Introduction
Given the increasing importance of achieving favourable pricing and market access to a product’s commercial success, it is critical that a pharmaceutical company designs its pivotal study to satisfy the evidence requirements of the payers as well as the regulators. To achieve regulatory approval a product must demonstrate clinical efficacy, usually in a placebo-controlled study, and an acceptable adverse events profile. However, payers typically have additional questions beyond this:
- Does the proposed trial design reflect clinical practice?
- Has the drug demonstrated an incremental benefit over existing treatment options?
- Does the drug offer an additional benefit in certain subgroups of patients?
- Is the trial long enough and have sufficient patients been recruited to be certain of the outcomes?
Engagement
Engaging in dialogue with payers and health technology assessment (HTA) bodies at an early stage is crucial to ensure that a proposed trial design satisfies these additional payer requirements. If this step is taken too late or not taken at all, a company may find that they do not have the necessary evidence to support a favourable price, or that significant market access restrictions are placed on their drug. As such, early dialogue with HTA bodies should form a key part of a company’s pricing and market access launch strategy.
There are multiple ways to engage with HTA bodies, each with differing benefits, drawbacks, costs and complexities. In the EU this includes:
- Meetings with individual national HTA bodies (e.g. NICE in the UK, the G-BA in Germany, and the TLV in Sweden)
- EUnetHTA’s Multi-HTA Early Dialogue, which provides simultaneous feedback from multiple HTA bodies
- The EUnetHTA / EMA Parallel Consultation, which allows drug manufacturers to obtain advice from both the EMA and HTA bodies at the same time. You can find further information on this process here
Processes
The level of product and therapy area uncertainty can drive the choice of which process is most appropriate. We explored this topic further in a poster presented at ISPOR in 2016, which you can find here.
Both NICE and the G-BA have highly structured procedures in place that provide manufacturers with valuable country-specific insights. The G-BA can provide advice on several aspects of their benefit assessment process, including:
- The documents required and procedural steps
- The most appropriate comparator
- The relevance of the clinical endpoints
- The study population and patient subgroups.
NICE’s Office of Market Access (OMA) provides a safe harbour environment that helps companies to understand NICE’s methods, processes and evidence requirements, and allows discussion of managed access approaches for drugs with immature evidence. Formal early dialogue with NICE provides manufacturers with detailed feedback from NICE-appointed experts on their clinical and economic evidence generation plans, helping to integrate cost-effectiveness considerations into these plans.
The Multi-HTA and Parallel Consultation processes are designed to provide applicants with a more streamlined procedure for obtaining feedback from multiple HTA bodies. They also encourage greater collaboration and alignment between stakeholders through a more structured interaction.
Remap Consulting has significant expertise in guiding companies through both EUnetHTA / EMA Parallel Consultation process, as well as national HTA scientific advice processes. We can help in identifying the most relevant HTA bodies to approach, develop the strategy and briefing book to ensure answers to your most pressing issues at the face-to-face meeting. Finally, we can guide you on how to implement the advice, and help you understand the implications for your clinical development program and product launch strategy.
Read our other blogs in the educational series here
We always welcome your thoughts and opinions on the topics raised here. Do contact us (Paul@remapconsulting.com, Tel: +44 7957 028493; Graham@remapconsulting.com, Tel: +44 7415 946778) or share your thoughts with us on LinkedIn (www.linkedin.com/company/remap-consulting), where you can also find the latest news.