Recently back from the beautiful city of Copenhagen, it was an honour to see some of the brightest minds in healthcare economics gather for another influential ISPOR conference.

This was the 26th annual European Congress for ISPOR, where GlobalData had the opportunity to share some of our key insights across a variety of subject matters, with poster presentations ranging from early access price trends in markets such as Australia, France, and the UK to various health technology assessment (HTA) archetype analysis and how inflation impacts oncology drugs in the US.

What became evident during the conference was the clear theme presented to attendees and described as “the nexus of policy and science.” As developers prepare for the next step in healthcare policy, topics such as the upcoming joint clinical assessments/EuHTAs were of main focus alongside a drive for greater use of real-world evidence in HTA decisions, and what to expect from the rise of AI and the part it can play in HTA evaluations.

Is the EU prepared for joint clinical assessment (JCA) and what countries have the most to gain?

The EU-wide joint clinical assessments are to apply from January 2025, starting with oncology and ATMPs, and it became clear from ISPOR how vital PICOs (Population, Intervention, Comparator[s], Outcome[s]) will be to the process by ultimately determining the size of evidence submitted, assessed, and reported. The goal is to minimise the number of possible PICOs to meet each country’s requirements while considering the comparators used in different markets. There is however still fear and uncertainty from developers that stems from concerns over possible lack of engagement and detail regarding the process, variation in submission across the EU, and the potential risk for duplication of work. These considerations bring another question: how will timelines be impacted when it comes time to make an HTA decision across various countries, and what does this mean for market access?

According to POLI, the average time for HTA decisions in Germany and Italy is currently 317 and 557 days, respectively.

For Germany, it is acknowledged that implementation of EU-wide HTA decisions will likely add another stream of similar activities to what already exists for the country, but it is believed the country will largely remain unaffected in terms of market access since the country was one of the key players in developing the joint assessments. From conversations at ISPOR, Germany already exceeds what is required from the new HTA process and considered that the new legislation would not likely impact decision-making in the country. However, this extra step may delay HTA decisions.

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In Italy, it is unclear how the JCAs will fit into AIFA’s committee, but ultimately, it is expected that time to HTA would improve and potentially speed up time to a first positive HTA decision, as Italy currently has one of the longest periods for time to first positive decision (709 days) in the 5EU markets (France, Germany, Italy, Spain, UK). Potentially the EuHTAs/JCA could significantly simplify the work based on pre-agreed PICOs, leaving more room for price negotiation. Other countries however may struggle due to limited local capacity to enhance HTA procedures alongside their possible HTA process, budgetary, or workforce constraints. Possible countries that therefore may struggle are Lithuania and Greece, as well as potentially Croatia, Poland, and Slovakia due to an already high proportion of negative outcomes.

What part can real-world evidence (RWE) and AI play when it comes to HTA decisions?

Another theme that was prevalent at ISPOR 2023 was the role RWE has in HTA decisions. This question includes the EMA’s current and planned guidance for real-world evidence in HTA outcomes and its associated real-world impact. Knowledge gaps still exist and are triggering research questions that can be aided by the use of real-world evidence.

There is room for greater conversation with regulators while also opening up conversations earlier. While official guidance was published two years ago that set the standard for the use of RWE, further work is being done that was highlighted at ISPOR, with a pipeline that includes elements such as reflection papers on using RWE for non-interventional studies, greater consideration towards comparative studies and interpretations, a possibility for having five dimensions of data quality, while also advocating for a published catalogue of data sources with descriptions of each database to promote data and study discoverability.

Some benefits of this would for example mean more evidence throughout the lifecycle of a drug and help in better representing certain races in clinical trials. While still in its infancy, it is obvious by just the volume of presentations given at ISPOR on artificial intelligence and machine learning (AI/ML) that there is a recognised value and a place for AI/ML in HTA and health economics and outcomes research (HEOR). When employed, it has the potential to address hurdles in predicting clinical and treatment outcomes, particularly for orphan diseases. Agencies such as NICE have started developing standards, validation, and evidence requirements that can be used in NICE submissions and help NICE appraisal but with guardrails to address concerns such as bias that come with AI/ML.

It truly feels like developers are currently sitting at an inflection point with that connecting “nexus” being the changes to come in key policy areas such as joint clinical assessments, real-world evidence, and AI, and each brings its respective benefits and setbacks for different markets.