Back in December 2020, we summarised the key trends our market access experts believed would dominate 2021.
As this year comes to a close, we’ve reviewed our predictions and examined the impact of these trends as the year unfolded.
To recap, we suggested the following trends would shape the pricing & market access industry in 2021:
- The pandemic causing HTA delays that would result in drugs in “critical” therapy areas (e.g. oncology) being prioritised
- The consequences of Brexit causing regulatory delays in the UK
- The implementation of innovative payment models to counter the rise of extremely expensive one-off gene therapies
- A growing desire for European collaboration in pricing and reimbursement
COVID-19 pandemic delays for Health Technology Assessments (HTA)
In our original article, we reported that while Germany had maintained their output of HTA publications in March-June 2020, France and the UK had reduced the number of HTA publications, 30% and 31% respectively, compared to the 2015-19 average in the same months. On the back of this, we speculated that the 2020 backlog would likely slow the market access of non-critical drugs in 2021.
Our own analysis suggests that NICE have been busy tackling this backlog. Looking at the absolute number of Single Technology Appraisals (STAs) published in 2021 (64) between January and December, this is more than the 39 that were published in 2020, and more than the 45 that were published in 2019. Additionally, when we look at the percentage of those publications looking at typical “priority” disease areas such as oncology, the percentage is relatively similar. Oncology assessments made up 53% (24/45), 63% (25/39) and 53% (34/64) of all published STAs in 2019, 2020 and 2021 respectively. Obviously, while there will be other factors in play, these numbers do not suggest that NICE are focusing only on oncology medicines deemed critical within this “bounce-back” year.
Clearly significant effort has gone into clearing the backlog, but HTA agencies will be under increasing pressure to keep up with the continuing need for product appraisals, as well as sticking to pandemic working practices that were initially temporary (e.g. virtual committee meetings)
Based off the analysis above, and the lack of reported HTA delays in France and Germany, it would seem that pandemic HTA delays were not a significant impact in 2021.
Consequences of Brexit causing regulatory delays in the UK
Given the divergence of the MHRA from the EMA, we predicted that the UK may lose status as an early launch country, with NHS patients facing delayed access to innovative medicines, if the MHRA required a separate dossier to be submitted.
2021 was a stop gap year for MHRA approvals. Since 1st January 2021, the UK will continue to adopt EMA regulatory decisions until 2023. Previous guidance published on the Accelerated Access Procedure and the Rolling Review procedure has not been updated since early January 2021, suggesting that this is still an area under development. Further guidance will be required soon for those small pharma companies and biotech’s who will need to decide on the prioritisation of resources.
Given the decision to continue the adoption of EMA regulatory decisions until 2023, we have currently not seen the impact of this trend happening in practice.
Affordability of cell and gene therapies
Last year, the extremely high price of Zolgensma (Onasemnogene abeparvovec) captured headlines across the world and raised further discussion on the price of one-off cell therapies. While we speculated that these therapies could lead to a boom in innovative pricing models, Zolgensma provides an interesting case study that the current funding approaches agreed in 2021 are still relatively routine.
In England, the basis of the approval of Zolgensma was a managed access agreement (MAA), including a confidential patient access scheme (PAS), with a discount from the list price that “ensure(s) a price that is fair to taxpayers”. In contrast, AIFA (Italy) reimbursed Zolgensma with both discounts on the ex-factory price and payment-by-result at fixed 12-month periods (12,24,36 and 48 months). Additionally, a recent press release by the Spanish Ministry of Health has announced that Zolgensma will be reimbursed via a managed entry agreement that utilises both a price-volume deal and performance-based milestone payments.
This mixture of approaches, but combining discounts, further evidence generation and performance-based milestones, suggests that European payers have not embraced new funding models that have been seen in the US. For now, the framework for the reimbursement of future cell therapies is well-defined.
Joint EU HTA procedure
In our original article, we had concluded that joint EU HTA, a long-term goal of the EU to align the HTA processes of all member states into a single process, was still a distant pipe dream. The joint EU HTA has always been troubled by significant country resistance, particularly led by France and Germany over several key issues (e.g., whether recommendations should be mandatory).
However, a big jump forward has been made with the announcement of the EU HTA regulation. Over the next decade, the Regulation will make it compulsory for manufacturers to submit a European-level HTA dossier for joint clinical assessment. A clear roadmap has been set out, with key stakeholders designated (e.g., EUNetHTA21 responsible for developing advanced HTA methodology) and obligations for member states.
While we had initially predicted that this joint EU HTA would continue to be significantly delayed, the developments over 2021 have surprised the industry. The impact of joint EU HTA was not seen this year, but with a plenary vote expected in December 2021 (and entry of the Regulation into force by Q1 2022), a massive step forward in the European joint HTA process is expected in the next decade with significant ramifications expected for manufacturers.