As healthcare systems across Asia Pacific (APAC) start to gather more real-world evidence (RWE), this article takes a look at how patient groups and the wider healthcare community can come together through public and private partnerships and platforms such as the Alliance & Partnership for Patient Innovation & Solutions (APPIS), to incorporate RWE into health policy decision-making and better reflect patients’ needs. Clinical data has been traditionally used to inform healthcare choices, but looking at RWE, in all its forms, has become increasingly important in understanding the more holistic impact on patients.

How is RWE being used in APAC?

Healthcare systems across APAC are incredibly diverse. In recent years, we’ve seen markets with national healthcare systems increasingly incorporating RWE into practises, including post-market pharmacovigilance, testing innovative treatments, insurance reimbursement, clinical usage, and healthcare policy decision-making.

Taiwan, for instance, has comprehensive national databases, including the Taiwan National Health Insurance Research Database (NHIRD), as well as electronic medical records, hospitals’ medical records, national registries, and investigator-initiated studies. These sources of data contain comprehensive, large-scale records such as demographics, prescriptions, and treatments, all of which can be used as powerful sources to generate RWE and shape healthcare policies.

Early access zones can also expedite approvals for innovative medication. In China, pilot zones like Boao Lecheng have generated RWE to support the approval of new drugs from the United States and Europe for wider use in other areas of China, creating the opportunity to reduce or even avoid the need for further clinical trials. We’ve seen drugs to treat rare diseases being successfully approved in China using this pathway, and in other disease areas, RWE is being used in conjunction with clinical trials to obtain full approval.

Other developed markets with robust and systemic healthcare systems, like South Korea, Singapore and Japan, also incorporate RWE to inform policies in areas of health financing, reimbursement policies, and healthcare innovation. Meanwhile, developing nations without nationwide healthcare databases may find it more difficult to generate RWE to inform healthcare policies, especially those that lack resources and infrastructure, but they are exploring other ways to generate RWE to expand the breadth and depth of their healthcare systems.

There is an opportunity for governments to leverage RWE to provide better care and coverage for their populations. However, due to the lack of resources, they often need to make trade-offs and issues may go unaddressed, especially those of underrepresented patient groups. This is where the healthcare community can step in to partner and fill the gap.

The role of RWE in shaping health policies for underrepresented groups

Governments commonly adopt randomised control trials for their policy formation processes, but that methodology has shortcomings that RWE can easily address. Most notably, vulnerable and underrepresented populations are commonly excluded from the trials.

Researchers avoid paediatric trials due to the lack of funding, and rapid developments of the body make it difficult to compare children of different ages. There are also ethical concerns as children lack the capacity to understand the potential risks of trials. Pregnant women, on the other hand, are traditionally excluded from trials out of ethical considerations for potential damage to foetuses and an increased perception of risk. Additionally, the elderly – typically those over 80 – are excluded due to heightened adverse reactions, especially in drug trials. However, they are still a significant population whose treatments need to be supported by evidence like RWE.

When weighing the pros and cons between RWE and randomised clinical trials, RWE is often the preferred option, as it presents concrete data on benefits, costs, and potential impact – data that the government places heavy consideration on when it comes to policymaking. It is important to note that the data required for policymaking and clinical trials are quite different. The former requires descriptive data – a disease’s background information, economic burdens of the disease and treatments, the percentage of population affected, and so on; data used for clinical purposes require more data elements such as sophisticated study designs and treatment outcomes.

How can patient groups fill the gap by generating RWE?

It’s not just medical professionals who can generate RWE. Equipped with the right data collection processes, patient groups (with support from clinicians) can create small, clean subsets of the population that are representative of what is happening in the real world and provide immediate evidence to inform policymaking. It is particularly beneficial in the rare diseases space, where we often see more patient groups.  

Patient groups are commonly underrepresented and under resourced. Through our interactions in China for example, we find that they are generally eager to find ways to systemically create change for those they are representing but are not equipped with the information or infrastructure to push for this change. Beyond policymaking, patient groups can play a role in collecting relevant data sets and first-hand patient reported outcomes that supplement their national health database and play a part in developing and refining their national registries and data sets with more inclusive data.

Challenges of RWE generated by patient groups

Patient groups have great potential to generate their own RWE, yet in Asia, many communities are not aware of RWE and its use cases. Many organisations don’t have a deep understanding of the importance of collecting data, and patients are unwilling to share their data due to privacy issues. Thus, encouraging patients and peers to share data for RWE becomes the first barrier we must address.

In some instances, groups like ASK Health Asia encourage patient groups to prioritise privacy by separating personal information from RWE and keeping personal patient information within the patient groups – especially if data collection was not performed by authorised institutions such as hospitals. Another method to ensure privacy is through deleting private information after specified durations so personal information won’t be leveraged for purposes other than its original intention.

In addition, we find that smaller patient groups without professional staff are not always aware of ways to ensure patient confidentiality. This is where collaboration between private and public sectors comes into play, as patient groups would benefit from professional partnership opportunities with academics, analytical organisations, and others who are well-versed in patient confidentiality and can offer guidance and support. Another aspect we must take into account is the capability of patient groups, organisations, and third parties in collecting usable real-world data, and the government’s willingness to leverage the evidence for policy making.

Platforms like APPIS have spent the last few years providing educational sessions to guide patient organisations and other healthcare stakeholders in the collection and usage of RWE in health policy shaping.

To ensure that data gathered is unbiased and is suitable to be taken into consideration by governments to inform decision making, patient groups will need to generate data without bias. A partnership with academics, clinicians, or other professionals will also be able to guide patient groups to develop rigorous protocols and methodologies and help them convert the data into suitable RWE.

We find that governments are willing to use RWE generated by patient groups, as long as the evidence contains usable data that are relevant to the population and collected using a proven methodology. Case in point: in China, the government has begun to pay more attention to RWE. More think tanks, organisations, and academic institutions are devoting their efforts into developing RWE tools, thus helping to raise awareness about it in the country. It is clear that public health institutions are keen to understand real values, outcomes, and budgets for innovative use cases that can help them make informed decisions around shaping healthcare policies.

What’s next for RWE in Asia?

To support patient groups to develop suitable RWE that helps shape healthcare policies, we need to raise awareness about RWE to a wider audience, dispel misunderstandings, and create frameworks that can be used by groups with limited resources to further facilitate collaboration between public and private stakeholders.

For instance, ASK Health Asia is in the process of developing toolkits with industry partners to create systemic designs for patient groups to collect data for RWE. They are also extending outreach to hundreds of patient groups, including umbrella patient organisations such as the Chinese Organisation for Rare Disorders (CORD), to raise awareness about the possibilities of RWE and assist them in presenting their data to local governments. By providing methods to collect and analyse data by themselves, ASK Health Asia empowers them to reach their own objectives and generate RWE use cases.

The toolkits aim to be presented to as many patient groups as possible through platforms such as APPIS, which convenes healthcare stakeholders to prioritise action towards addressing patient access barriers. Its initiatives such as the APPIS Summit, which took place last month, and local APPISx sessions, which focus on country-level engagements, play an important role in facilitating the shaping of health policies around RWE through dialogue and knowledge-sharing year-round.

In India, for example, the opportunity of RWE lies in addressing the gaps in healthcare data – which is commonly paper-based and fragmented, making it difficult to track patient outcomes and identify trends. This was the topic at hand during a 2023 APPISx India session, which brought together patient organisation leaders and medical experts, including Dr Moni Abraham, Medical Director & CEO of Karkinos Healthcare and an RWE expert. The session illuminated how RWE can help provide a more comprehensive understanding of a treatment’s safety and effectiveness for patients in real-world settings, which in turn plays a crucial role in shaping healthcare policies and practises.
Another example of collaboration between public and private organisations for RWE development is the Taiwan Society for Pharmacoeconomic and Outcomes Research (TaSPOR). The organisation is currently in the process of establishing a task force in collaboration with the United Kingdom’s National Institute for Health and Care Excellence (NICE) International. The task force will incorporate NICE’s RWE Framework to develop a comprehensive RWE framework in Taiwan, which will establish terminologies that different stakeholders, from patient groups and hospitals to governments, can align on to better facilitate cross-sector collaboration.

To support the task force’s framework development process, TaSPOR invited Taiwan’s Centre of Drug Evaluation (CDE) to provide case studies of how the CDE has used RWE in their review process in policy shaping efforts. Once TaSPOR sets up a first draft of the framework, the organisation will invite different healthcare stakeholders, including patient groups, to provide feedback and advise on the adaptability of the framework in Taiwan – a true example of public-private partnership.

It’s a win-win situation

RWE developed by patient groups in Asia can inform and create new healthcare policies, especially in countries with less resources, to accelerate treatment to groups that are underrepresented, including patients living with rare diseases. Empowering patient groups to take on the challenge of generating RWE and making this process better known and understood will create a positive influence for governments, providing them with a clearer picture of their population’s needs and encouraging them to incorporate RWE into more policy formation processes.

In addition, sharing these success storeys about patient groups that helped generate RWE that have influenced health policies can inspire and accelerate the development of robust patient organisations and communities, especially in developing countries. RWE generation models in more developed countries can even build global pioneer models for RWE in emerging economies and ultimately support systemic data infrastructures for RWE.

RWE can be developed through national databases, but data from patient groups have the possibility to act as another source of relevant data that can further improve on current RWE ecosystems, shining a spotlight on underrepresented patient groups with lesser-known diseases, and influencing impactful policy making decisions. Enabling partnerships between different stakeholders and regions is thus a significant component to the collective improvement of healthcare systems across APAC, which are in turn crucial in accelerating patients’ access to treatment, especially in countries where they need it most.

By bringing healthcare communities together across patient groups, academia, and public policy, and more, we can work together to better use RWE to facilitate access for patients.

--Issue 64--