In the world of rare diseases, real-world evidence can provide an essential supplement to boost HTA submissions and accelerate clinical research.
Real-world evidence (RWE) is becoming increasingly important in a changing clinical studies world. The GlobalData Mid-Year 2024 State of the Biopharmaceutical Industry survey interviewed over 120 industry professionals and found that RWE was ranked as the fourth most impactful trend for the coming year, ahead of cell and gene therapies and artificial intelligence (AI).
The amount of real-world data available has surged in recent years with the advent of wearables and mobile health (mHealth) apps that generate real-time data directly from patients. There is now strong adoption of electronic health records (EHR) across Europe, which provide access to medical history, diagnoses, and prescriptions, making them a critical source of RWE. Patient registries are another valuable method for collecting real-world data through observational study methods. They facilitate the collection of standardised information about groups of patients sharing a specific condition, which can be critical for understanding treatment outcomes, disease progression, and the overall healthcare landscape in the region.
Alongside the impact on clinical trials, RWE is set to play a key role in the conduct of health technology assessments (HTAs). While concerns remain over data privacy and validity, when used in the right way RWE can assess the real-world efficacy of treatments and improve overall decision making in HTAs.
The power of empirical evidence
One respondent to the GlobalData Biopharmaceutical Industry survey, a VP based in North America, noted: “Empirical evidence is powerful, more so than AI predictions. Real-world evidence will continue to impact trends in anti-obesity meds, immuno oncology, and biologics.”
By gathering insights derived from real-world practise rather than randomised controlled trials (RCTs), RWE can provide a more comprehensive picture of how treatments perform in broader, less controlled populations. This could help to overcome the bias often seen in RCT data, since the types of people who are able to participate in a trial are very often unreflective of the population.
One region where RWE could have a particularly significant impact is the Nordic countries (Norway, Denmark, Sweden, Finland, and Iceland). These nations have extensive health registries, such as the Swedish National Patient Register and Danish National Health Data, as well as reliable prescription databases. These data sets can together be marshalled to create a credible picture of real-world health across different demographics.
“The national patient registries in the Nordics are invaluable,” says Laila Mehkri, Senior Health Economist at Abacus Medicine Pharma Services. “Their records are among the most comprehensive globally. While this data may not become a mandatory component of HTA submissions, it has great potential to support and validate them.”
The completeness of this data across demographics makes it highly prized in the pharmaceutical world and can be extremely helpful for informing HTAs.
How real-world data complements RCTs
Unlike RCTs, which measure efficacy in controlled environments, real-world evidence evaluates how a treatment performs in everyday practice, providing evidence on actual patient outcomes. When RCT data is limited or not generalisable, HTA bodies can use RWE to make informed decisions about reimbursement, pricing, and the overall value of health interventions. RWE is also vital for post-marketing surveillance and monitoring the long-term safety and efficacy of a drug.
“RWE can play a pivotal role in HTAs. When combined with clinical studies, expert opinions, patient testimonials, and other relevant data, RWE significantly strengthens an HTA submission,” says Mehkri. “Moreover, RWE not only demonstrates the value of a treatment but also clarifies its appropriate indications. It provides valuable insights into the disease itself, offering the industry a deeper understanding of treatment impact and patient needs.”
RWE is already being utilised in a variety of ways to inform HTA submissions. As discussed, it can fill gaps in clinical trial evidence, providing additional insights where RCT data may not reflect real-world settings or diverse populations. Since RWE can be collected over extended periods, it assesses the long-term safety and efficacy of treatments post-approval. Additionally, in the world of rare diseases, RWE is essential where RCTs are difficult to conduct and can help justify early access and reimbursement decisions.
HTA attitudes to real-world evidence are changing
As a result, HTA bodies are increasingly recognising the value of RWE, particularly in rare disease use cases where the RCT data is insufficient. In the Nordics, HTA bodies such as the Swedish Dental and Pharmaceutical Benefits Agency (TLV) and the Danish Medicines Council are gradually integrating RWE into their evaluations. However, RWE is being used in complement to traditional clinical trial data rather than as a replacement.
It is still important to note that there are some challenges with the use of RWE in HTA submissions. This includes the data’s generalisability across markets, where clinical practices and population demographics can differ significantly from country to country. This may require companies to gather and compare data from multiple markets within a single study, thus increasing the complexity of the research.
Moreover, concerns persist regarding the confidentiality of real-world data, particularly in the context of rare diseases where patient populations are very small. “The GDPR, the EU’s data privacy law, plays a significant role here,” Mehkri explains. “Patient registries are limited in the information they can disclose, including small patient numbers, as they must comply with strict privacy safeguards—especially if there is any risk of individuals being re-identified from that data. Whenever possible, fully anonymizing or suppressing small counts is recommended, along with robust documentation to justify the data-sharing approach.”
A partner to support real-world evidence use cases
Despite these challenges, real-world evidence will remain essential in a pharmaceutical world increasingly focused on addressing the gaps in the rare disease treatment landscape. At Abacus Medicine Pharma Services, we have been using real-world insights to support the commercialisation of our partners’ rare disease assets in European markets such as the Nordics for many years, helping international companies access new markets and bring their products to patients in need.
Real-world evidence has become a particularly important part of our health economics and outcomes research (HEOR), a key service we provide for evaluating the value of rare disease interventions and demonstrating their value to stakeholders such as patients, physicians, payers, and regulatory authorities. This enables our experts to quantify the economic burden of the disease, assess the cost-effectiveness of the new intervention compared to any existing treatments, and justify the proposed pricing strategy.
Navigating the complex landscape of drug development can be challenging, but real-world evidence can be decisive in proving a treatment’s value to the commercial landscape. With the quality and volume of patient data only increasing in the Nordics and other regions, RWE will continue to provide an essential complement to clinical research data in the years ahead.
Interested in learning more about how we can help demonstrate the value of your rare disease asset?