This year, Dr. Deepti Tayal, our CEO, attended several Real World Evidence, PICO, and Market Access related summits. The key topics of discussion at those events revolved around the same set of questions: Where is AI adding real value in RWE? How should early evidence be planned? How prepared are biopharma and healthcare companies for the EU Joint Clinical Assessment? What are the global patterns Europe may be overlooking? She gives her take on all of those in this excellent piece, along with how Ingenious e-Brain is navigating its consulting approach to better serve pharma and life sciences clients.
1. Where is AI adding real value in RWE, and where is it still hype?
AI is clearly establishing its value in the core work of real world evidence (RWE). It helps extract clinical variables out of the unstructured physician notes, speeds up literature review, and improves data curation and linkage. In the rare diseases segment, AI plays a vital role in the patient finding space assisting clinicians in reviewing the results. The hype comes in when AI output is presented as evidence without any clear validation. That’s how HTA bodies and payers will assess AI-enabled RWE. They’ll look in terms of transparency, reproducibility, and whether it can be checked against human abstraction, rather than how quickly it was produced.
2. What is the most common mistake companies make when planning Real World Evidence for access?
The most common mistake is to treat Real World Evidence as a late-stage solution rather than as part of the evidence strategy. If you’re only looking at RWE in the context of a late stage (post Phase III), your comparators, endpoints and target populations are likely all already locked in, and the data simply won’t be able to answer the questions that payers actually ask. RWE for access is best when you work backwards from the HTA question and build it into your development plan at an early stage, that is, after Phase 1 or 2.
3. The EU Joint Clinical Assessment (JCA) is now live for oncology and ATMPs. What are early experiences telling us, and what were companies least prepared for?
The first JCA reports are a real indicator of just how complex the assessment questions can be. Tovorafenib had eight PICOs proposed, but the report only covered one subpopulation. Onasemnogene abeparvovec had fourteen PICOs, of which seven were assessed. From scoping to final report, it took roughly 12 to 16 months. Companies were least prepared for three things:
- The number of comparators requested across Member States.
- The short window between receiving the final PICO and submitting the dossier.
- The level of scrutiny given to trials run mostly outside Europe.
Indirect comparisons are acceptable, provided there is a clear methodological justification.
4. EU Joint Clinical Assessment (JCA) will extend to orphan drugs in January 2028 and to all new medicines in 2030. What should companies with 2027 to 2030 launches be doing now?
Companies should start working on predicting their PICOs now, whilst the trial design is still something that can be influenced. Priority actions include:
- Mapping relevant comparators across key Member States.
- Testing whether indirect comparisons are feasible before late-stage studies are finalised.
- Considering a Joint Scientific Consultation.
For orphan products, you need to plan your natural history data and your external control strategy right from the start, because small patient populations leave little room to fix gaps later. Internal ownership is also important in this regard, with HTA responsibility moving from the local country teams to the global Market Access and HEOR functions.
5. What global patterns may European companies be missing?
A growing share of assets entering Europe are licensed from China and the wider APAC region, and their trial data often do not reflect European standards of care. That gap surfaces quickly in JCA.
The pivotal trials behind many of these assets were largely run in Asia, with comparators and standards of care that do not always match European practice. Early JCA reports have already shown how closely assessors look at evidence from outside Europe. Companies in-licensing these assets should check whether the data will work for JCA as part of due diligence, not after the deal closes.
The US is also more advanced in using real-world data after launch and in outcomes-based agreements. In the US, payment for expensive therapies is increasingly tied to outcomes measured after launch. Rebates linked to outcomes and pay-over-time models for gene therapies, and the CMS Cell and Gene Therapy Access Model applies this approach to sickle cell disease across multiple states. The lesson for Europe is to plan post-launch data collection early, so evidence can support both reassessment and outcomes-based agreements.
6. How is Ingenious e-Brain adapting its own approach, including the use of AI, to what you heard at the summit?
After attending a few summits this year, our focus is on helping clients prepare for JCA earlier in development. That means moving PICO prediction, comparator mapping, and evidence gap analysis upstream. We use AI to speed up evidence screening and synthesis, and every output is reviewed by subject matter experts (SMEs) so it meets the standard HTA reviewers’ expectations.
Closing Perspective
The future of market access will be shaped not by the volume of Real World Evidence generated, but by how early it is planned, how rigorously it is validated, and how well it answers the questions HTA bodies and payers actually ask.
