Pharmaceutical Market Europe • March 2022 • 9
THOUGHT LEADER
Health technology assessments: understanding the global landscape and considerations for drug development
By Manpreet Sidhu and Ben Cieply
Health technology assessments (HTA) inform national decisions for market access and reimbursement of new pharmacotherapies; countries across Europe have well-established processes that determine the clinical and economic value of new treatments. A metric that is employed ubiquitously across HTAs is the relative medical advancement over the standard of care. This remains the primary determinant for access and price decisions in some countries.
Other markets combine the relative medical advancement with the incremental increase in cost into one assessment through pharmacoeconomic modelling. The National Institute for Health and Care Excellence (NICE) in the UK has long used cost-effectiveness assessments as the centre of its HTA, price and reimbursement process. Cost-effectiveness assessments have more recently been implemented in France as part of the Haute Autorité de Santé’s (HAS) evaluation of highly innovative treatments, and payers in Spain are considering incorporating more economic analysis in their HTA reviews.
Manpreet Sidhu
Ben Cieply
Outside Europe, countries like Australia, Canada and Japan have established HTAs. Each country includes pharmacoeconomic modelling in its evaluations. Across the globe, HTA’s role will continue to expand as centralised payer systems evolve. These HTAs will most likely mirror one or more of the well-established frameworks employed across Europe. While the US system remains largely fragmented across private payers, the emergence of the Institute for Clinical and Economic Review (ICER) has introduced an element of HTA that many US payers are taking into consideration.
Historically, pharmaceutical companies have prioritised market access initiatives in the US and Europe due to the global revenue represented by these regions. While North America, driven by the US market, still represents more than 50% of drug revenue, the sum of ‘rest-of-world’ has reached a level comparable to Europe’s. Accordingly, companies are broadening their early health economics and outcomes research (HEOR), pricing and market access research initiatives and investment on a global scale.
Despite sharing the common goal to quantify new therapies’ value, many nuances exist between HTA and payer systems. The best example is the quality-of-life adjusted life year (QALY) based assessment, which is central to the assessments in UK and Australia. In contrast, QALY based assessment is not relied upon for national HTA decisions in Germany and has varying degrees of influence in other markets.
Other value drivers that vary by country include a drug’s impact from a societal perspective or on work productivity – not all country frameworks incorporate these. Drug value assessment timelines can differ tremendously in cost-effectiveness markets relative to others. Overall, expectations and requirements around endpoints and comparators must be considered on a country-by-country basis. For example, some payers will be hesitant to reimburse new agents based on surrogate endpoints alone and/or they may or may not consider ‘indirect comparisons’ if the standard of care was not included in the clinical trial. The HTA comparator may also set a price benchmark, which can limit the achievable price for a new product – especially if it’s a generic.
This list of country-level nuances only scratches the surface. Still, it illustrates the need for sponsors to consider pricing and market access implications early and often. Most sponsors are already following through with this, illustrated by three select examples:
Early asset evaluation
Sponsors often evaluate the competitive environment, efficacy thresholds for differentiation and value, and achievable price ranges at early clinical stages. Early evaluation of market potential is especially essential when multiple indications are being considered.
This may include evaluation of the treatment and payer landscape through secondary research, systematic literature review and primary payer/clinician research.
Clinical trial design
When feasible, phase 3 trials should be optimised to meet payer expectations. Gaining payer insights on high-priority patient populations, endpoints, comparators and duration can be gathered through primary research and/or formal early HTA advice. This step allows sponsors the opportunity to make changes to their clinical trial protocol, ensuring trials generate payer-relevant data. Even when recommendations from early HTA advice cannot be feasibly incorporated into the trial, developing an evidence generation plan can mitigate these challenges with payers, if they are identified early enough.
Early economic model
Efficacy thresholds from an initial target product profile (TPP) can be used to develop an economic model to understand the implications of different efficacy scenarios. This will provide insights around the price ranges that will be considered cost-effective by payers who use pharmacoeconomic assessments.
Manufacturers benefit from understanding the market-specific HTA landscape. They can identify and quantify opportunities and challenges early on, establish value drivers for their asset and generate evidence to capitalise on their trial programme. With their findings, manufacturers can develop a formidable strategy from evidence generation to implementation.
Manpreet Sidhu is Executive Principal, Global Head, Global Health Economics, Outcomes Research and Epidemiology and Ben Cieply is Lead Consultant, Global Pricing, Market Access and Reimbursement, both at ICON