The increasing importance of Real World Evidence (RWE) and the need for a multi-stakeholder approach

In this guest blog post, Takeda’s Andre Vidal Pinheiro evaluates the value of partnerships across the medicines lifecycle and why real-world evidence (RWE) is fundamental for healthcare systems to provide better and tailored outcomes for patients. 

The world is moving quickly and the pace of digital transformation is unprecedented. The proliferation of data and the greater technology pool can benefit decision-making and partnerships. It can ultimately offer greater access to therapies to patients, especially in rare diseases. Within the EU, the life sciences industry requires a strong innovation framework across the patient journey so that such technological advancements can be used to the benefit of all stakeholders.

There is no one-size-fits-all approach to value-based healthcare and data generation & collection. Each model should be adapted to the different needs of the different populations as well as in the different geographies. Often we will need to experiment a lot to learn which approach works best, and this is the reason why collaborative efforts – such as partnerships – across the healthcare system, help to deliver those better outcomes for patients and have a positive impact on the overall healthcare system.

Bridging Gaps through Real-World Evidence (RWE)

Clinical studies have and always will be the foundation to assess the efficacy and safety of new therapies. However, the rise and use of real world evidence (RWE) can complement clinical studies, and in many cases, provide answers to questions that cannot be addressed by randomized clinical trials (RCTs). As an example: if we want to assess what the real world utilization of a given therapy is, interventional studies will never answer this question. Another common situation in rare diseases is the scarcity of natural history data, which is often impractical (and sometimes impossible in chronic life-long diseases) to collect prospectively. Finally, clinical studies feasibility may limit the nature, number and quality of comparators to fully reflect standard of care across all countries.

This is the reason why it is fundamental for us to think beyond just interventional clinical trials and complement the information that RCTs can bring us with real world evidence. The challenge is, and I speak from an access perspective, that not all HTAs and payers have the same frameworks for acceptance of RWE and how it can inform decision-making. Part of the challenge is the heterogeneity on data collection methodologies and use of digital when it comes to the real world evidence generation. Significant standardization, legal/regulatory framework, scope and awareness/ education is still needed, for both further application and acceptance.

It’s also the perfect timing for such discussions given the road to the pan-European Joint Clinical Assessment. RWE can contribute towards a JCA process that truly improves the quality of clinical assessments, and highlights potential benefits and uncertainties around new technologies to the benefit of all member states’ HTAs. With the JCA seeking to answer questions from multiple views (e.g. variance in standard of care across countries), RWE can provide invaluable evidence to adequately address such questions, in addition to RCTs.

Electronic Health Records

Over the last decade, we’ve seen huge developments in digital innovation, including the increasing proliferation of digital health records and data sets available which can help improve scientific understanding and decision-making in general. The challenge we face often is the fragmentation of those data sets, which can vary tremendously, not only across member states within Europe but also within the country. This makes the analysis and the use of aggregate data very challenging.

The other limitation is the heterogeneity in terms of rules regarding ownership of the data, utilization of the data and patient consent. The European Health Data Space (EHDS) does provide a framework to empower individuals through increased digital access and to control their electronic personal health data at national level. The EHDS also supports their free movement as well as fostering a genuine single market for electronic health record systems that are relevant for healthcare and drug development. For HTAs, payers and industry it also offers a common language and understanding of array of potential data sources, standardization and convergence of methodologies, a better understanding of their limitations, and increases the overall quality of outputs.

Go for Rare

The need for real-world evidence is particularly critical for rare diseases because the data surrounding these diseases are scarce. It is well known that looking at reimbursement and access indicators, rare diseases tend to take longer to achieve reimbursement across Europe. And in many countries, the access that they get is more restricted beyond their regulatory approval than non-orphan drugs. Often, this is due to uncertainty around the evidence that exists through classical randomized clinical studies/trials. So, once again, real world evidence can help to accelerate patient access to orphan drugs. RWE offers more information on the disease, epidemiology, patient journey and unmet need, as well as effectiveness, safety and utilization of new technologies, which ultimately reduce the uncertainty in decision making.

The question still stands on how RWE and data can be collected for this purpose. EHDS would help. A common framework could foster investment and new ways of evidence generation. Would empower patients and lead to greater transparency and predictability. Though this alone would not be sufficient, it also requires that stakeholders re-think their ways of engagement and collaboration.

A Multi-stakeholder Approach

At Takeda, every decision we make starts with a patient and follows the very strict framework of assessing the benefit to patients, trust, reputation, and business, in this order. We are constantly looking for opportunities to engage with stakeholders in different countries across different disease areas to further the discussion and provide additional benefits to healthcare systems and patients. We need to constantly challenge ourselves to think differently and innovate with courage.

Whether it’s through exploring artificial intelligence algorithms and electronic medical records to identify patients with rare diseases that are lost in the system, to helping increase efficiency in the diagnosis and treatment of rare diseases, or exploring alternative ways to collect real-world evidence through patient diaries or apps, we always seek to find opportunities for true partnerships with our stakeholders.

One of the partnerships I am proud of is RWE4Decisions. It’s a true multi-stakeholder initiative that promotes the use of RWE to inform decisions by healthcare systems, clinicians, policy makers, industry, payers and patients, all based on the principles of collaboration and transparency. Will take the opportunity to highlight two events where RWE4Decisions will promote discussions on this topic: At the World Orphan Drug Conference in Barcelona, on the 15th November we will host a panel on “How can the European Health Data Space help determine value of Orphan Drugs”. Also on 24th November 2022, in Brussels, we will have the “RWE4Decisions Symposium: Policies & Partnerships”. You can find out more in

Collaboration and Transparency

The old model – where industry was the main contributor to evidence generation on treatments and played a key role in dissemination – is gone. Now, we have many different sources of data that are generated by an increasingly greater number of groups – from clinicians to health insurers and patient groups. Through innovations in data and technology, real world evidence generation has become more democratic, and as a consequence, a shared responsibility.

Within the realm of collaboration, dialogues should involve all stakeholders throughout the lifecycle of the technology, to align on big scientific and public health questions. This way, evidence generation plans and partnerships can be more readily established, to realize the full potential of real world evidence. To note that questions about a new technology yet to reach a patient will be completely different from a therapy already used for several years and where multiple alternatives exist. This is why such dialogue and collaboration needs to be iterative in nature.

In order for a higher degree of collaboration and flexibility to work, transparency becomes essential. Real-world evidence needs should be shared publicly and discussed more broadly. It may also require standardization in nomenclature and methodology to facilitate understanding across stakeholders. We should continue to strive for more inclusive consultation and discussion processes leading to formulate the questions, as well as interpreting the answers, on RWE. This is particularly critical in the regulatory space and in pricing and reimbursement decisions, where we can do much better.

Harnessing RWE for EU Innovation

It’s clear that real world evidence will play an increasingly greater role in the current and future development of healthcare systems. The complex processes behind classical clinical development and RCTs require a lot of resources, and due to their interventional nature, they will never accurately represent the realities of different healthcare systems. Even when trying to be as inclusive as possible, considering different needs regarding endpoints, patient populations, and comparators, RCTs will never sufficiently meet the needs from all stakeholders, everywhere. Real-world evidence is the missing link that can be used to complement those data. And as technology evolves, collaboration to effectively tackle the big scientific questions will require new levels of collaboration. Through multi-stakeholder partnerships like RWE4Decisions, we’re on the right path to harness it and to drive biopharmaceutical innovation.

October 27, 2022

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