Marc Garcia and Michael Burnill from Research Partnership’s Market Access team attended the 2023 World Evidence, Pricing and Access conference in Amsterdam. With over 1,200 delegates and 300 speakers present, the congress remains the largest of its kind in Europe. In addition to the core topics of evidence, pricing and access, the three-day event offered in-depth insights into a range of specialist areas including, but not limited to, Advanced Therapy Medicinal Products, Healthcare Technology Assessment and Patient Reported Outcomes. In this write-up, Marc and Michael list their three key takeaways.
1. Patient perspective on payers’ minds
Day one of the conference kicked off with an insightful discussion regarding the patient perspective. It goes without saying that the patient experience is fundamental to our endeavors within the field of Market Access – yet their perspective can often be neglected or overlooked and there is a need for payers to understand the patient journey to ensure it’s taken into their decision making. To help correct this, speaker Christoph Glaetzer, Chief Value and Access Officer at Janssen, suggested manufacturers should identify clinical unmet need that is meaningful for patients and develop wider insights on the patient journey (e.g., behavioral, clinical, or long-term care management), ensuring the patient voice is taken into consideration in overall decision making. Beyond the patients themselves, Christoph suggested considering wider stakeholders. Similarly overlooked is the complete view of the caregiver who themselves often dedicates a lot of their time to the patient and is fully aware of patient’s needs.
In rare disease areas, where HTA agencies are removing the ‘privileges’ that drugs in these areas used to have, such as accepting less data due to the high complexity of conducting clinical trials, it becomes important to not only understand HTA basis of each country but also what payers are keen to see in addition to randomized control trials (RCTs). Patient Reported Outcomes (PROs) are perceived as ‘add-on’ information but, as Simon Shohet from Amicus Therapeutics in a separate presentation highlighted, earlier payer research would provide useful insights on which PROs are likely to be considered by HTA bodies. A pre-established trial with specific PROs (in contrast with generic measurements such as EQ-5D) has the potential to drive the appraisal into a successful reimbursement.
2. Advanced therapies challenges and experience
Another key theme at the congress was the market for Advanced Therapy Medicinal Products (ATMPs), which is growing rapidly due to its high technology treatments that offer a personalized and potentially curative treatment for severe diseases such as leukaemia and spinal muscular atrophy. However, a number of speakers at World EPA noted there are several challenges to bringing ATMPs to the market, such as the small patient numbers, uncertain clinical outcomes, and questions relating to durability of effect. In addition, the evaluation of a one-time high-valuable treatment presents an affordability and financing challenge to the system.
Outcome-based agreements (OBA) are a way to overcome some of these challenges and secure reimbursement after the country-specific HTA body assessment. In addition, OBAs help to tackle the challenge by avoiding the upfront payment of some product costs. Lara Pippo from CSL Behring shared specific examples of innovative contract approaches for gene therapies in Italy, where Luxturna and Zolgensma have been reimbursed through an OBA system. Luxturna has a payment-by-results agreement, while Zolgensma agreed a pay-at-performance system where price is based on clinical outcomes achieved. Although payment-at-performance is not applicable anymore in Italy due to an accounting rule of the public administration as current expenses are not depreciable, overall, OBAs have helped to secure access to gene therapies in Italy while maintaining the sustainability of the healthcare system. In addition, the average time for ATMP reimbursement is not so different from standard therapies and in some cases can be shorter due to Early Access Programs, meaning OBA’s have also accelerated the access process.
As we see more ATMPs come to market, it is important manufacturers utilize past experiences to ensure it is given the greatest chance of overcoming challenges and ultimately facilitating reimbursement.
3. RWE and the new HTA
The third hot topic of note at the event was Real World Evidence (RWE), which helps to bridge the gap between clinical trials and real-life value demonstration for stakeholders making access decisions. Each country has its own RWE guidance and recommendation and, as Helene Chevrou-Severac from Alexion Pharmaceutical mentioned in her presentation, there is a great opportunity to set up the structure and governance to create high quality data registries and to converge across countries in common elements of the methodology for RWE collection and analysis looking into the near future with the EUnetHTA.
For example, in Germany, the ambition is to assess suitability of RWE for use in the benefit assessment of interventions when the evidence from trial is insufficient to derive meaningful conclusions. In addition, the G-BA website includes a list of RWD collection requirements with the aim to have comparative data pre-determined in a clinical trial.
In contrast, the ambition in the UK is to use RWD to resolve gaps in knowledge and drive forward access to innovations for patients to inform NICE guidance in the HTA process. The NICE framework recommends including the proposition of new methods to study comparative effectiveness from non-randomized real-world studies and use external control for comparing with single arm trials.
A different approach is taken in France, where HAS tends to prefer the use of RWE in second revision rather than in the first submission and encourages manufacturers to present an external control for a new comparator launched after RCT results or in single arm studies. However, HAS is aiming to bring recommendations to manufacturers to improve the methodology used to collect RWE and get robust and trustable results.
How current differences between country HTA bodies’ approaches to RWE will fit in the future EUnetHTA is still unknown. The EU aims to replace the redundant evaluation of clinical evidence performed by multiple national HTA, and the use of RWE adds complexity to the vision of a common HTA. In 2025, EUnetHTA assessment will become mandatory in oncology and ATMPs, thus planning early payer research to pre-stablish the basis for product launch and post-launch RWE will become key, especially in those disease areas with a small number of patients.
Preparing for this changing paradigm will be essential, and as a reference, we can investigate what similar experiences have happened in recent years. Andrew Olay from Orchard Therapeutics presented previous joint HTA learnings from BeNeLuxa and Finose HTA collaborations. Although it seems obvious, we should be aware that despite relying on a joint HTA report, country reimbursement recommendations and decisions might be different. Within Beneluxa and Finose countries, national processes are generally followed during the joint HTA process; value dossiers are submitted to each country directly and should fulfil country specific requirements. Surprisingly, despite there having been just a few treatments assessed by joint HTA ( 3 in Beneluxa and 1 for FINOSE), the evaluations have generally been completed within 6-8 months, thus reflecting that evaluations may not need to take longer than they currently are. In conclusion, pharmaceutical companies need to be aware of the joint HTA guidelines and take the future EU joint HTA as a platform of reference for product recommendations.
4. What wasn’t mentioned at World EPA?
Interestingly, there were a number of hot topic issues that received very little coverage at the event, specifically Artificial intelligence (AI) in the context of Market Access Market Access departments are facing new challenges which require more accurate and granular data to tackle them. For example, AI could be used as a solution for creating a predictive value pricing model. In order to adjust treatment approval and reimbursement, pharma companies could predict value prices for their treatments based on the analysis of patient outcomes and clinical trials thus maximizing profit and efficiency.
Another aspect where AI could improve MA efficiency is on reimbursement. It is important to understand what causes delays after EMA or FDA approval and AI can help to uncover and correct inefficiencies when drafting HTA submissions using machine learning techniques. In addition, AI can be used to reveal factors that lead previous submissions to be more effective. These insights can then be used to guide further submissions strategies and predict both time-to-market and probability of success for budget planning.
AI could be a useful tool for MA departments and help them to not only optimize strategic planning and become more efficient overall. Therefore, it will be interesting to see how discussions around AI play out at future events. It will also be interesting to see how manufacturers and HTAs implement or indeed combat the rise of AI as it’s true value becomes more apparent.
Networking and sharing experiences
In addition to the array of content detailed above, EPA 2023 granted Marc and Michael the opportunity to network with clients, payers, and providers alike. Attending the event was a great opportunity for Research Partnership’s market access team to catch-up with a variety of both familiar and new faces and explore how best Research Partnership can further strengthen and optimize our diverse offerings.
