Principles and benefits of HTA early scientific advice
Uncertainty and inappropriate trial design are key reasons why medicines may fail to achieve optimal outcomes in health technology assessment (HTA). Scientific advice from HTA agencies can identify areas of uncertainty regarding payer evidence requirements, and when implemented can result in stronger HTA submissions.
In this update, we summarize the key principles and benefits of seeking early scientific advice (ESA), specifically the ESA processes of NICE, CADTH, HAS, and the G-BA. Although each agency’s approach differs, the underlying principles and benefits of ESA are the same.
ESA offers applicants the opportunity to put specific questions to the agency, in order to approach the clinical development process in a way that aligns with the agency’s expectations.
In all instances, ESA engagement with regulatory or HTA agencies is confidential and non-binding; applicants may choose not to act on the outcomes of their discussion.
Furthermore, ESA is not an early assessment, nor are pricing discussions permitted. The advice and recommendations received are not an indication of future appraisal outcomes.
In addition to individual agency consultation, joint scientific advice processes are available. Joint scientific advice involves collaborative ESA provided by two agencies simultaneously and is a time- and cost-efficient way to seek advice from multiple HTA agencies, or an HTA agency alongside a regulatory agency.
Regardless of the type of advice sought or agency consulted, a briefing book outlining the manufacturer’s position and questions for discussion with the agencies must be developed and submitted. Most agencies also offer a period of time after a face-to-face or virtual consultation in which clarifications of final advice can be requested.
Early scientific advice helps enable discussions with HTA agencies
When implemented, ESA recommendations routinely result in stronger HTA submissions by reducing payer uncertainty, which often leads to suboptimal HTA outcomes.
A number of common topics may be discussed through ESA, including:
1. Optimizing the pivotal trial design Input on trial design can help manufacturers optimize their development process by identifying key recommendations for aspects such as endpoints, enrollment, and comparator choice.
2. Understanding payers’ expectations Awareness of the payer’s perspective at an early stage can strengthen strategic plans, enabling manufacturers to tailor a submission dossier that is relevant for each market, facilitating local payer understanding and expectations of the disease area, therapy, or clinical practice.
3. Developing evidence that demonstrates additional value Engaging with payers on supplementary evidence needs allows manufacturers to strengthen supporting arguments and generate a comprehensive evidence package; for example, the approach to generating an external control arm in support of a registrational single-arm trial.
4. Integrating cost-effectiveness considerations into HTA submissions Validating the cost-effectiveness model structure, inputs, and assumptions with input from HTA agencies can reduce uncertainty in manufacturers’ economic analyses that may otherwise result in unacceptable ICERs.
Understanding the concept of early scientific advice and its purpose across HTA markets
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