setting the tone by highlighting the quality of life for patients is crucial, followed by having a clear ask around what they can do to help. I encourage patient organisations to cast a wide net and think about who can support on your journey. I have seen first-hand how patient representatives can emphasise the harsh reality of disease and highlight the need for improved access to care and treatment to improve the quality of life of patients. If we don’t tell our story, politicians may not appreciate the scale of the problem.
Utilising RWE effectively can be an overwhelming experience for patient organisations, but we don’t need to start from scratch or do it alone. Being part of the APPIS community can help you gain access to helpful resources and a network of members who can share best practices around amplifying the patient’s voice to secure access to healthcare and policy change. By engaging with a broad community of stakeholders we can exchange ideas and build comprehensive policy asks that can help to deliver improvements in the care and quality of life of patients.
Although the burden of diseases like SMA is something no family wants to endure, the strength of the patient community is hugely powerful and can be a valuable support in tough circumstances. We now have an opportunity in an increasingly connected world to interact with each other and amplify our advocacy. APPIS brings together stakeholders from across the healthcare landscape and provides a unique platform to share best practice around how we improve the lives of patients. Patient groups should be consulted throughout the disease cycle if we are to develop impactful and long-lasting health policy. One thing is certain, everyone has a role to play and by engaging with all stakeholders, we can ensure that care for patients with SMA and other conditions can be levelled up.
You can find out more about APPIS 2023 here1, where you will be able to rewatch the insightful presentations, download useful reports from the APPIS Resource Centre and join our growing community.
Ruth Kuguru is the Executive Director, Communications & Patient Engagement for Novartis Asia Pacific, Middle East, and Africa. In her role, she leads her team to foster open, inclusive, and strong partnerships with patient communities, media, and healthcare leaders in Asia Pacific, Middle East and African countries. She is passionate about the impact strong collaboration can have in accelerating access and developing innovative solutions for patients.
Julie Cini founded Spinal Muscular Atrophy Australia in 2005, and had grown a successful non-profit patient advocacy group. For 18 years under Julie’s leadership in this organisation has seen her successfully campaign for increased awareness, diagnosis and better access to treatment for SMA patients. Julie’s consumer led research has had a great impact on the Pharmaceutical Benefits Advisory Committee (PBAC) and highlighting the severe unmet need within the SMA community for treatment. She continues to work with the policy makers to ensure patient evidence is valued and addressed as part of the submissions.
