New innovative HTA methods for supporting novel approaches to deliver better precision medicine

New innovative HTA methods for
supporting novel approaches to
deliver better precision medicine

  W. Goettsch, PhD1,2

  1. National Health Care Institute (ZIN), Diemen, the
     Netherlands

  2. Utrecht Institute for Pharmaceutical Sciences, Utrecht
     University, Utrecht, the Netherlands

Defintion and (traditional) focus of HTA
•   Health technology assessment (HTA) is a multidisciplinary process
    that summarises information about the medical, social, economic
    and ethical issues related to the use of a health technology in a
    systematic, transparent, unbiased, robust manner. Its aim is to
    inform the formulation of safe, effective, health policies that are
    patient focused and seek to achieve best value.

•   Traditionally focus on:
    • single technologies, mostly pharma and medtech
    • National or regional recommendations to support reimbursement
      and pricing policies

2

Changes in focus of HTA

•   Internationalization (increasing efficiency, quality & predictability)
     o Medical vs non-medical (joint REA by EUnetHTA)
     o Alignment between stakeholders
        •   Within HTA (EUnetHTA)
        •   Outside HTA (regulators, health care providers, patients etc.)

•   Real world data

•   Personalized treatments
    o Smaller populations
    o Combination technologies
    o Different sequences of treatments

3

Development of more personalized treatment concepts
                                                                                    CAR-T treatment in practice (USA)

Franken, MG. et al. Policymaker, Please Consider Your Needs Carefully: Does   Schulthess D, Gassul D, Makady A etal. Assessment of Chimeric Antigen T-cell
Outcomes Research in Relapsed or Refractory Multiple Myeloma Reduce           Therapy (CAR-T). Effectiveness in practice. “Drug Information Association (DIA)
Policymaker Uncertainty Regarding Value for Money of Bortezomib? Value in     Congress, Boston, June 2018
Health 2014, Volume 17 , Issue 2 , 245 - 253

4

More focus on later stages of life cycle of pharmaceuticals

                                                                                          Comparative or full HTA
Use of technology in health care

                                                                                      (supporting appropriate use)

                                                                                                              HTA
                                                                                                              STAKEH
                                                                                                              REGU
                                                                 Initial
                                    Presenting and                HTA
                                                                                   Additional data
                                      discussing                                     collection
                                     requirements
                                                          Assessment for
                                    studies in ED*
                                                              market
                                                           authorization
                                    Collecting evidence
                                                                Time line of innovation                *Early dialogue
                                   in early development

5

NL Experiences with conditional funding (CF) and additional
data collection (2006-2012)

  T=0: Assessment                         Outcomes Research                     T=4: Re-assessment
  •Therapeutic value & 1e CE/BIA          •Dutch clinical practice              •Conclusions
  •Uncertainties identified               •Eg. registries                       •Advice to MoH on reimbursement
  •Outcomes research proposal discussed

Feedback from stakeholders on these experiences

                                               *Makady etal. Value in Health 2018 (accepted).               6

International Initiatives & Future Directions
• EUnetHTA WP5B: Core datasets for HTA registries
• EMA Registry initiatives: For instance qualification of disease
  registries
• IMI-GetReal I & II: RWE use for clinical effectiveness of drugs
• IMI-BigData projects, IMI-EHDEN: Using registries and big data
  generating RWE, collect health data in common data structure

• ISPOR/ISPE Special Task Force: Good procedural & reporting
  practices for RWE studies
• H2020 HTx (next generation HTA, 2019-2024) project:
   • develop methods that can integrate RWE and RCT in order to
     predict (cost-)effectiveness in small populations
   • To pilot implementation of these methods in Europe with the HTA
     bodies (EUnetHTA) and their stakeholders
   • Involvement in HTx from HTA, academics, patients, regulators etc.
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Conclusions
New HTA methods are needed:
• support internationalization
• adaptation to a new era of personalized medicine by combining RCT with
  RWD/big data
• alignment with regulators but also healthcare providers, patients and technology
  producers

In the coming years this will be supported by
• Interaction of HTA organisations in EUnetHTA
• Development of more personalized, methods through IMI and H2020 projects
• Implementation of these models in HTA practice;
    o EUnetHTA, IMI-GetReal, big data and H2020-HTx (international)
    o But again in collaboration with regulators, clinicians, patients and technology producers

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