Working Groups

The HTAi-DIA Working Group

Health Technology Assessment (HTA) is a multidisciplinary process to determine the value of a health technology at different points in its lifecycle, all in service of promoting an equitable, efficient, and high-quality healthcare system. By the very nature and complexity of health technologies, as well as methodological challenges associated with their assessments, uncertainty will always exist at some level in both regulatory and HTA informed decision-making. This has been underscored by the onset of the global health care crisis associated with COVID-19. The pandemic has disrupted attitudes, structures and processes – collectively leading to increased levels of uncertainty and complexity. A far more layered, nuanced and flexible response from all stakeholders in the health care ecosystem is required to achieve equity, efficiency and quality health care.

Purpose

With the above in mind, the intent of the HTAi-DIA Working Group is to explore how to manage uncertainty in the regulatory-HTA interface across the health technology lifecycle. Specifically, we seek to create a platform and convene conversations between all key stakeholders in the healthcare system that share a common understanding – not simply information and data – to manage uncertainty in regulatory and HTA decision-making on drugs. The better and more effectively this is done, the greater the benefit for patients and healthcare systems.

It is understood that the processes and methods for exploring and dealing with uncertainty are context and situation specific. Context includes jurisdiction, HTA structures and setting/remits which vary globally, for example regarding established social values or practices in a specific country, at institutional or individual level, or in a specific disease area. In some countries, situations where technologies are considered for children or disadvantaged populations, may also be allowed additional tolerance for greater uncertainty. This is all affected by the appetite for risk within a decision-making organization generally, as well as for the individuals deliberating on its behalf.

During the 2021 HTAi Global Policy Forum, held virtually on February 22-26, 2021, key challenges and opportunities regarding uncertainty in HTA were discussed. Following the discussions, and building on recommendations made during the Forum, HTAi and the Drug Information Association (DIA) initiated a Working Group to provide guidance on management of uncertainty in the regulatory-HTA interface across the drug’s lifecycle.

Members

Wija Oortwijn
Inkatuuli Heikkinen
Milou Hogervorst
Rick Vreman
Indranil Bagchi
Inaki Gutierrez-Ibarluzea
Bettina Ryll
Hans-Georg Eichler
Elena Petelos

Sean Tunis
Claudine Sapede
Wim Goettsch
Rosanne Janssens
Isabelle Huys
Liese Barbier
Deirdre DeJean
Valentina Strammiello
Dimitra Lingri

Melinda Goodall
Magdalini Papadaki
Massoud Toussi
Despina Voulgaraki
Ania Mitan
Sharon Gorman
Ansgar Hebborn
Stefan Sauerland
Ian Watson

Outcomes

Panel Presentation: Deep Dive On Uncertainty – A Multistakeholder Discussion Hosted By The HTAi-DIA Joint Working Group

Panel 13 from the HTAi 2022 Annual Meeting in Utrecht, the Netherlands

Date: June 27, 2022

Panelists:

Abstract: To accelerate access to innovations for patients, HTA bodies, regulators and stakeholders are facing increasingly uncertainty in their decision-making processes. To better understand the complexity of “uncertainty”, HTAi has formed a multistakeholder working group with DIA to advance this discussion.

Watch it on-demand

Panel Presentation at DIA Europe March 2022: Uncertainty Management in Regulatory and Health Technology Assessment Decision-Making – Guidance of the HTAi-DIA Working Group

Date: March 23, 2023

Panelists:

Panel Presentation: Evidence – Interaction – Trust – Confidence

Panel 31 from the HTAi 2023 Annual Meeting in Adelaide, Australia

Date: June 27, 2023

Panelists:

Wija Oortwijn, Netherlands
Iñaki Gutierrez-Ibarluzea, Spain
Hans Peter Dauben, Germany

Abstract: The panel will describe results of the HTAi-DIA working group on uncertainty on how to gain trust and confidence as well as a proposal of how to follow a pathway to increase common action on trustful data as need for future actions in healthcare and health service industry.

Publication: Uncertainty Management In Regulatory And Health Technology Assessment Decision-Making On Drugs: Guidance Of The HTAi-DIA Working Group

Publication Date: June 16, 2023

Journal: International Journal of Technology Assessment in Health Care | open access article

Authors:

Abstract
Objectives: Uncertainty is a fundamental component of decision-making regarding access to, pricing and reimbursement of drugs. The context-specific interpretation and mitigation of uncertainty remain major challenges for decision-makers. Following the 2021 HTAi Global Policy Forum, a cross-sectoral, interdisciplinary HTAi-DIA Working Group (WG) was initiated to develop guidance to support stakeholder deliberation on the systematic identification and mitigation of uncertainties in the regulatory-HTA interface.

Methods: Six online discussions among WG members (Dec 2021-Sep 2022) who examined the output of a scoping review, two literature-based case studies and a survey; application of the initial guidance to a real-world case study; and two international conference panel discussions.

Results: The WG identified key concepts, clustered into twelve building blocks that were collectively perceived to define uncertainty: ‘unavailable’, ‘inaccurate, ‘conflicting’, ‘not understandable’, ‘random variation’, ‘information’, ‘prediction’, ‘impact’, ‘risk’, ‘relevance’, ‘context’, ‘judgement’. These were converted into a checklist to explain and define whether any issue constitutes a decision-relevant uncertainty. A taxonomy of domains in which uncertainty may exist within the regulatory-HTA interface was developed to facilitate categorization. The real-world case study was used to demonstrate how the guidance may facilitate deliberation between stakeholders and where additional guidance development may be needed.

Conclusions: The systematic approach taken for the identification of uncertainties in this guidance has the potential to facilitate understanding of uncertainty and its management across different stakeholders involved in drug development and evaluation. This can improve consistency and transparency throughout decision processes. To further support uncertainty management, linkage to suitable mitigation strategies is necessary.

Early HTA Terminology Working Group

Clear definition of the term “early HTA” is lacking and there is confusion with related terms such as “early advice”, “early dialogue”, and “development-focused HTA”. Reaching a consensus on what is meant by each of these terms will form a foundation for discussion and dialogue about the best methods to adopt and improve the profile of the work.

Purpose

The HTAi Early HTA Terminology Working Group aims to reach consensus on the definition of early HTA and related terms (early dialogue, early advice, development-focused HTA), to clearly delineate what does not constitute early HTA and link proposed definitions with existing HTA and early awareness definitions in the HTA Glossary. The methods are as follows:

Develop definitions and supporting materials for Delphi process, which includes:
- Rapid review of the literature for existing definitions and frameworks
- Core working group and Advisory Board develop definitions and supporting materials informed by the rapid review
A two-stage Delphi process
- Initial round presenting definitions and supporting materials to individuals involved in evaluation of new health technologies from industry, agencies and academia.
- Revised definitions and supporting materials circulated for rating. Consensus will be considered to be reached if 70% of respondents agree or strongly agree. If consensus is not reached on a particular aspect a further round may be considered.

Members

Leaders

Janneke Grutters
Janet Bouttell

Core Members

Neil Hawkins
Jon Karnon
Carla Fernandez
Michelle Tew
Wang Yi
Sulafa Ahmed
Laura Sampietro-Colom
Waseem Ahmed
Payam Abrishami
Erika Seyahian
Samuel Owusua Achiaw
Debjani Mueller
Nora Ibargoyen Roteta
Iñaki Gutierrez Ibarluzea

Advisory Members

Yves Verboven
Chris McCabe
Andrew Partington
Dalia Dawoud
Rabia Sucu

Outcomes

Expected outcomes include definitions of the following terms:

Early HTA
Development-focused HTA
Early Advice
Early Dialogue

The terms will be proposed for inclusion in the HTA Glossary. A scientific article describing the work of the WG will be prepared for submission to IJTAHC.