Pharma 2024

Apr 16, 2024 - Apr 18, 2024, CCIB, Barcelona

Transform Pharma. Reinvent Healthcare. Unlock Access.

How pharma is unleashing the benefits of real-world evidence along the value chain

Real-world evidence (RWE) is providing a fast track to approval and reimbursement.

By collecting and analysing real-world data (RWD), pharmaceutical companies are generating key evidence that supports regulatory authorisations and influences decisions at health technology assessment bodies and payers. Yet, in a young, emerging field, the industry needs to evolve to unleash the full benefits of RWE.

Experts discussed the current landscape at a Reuters Pharma 2023 panel, sharing the strategies behind successes and details of what needs to change to maximise the value of RWE. Catia Proenca, Director in Real World Solutions at Alira Health, set the scene.

“The more data is being collected and the more healthcare systems are being digitalised, we're starting to see real-world data being used in different ways and even ... towards the direction of using real-world data to demonstrate treatment effects,” Proenca said.

Using RWE to unlock markets

Other panellists shared examples of how RWE is already providing value. Gorana Capkun, global head, RWE analytics and enablement at Novartis, discussed work to validate a PI3K inhibitor in PIK3CA-related overgrowth spectrum (PROS). A French physician used the drug, alpelisib, in PROS off-label, generating evidence of efficacy in children with the rare diseases. The FDA encouraged Novartis to collect more data in the indication, leading to an approval based on the results of a retrospective chart review study.1

RWE empowered Novartis to quickly win approval to address an unmet medical need. Rolland Kacsoh, global product director, iron disorders, and rare anemias at Novo Nordisk, told a similar story, explaining how two of the seven indications treated by recombinant factor VIIa product NovoSeven are supported entirely by RWD.

Novo Nordisk used RWD to show the efficacy of the product in the rare inherited blood clotting disorder Glanzmann's thrombasthenia and postpartum bleeding.2,3 Kacsoh outlined how Novo Nordisk worked with European regulators to identify and provide the data to support the postpartum bleeding approval.

“We had a very productive and robust collaboration, back and forth several times, around the different real-world data sources around the world that could be accessed and used to demonstrate the safety for the product. Also, [we discussed] which ones we would exclude and how we might treat and work with the data so that it was satisfactory to them,” Kacsoh said.

Forming strategies for showing value

The early successes have happened despite challenges that are preventing companies from unleashing the benefits of RWE along the value chain. Companies are still forming and communicating best practices in the emerging area.

“Every single team is trying to do something, but I think we don't really have a clearly defined process of how to do it,” Capkun said. The Novartis RWE expert explained that sometimes the knowledge needed to unleash the benefits is siloed to part of a company, or the organisation lacks a structure that enables all teams to get “together around the table, thinking about evidence generation and all [the] possibilities.”

The challenge, as Capkun sees things, is figuring out how to integrate RWE into organisations. When RWE expertise is siloed, companies might only start thinking about how they can use the resource to support a product after winning approval, potentially setting back their plans to receive reimbursement.

Kacsoh discussed how Novo Nordisk has organised its rare disease teams to ensure RWE is considered early and effectively in the drug development process.  By involving a part of the commercial team in evidence generation planning from preclinical stages onward, rather than only getting involved in phase 3 as is traditional, Novo Nordisk is able to prepare for commercialization more effectively. This early involvement includes patient-reported outcomes, market research, and more.

“Contacting and reaching out to thousands of patients very, very early on is so useful to be able to make sure that when you're getting to the clinical trial, you're not just thinking about what the regulatory folks are going to be looking for as a primary outcome, but you're including the outcomes that are going to matter to the payer,” Kacsoh said.

The need to consider the demands of regulators and payers reflects the fact the groups – regulators and payers - have different priorities. As Omar Ali, visiting lecturer, value-based pricing and innovative contracting of new medicines at the University of Portsmouth, put it, “regulators don't care about value, it's not their job.”

Ali, a former adviser to the UK National Institute for Health and Care Excellence, went on to push back against the idea that regulators and payers should align, saying that “the arm's length distance” between the two groups is “somewhat healthy.” Using a car analogy, Ali said the regulator’s job is to establish that a vehicle “is not going to catch fire” but it has no interest in features that add value such as a sunroof.

The future of RWE

With regulators and payers set to demand different evidence for the foreseeable future, companies need to plan drug development programs capable of efficiently addressing all requirements. Capkun identified pragmatic designs, studies that assess interventions in real-life conditions, and hybrid trials that combine pragmatic and randomised concepts as the future.

“If we don't go pragmatic, if we do not actually test how something will work in the real world, ... we are going to run into more complex situations. I think it is very, very important that we start thinking on a more regular basis, how can we combine RCTs with real-world data, not as separate entities, but also together. I think that's the future: prospectively thinking about combinations,” Capkun said.

Kacsoh added that Novo Nordisk is working to future proof its operation by investing in PRO collection methods. Specifically, the company is focusing on how data, digital, devices, and diagnostics can support its operations. Through such investments, biopharma companies are building on early successes in RWE, knocking down the remaining barriers and unleashing the benefits across the value chain.


1.     FDA approves Novartis Vijoice® (alpelisib) as first and only treatment for select patients with PIK3CA-Related Overgrowth Spectrum (PROS). Novartis (2022).

2.     NovoSeven® RT for Glanzmann’s thrombasthenia. novoMEDLINK

3.     CHMP recommends approval of Novo Nordisk’s hemophilia drug for postpartum bleeding. (2022).

Pharma 2024

Apr 16, 2024 - Apr 18, 2024, CCIB, Barcelona

Transform Pharma. Reinvent Healthcare. Unlock Access.