Principles of HTA Early Scientific Advice for market access

In this, the first of his 2-part series for market access professionals seeking to understand the key principles and benefits of Health Technology Assessment (HTA) Early Scientific Advice, Harrison Davis explores:

• What is scientific advice? 

• How do Early Scientific Advice processes and discussion topics vary between HTA agencies?

• What underlying principles do HTA agencies commonly follow? 

Harrison is a Senior Consultant at PRMA Consulting, part of Fishawack Health, and has worked on a number of successful projects relating to HTA Early Scientific Advice. 

What is scientific advice? 

In essence, scientific advice describes the engagement with regulatory or HTA agencies in a structured process that allows companies to discuss and seek advice on their clinical development process for a particular asset, or in some cases, medical devices or diagnostics. You may also see references to early dialog or consultations as different agencies have their own terminology. 

Depending on your specific development questions, different types of scientific advice may be relevant.

• Regulatory Scientific Advice: Manufacturers may seek national regulatory advice; FDA EMA, Health Canada, TGA, and MHRA all have their own national scientific advice services, and within those there exist some specialized pathways, such as protocol assistance specifically for developers of orphan medicines and parallel assistance between the EMA and FDA.
  
  
• HTA Scientific Advice: Some HTA agencies offer individual national HTA advice and can be consulted on a 1:1 basis. I summarize the scientific advice processes of NICE, the G-BA, HAS, and CADTH in the second part of this series.
  
  
• Joint Scientific Advice: Several pathways exist for agencies to collaborate on or share scientific advice simultaneously, and provides a time and cost-efficient‑ approach to seeking advice from multiple agencies. In England and Canada, national HTA and regulatory joint advice can be sought. NICE and CADTH have formal processes for joint HTA advice and, despite major updates to their processes, EUnetHTA still presently offers multi-HTA early dialog with EU member state HTA bodies.
  
  
• Early Scientific Advice: A number of these agencies do offer advice at any stage of development, such as regulatory post-authorization safety study advice, to improve the design of studies for collection of further data on safety once the product is on the market, or NICE’s Preliminary Independent Model Advice, which can also be post-authorization.

In my work within Policy, Access, Value, and Evidence at PRMA Consulting, I am noticing that our clients are predominantly concerned with HTA Scientific Advice and most HTA Scientific Advice, as we will explore shortly, is early in clinical development, where the opportunity to shape the design of pivotal trials is still open.

Early Scientific Advice processes and discussion topics vary between agencies but follow common underlying principles

Scientific advice is a series of formal and structured processes that are offered by a number of agencies, chosen depending on the purpose and scope. But despite the number of different agencies and pathways, there are common underlying principles for them all.

Why seek Early Scientific Advice from HTA agencies?

Advice is sought to mitigate future HTA or regulatory challenges; importantly, given its commercial sensitivity, the advice is confidential.

Scientific advice is also non-binding, meaning that the manufacturer is not obliged to follow the advice received. We may see this where it conflicts with the manufacturers’ commercial aspirations.

Types of Early Scientific Advice

Whether the advice sought is via 1:1 agency consultation, or Joint Scientific Advice between multiple agencies, all are formal fee-based services with application lead‑times to consider, so understanding exactly which type of ESA and with which agency it is required saves the manufacturers time.

What does Early Scientific Advice not include?

The limitations on what advice can be sought does vary from agency to agency, even of the same type; that is, not all HTA agencies will provide advice on the same issues. However, common to all is the exclusion of pricing discussion.

Furthermore, advice is not an indication of HTA or regulatory outcomes; implementation of the advice does not guarantee approval or reimbursement but seeks to remove barriers that may otherwise challenge it.

Key steps for gaining Early Scientific Advice

Finally, the processes all include submission of a briefing book that outlines the company’s position and questions for discussion with the agency or agencies. These are reviewed by agency members ahead of a face-to-face or virtual consultation meeting, after which the agency formalizes their advice in a response, often with the opportunity for the company to request clarification of final advice.

Benefits of utilizing Early Scientific Advice within market access

In the second update in this series, I summarize the benefits of utilizing HTA Early Scientific Advice within early development planning. Continue reading…

For a deeper dive into the benefits and key considerations of leveraging HTA Early Scientific Advice, you can watch our on-demand webinar:

This free resource provides best practice for prioritization and feasibility assessment of assets for HTA Scientific Advice in key markets. Viewers will also gain practical recommendations for preparation and implementation of HTA Scientific Advice meetings that will increase the value of your consultation.