Back in December 2021, we summarised the key trends our market access experts believed would dominate 2022.
As this year comes to a close, we’ve reviewed our predictions and examined the impact these trends had on the European pricing and market access landscape the year unfolded.
To recap, we suggested the following trends would shape the pricing & market access industry in 2022:
- The rapid development of a joint EU HTA. What does this mean?
- The Expansion of Cross Border Collaboration for Competitive Pricing Negotiations and Accelerated Access of High-Cost Therapies
- Effective P & R Processes to Ensure the Future of Gene Therapies in the EU
- EU Net Price Disclosure: Industry Verses Payers, But Who Will Come Out on Top?
- Continued and Increasing use of Post-Marketing Evidence Collation to Justify Value in Market Access
The rapid development of a joint EU HTA. What does this mean?
In our original article, we had predicted that the EU HTA Regulation would come into force in Q1 2022 following a plenary vote in December 2021, that the Coordination group would be set up and that draft guidance would begin to be issued.
All these expectations have come to fruition meaning that the processes are currently being moulded into something tangible rather than a far off, conceptual pipedream which it has been for so long. Following the appointment of the coordination group (HTACG) they have been working on establishing how the EU HTA process will function determining the methodology and processes for assessing the evidence including how to define PICO, comparators, and endpoints for the assessment, of which some guidance and templates have already been published. Further information on this is anticipated once the stakeholder group is also set up in the near future. However, there has been controversy from European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) and European Federation of Pharmaceutical Industries and Associations (EFPIA) – read our summary of it here – over whether the current methodologies truly satisfy the needs for all the member states to make pricing and reimbursement decisions. Some are worried that on a national level there may be duplication of work if the parameters of the joint assessment don’t confer to those of the member states.
Going into 2023, we predict that we will see some of these concerns beginning to be ironed out which may lead to countries building frameworks and writing legislation ready for the application of the Regulation in 2025. We also expect for further guidance to be issued specifically on the joint scientific consultation for which there are still several documents left unpublished.
Improved assessment and pricing initiatives through cross border collaboration
In our article at the start of the year, we predicted that the trend of increased trend of cross-border HTA collaboration initiatives, and the resulting number of joint HTA’s, would rise in number. This was primarily driven by the desire to pool resources and accelerate patient access. BeNeLuxA have been busy completing the pharmacotherapeutic, pharmacoeconomic and budgetary assessment for Libmeldy (atidarsagene autotemcel) in Ireland, Belgium and the Netherlands. The reimbursement submission was submitted in December 2021, with the development of the scientific and economic report announced in October 2022. In the words of BeNeLuxA, this will “facilitate agreement on reimbursement terms and be the basis for joint negotiations should the company and the Beneluxa Initiative decide to enter into such a process”. In comparison, the assessment of Zolgensma (Onasemnogene abeparvovec) took one year to complete between May 2020 and May 2021, with pricing negotiations completed in October 2021. One promising area of collaboration is the recently announced initiative between organisations in nine countries (Canada, Denmark, Norway, Iceland, Ireland, Netherlands, Portugal, Belgium, Sweden) regarding knowledge exchange in procurement, negotiations and expensive drugs with a lack of evidence on effectiveness.
No news of pilot joint assessments or pricing negotiations have been reported by the Visegrád Group (Poland, Hungary, Slovakia and the Czech Republic) or the Valletta Declaration (Croatia, Cyprus, Greece, Ireland, Italy, Malta, Portugal, Romania, Slovenia), as suggested. With the spectre of EU HTA looming large over European market access, and some (currently) isolated calls for joint EU procurement schemes, it is perhaps not surprising that such initiatives are yet to get off the ground.
Effective P & R Processes to Ensure the Future of Gene Therapies in the EU
Multi-stakeholder collaboration was fundamental to the development of the pricing & reimbursement (P & R) landscape of Advanced Therapy Medicinal Products (ATMPs) in 2022. This collaborative action saw great momentum being made in optimising and aligning on assessment procedures and requirements to increase likelihood of ATMPs meeting regulatory and health technology assessment (HTA) requirements. In September, the European Medicines Agency (EMA) launched a pilot project to assess what further support or regulatory tools are required to increase access for patients to ATMPs. The project will support the EMA with its ongoing work with the Joint European HTA body to establish a more integrated approach between the two processes to support greater access to patients for these novel treatments.
Slower progress has been made with respect to implementing payment models that can increase the affordability of ATMPs. As of mid-2022 there were more than 2,000 gene therapies in development worldwide targeting broad therapeutic areas from oncology to infectious disease. It seems surprising, that in a year that has seen the scope of ATMPs broadening to non-rare diseases, greater effort has not been focused on generating innovative payment models to help mitigate payer uncertainties associated with high costs and limited clinical data packages of these novel therapies.
EU Net Price Disclosure: Industry Verses Payers, But WHO Will Come Out On Top?
Our predicted trend for 2022 was that the WHO would be the vanguard of an increased political push towards transparent net prices (as outlined in the Oslo Medicines Initiative), with inevitable opposition by the pharmaceutical industry. The primary motivation from a payer perspective is a reduction in the cost of medicines, and a subsequent increase in patient access to medicines. The biggest event over the past year is the announcement of a multi-stakeholder, ‘neutral’ platform between member states of the WHO European regional and relevant stakeholders (including industry representatives) as a method to foster discussions and “jointly identify and agree on potential solutions for equitable access to medicine”. The move to create the neutral platform was outlined in the technical report published by WHO in September 2022.
Interestingly, the document also highlighted three other areas for further exploration:
- Pooled procurement and other collaboration initiatives, and good practices identified
- Creation of a framework for differential or equity-based tiered pricing
- The commitment to more appropriate use of external reference pricing for novel medicines, and impact on parallel importation
How has this gone down with the pharmaceutical industry? While it is still early days, there seems to be cautious optimism that a collaborative approach could work for both parties. For example, the EFPIA published a statement saying that the move is ‘welcomed’, but recognising that any possible net price solutions should look to balance both the sustainability of European healthcare systems and the pharmaceutical industry.
Continued and Increasing use of Post-Marketing Evidence Collation to Justify Value in Market Access
Finally, when gazing into our crystal ball for 2022 we predicted that the requests from HTA agencies for post-marketing evidence collation would increase, in order to better inform initial price and access discussions or reassessments. This would potentially be mirrored by other HTA agencies replicating the approach pioneered by NICE, HAS and the G-BA. Currently, only Zolgensma sits on the G-BA’s list of drugs that have a requested RWE data collection requirement following Early Benefit Assessment. However, that will no longer be the case. On the 3rd November 2022, it was announced that the G-BA was requesting real world evidence collection for fedratinib, an oral JAK2 inhibitor for the treatment of myelofibrosis that was previously assessed to have a non-quantifiable additional benefit. The RWE collection should be specifically designed to allow comparison against ruxolitinib (which more accurately reflects the German standard of care) and address other uncertainties (e.g. meaningful data for overall survival), beginning in mid-2023. Such requests for supportive RWE are not appropriate for every drug: in May 2022, the G-BA announced it would not request RWE for the somatic cell therapy Tabelecleucel, despite the likely need to address uncertainty, due to the difficulty in implanting a national trial.
While NICE published their RWE framework in June 2022, perhaps the more eye-catching news regarding post-marketing evidence collection was the announcement of the Innovation Medicines Fund in the same month. The IMF will essentially act as the Cancer Drugs Fund, but for non-oncology medicines. More information on the fund can be found here.
The potential impact of real-world evidence (RWE) to aid in evaluation of clinical benefit for ATMPs is continuing to evolve. The DARWIN (Data Analytics and Real-World Interrogation Network)-EU initiative announced expected delivery of over 150 studies by 2025 to support HTA decision-making, which is hoped can be a valuable tool to provide post-approval data.
Conclusion
While our predictions for 2022 were fairly accurate, the rapidly approaching deadline for the implementation of joint EU HTA seemed to overshadow the rest of our trends. Looking forward, the impact of joint EU HTA is likely to be even more disruptive in 2023. To learn more about joint EU HTA, and how it could shake up the European pharmaceutical industry, join our webinar on 1st December by signing up on the link here.