Biotechnology companies often do not have the market access resources comparable to large pharma. While this can pose a challenge, there is a considerable opportunity for biotechs to reduce this disadvantage by using health technology assessment (HTA) data in innovative ways to inform and drive their market access decision-making.
With our innovative technology platform and focus on data quality, we have been helping biotech companies understand the market access space for bellwether HTA agencies for the past seven years. Throughout our experience with biotechnology companies, we continually see four assumptions about the HTA process and market access:
- The data collected and submitted for regulatory approval will be sufficient for HTA and reimbursement.
- Just like in the US, market access in other countries happens rapidly after regulatory approval.
- Global market access planning begins after phase III clinical trials.
- If my clinical results are great, I don't have to worry about cost.
In this four-part series, we will assess each of these assumptions and, through data-driven analysis, reveal how each is a misconception that may be derailing the market access process.
Part 1: All Reviews are Created Equal
Traditionally, the drug development process has focused on gaining regulatory approval and the pivotal clinical trials form the foundation of this approval. But are these evidence sources sufficient for HTA and reimbursement?
We took a data-driven approach and compared the methodology of regulatory agencies and HTA agencies to determine how much overlap exists. We compared the preferences and requirements of the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to those of seven bellwether HTA agencies—NICE, SMC, G-BA/IQWiG, HAS, CADTH, and PBAC—on the key dimensions of:
- Clinical trial study design
- Clinical trial outcomes / endpoints
- Quality of life (QoL)
- Utilities / cost-effectiveness
Regulatory agencies and HTA agencies have different objectives in evaluating the clinical evidence base. This is not surprising since they have different priorities—regulatory agencies are focused on the efficacy and safety of the product, while HTA agencies are focused on the clinical effectiveness and, in some cases, the cost-effectiveness of a product.
|Study Design||Adequate, well-controlled studies vs. placebo or active comparator||Randomized controlled trials vs. standard of care|
|Comparators||Placebo or active comparator||Standard of care|
|Outcomes / Endpoints|| – Clinically meaningful (dependent upon disease condition
– Surrogate end points are acceptable
| – Final endpoints / patient-relevant endpoints
– Surrogate endpoints are not acceptable
|Quality of Life (QoL)||Not required||Required for some agencies|
|Not required||Required for all agencies except G-BA / IQWiG|
The slight differences in the regulatory and reimbursement evidence requirements reflect these goals. The regulatory agencies are willing to accept more types of clinical trial study designs (if justified) and are less restrictive in the comparators required in order to demonstrate efficacy and safety. In contrast, HTA agencies are more concerned with evaluating high-quality evidence of the drug against the "correct comparator," or the country's standard of care. HTA agencies differ in their endpoint preferences compared to regulatory agencies; regulatory agencies are more willing to accept surrogate endpoints than HTA agencies and HTA agencies are more likely to request or require QoL data.
Because regulatory and HTA agencies have different objectives, it is intuitive that their evidence requirements differ. Further, as outlined in the table above, the dimensions and relative values on which agencies evaluate drugs differ. Drug development is a very time-sensitive and costly endeavor during which manufacturers, especially in biotechnology, must continually balance the needs and costs associated with bringing a drug to market. Therefore, considering specific and targeted strategies for each reimbursement and regulatory agency is the most efficient and cost-effective approach to gaining optimal market access.
Stay tuned for part two of this series where we will examine the relative speed of market access after regulatory approval.