In keeping with our commitment to technical excellence in health economics and outcomes research, we shared complimentary micro-tutorials during ISPOR Europe 2023 in Copenhagen. Our micro-tutorials offer a unique opportunity for one-on-one engagement with our renowned experts, delving into topics of scientific and strategic significance for HTA. In case you missed us at ISPOR and are interested in our topics, please contact us to discuss with our experts.
The micro-tutorials:
Real-world external control arms in HTA: Opportunities and challenges
Tutors
Alex Turner, PhD
Director, RWE and Biostatistics
Content
- A historical perspective on the acceptability of external control arms in HTA
- A review of current HTA guidance relating to real-world external control arms
- A review of current HTA decision-making relating to real-world external control arms (case studies)
- Recommended practices in designing and executing an external control arm strategy
Audience
- Those with a responsibility for real-world evidence generation
- Those working with assets likely to approach the market with single arm data comparing interventions
HTA decision-making: A case for discrete choice experiments?
Tutors
Frauke Becker, PhD
Principal, HEOR
Yemi Oluboyede, PhD
Principal, HEOR
Content
- Inclusion of patient preferences in HTA: Advantages and challenges
- Evidence generation
- Value propositions
- Use of discrete choice experiments (DCEs) in HTAs: Status quo
- Exploring HTA agencies’ stance and available guidance
- Generation of robust quantitative preference data for future reimbursement decisions
Audience
- Market access and HTA teams seeking to understand how evidence from DCEs can be included in HTA submissions and used to strengthen value propositions
Development and validation of patient-reported outcome tools to support HTA submissions
Tutors
Yemi Oluboyede, PhD
Principal, HEOR
Frauke Becker, PhD
Principal, HEOR
Content
- Patient-reported outcomes (PROs)
– Provide the patients’ unique perspective on the consequences of treatment, the impact of disease symptoms, functional health and well-being
– Provide evidence for shared decision-making, clinical guidelines and health policy (Calvert et al. 2018)
- Use of valid and reliable generic and disease-specific PROs in the context of evidence generation is fundamental
- Exploring available guidance in terms of the generation and use of robust PROs is essential for evidence generation to support HTA submissions
Audience
- Market access, HTA and HEOR teams seeking to understand how to identify existing and develop new PRO tools that are valid and robust for use in clinical trials and informing strategic decision-making around HTA submissions
Reference:
Melanie C, Kyte D, Mercieca-Bebber R, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: The SPIRIT-PRO extension. JAMA 2018;319(5):483-494.
Strategic investment throughout the product lifecycle: Evaluating the evolving literature review landscape
Tutors
Dr Donna Fountain, PhD
Partner, Market Access
Content
- How to strategically leverage literature review evidence from early-phase clinical development to post-launch value generation
- Choosing the right literature review methodology to support commercial objectives across multiple markets: Systematic, targeted, scoping, umbrella and living reviews
- Considerations for literature review planning to align with evolving HTA requirements in Europe
- An overview of the artificial intelligence landscape in literature review methodology
Audience
- Market access and HEOR teams seeking to understand how evidence generated through tailored literature review methodologies can be applied to inform strategic decision-making during early clinical development, HTA reimbursement pathways, and broad value right through to post-launch
Early scientific advice in the era of joint clinical assessments
Tutors
Michael Lees
Partner, Market Access Strategy
Cécile Rémuzat
Principal, Market Access Strategy
Content
- A brief contextualization of early HTA scientific advice and reasons to get engaged
- An overview of early HTA scientific advice opportunities in Europe (national/EU joint scientific consultation)
- Future perspectives and key strategic considerations when engaging into early HTA advice within the new EU HTA landscape
Audience
- Market access and HEOR professionals, particularly those working in earlier development, who need greater understanding of the impact of the new EU HTA Regulation on early scientific advice processes and subsequent HTA processes in Europe to inform evidence and launch planning for new medicines
Early access programs for pharmaceuticals in France
Tutors
Clément François
Partner, HTA operations
Cécile Rémuzat
Principal, Market Access Strategy
Content
- An overview of early access opportunities in France
- A status of early access granted since the reform implementation
- Strategic considerations to decide to engage into early access process in France and how to get ready for it
Audience
- Market access and HEOR professionals who require more understanding of the reform to the French early access programs and how these programs can be used to support market access and HTA processes in France and beyond, in addition to ensuring early access to patients in areas of high unmet need
Optimizing pricing and market access outcomes in Japan
Tutors
Michael Lees
Partner, Market Access
Yoshie Onishi, PhD
Principal, Market Access and Putnam Japan Lead
Clément François, PhD Partner,
Market Access
Content
- Overview of Japanese approach to initial drug pricing, re-pricing and HTA
- Key drivers of price premium in Japan, including strategic choices and evidence needs to optimize the price premium
- Post-launch price and reimbursement adjustments in Japan, including the role and impact of HTA
- Emerging trends in Japanese pricing and market access
- Key strategic considerations to optimize pricing and access in Japan and to incorporate Japanese needs into global strategy
Audience
- Market access and HTA experts seeking a comprehensive understanding of Japanese pricing and reimbursement processes and the key strategic questions to be addressed by Japanese and global teams to optimize reimbursement outcomes in Japan
Distributional cost-effectiveness analysis: Capturing equity impacts in economic evaluation
Tutors
Aurelie Meunier
Director, HEOR
Content
- Introduction to DCEA: When and why conducting a DCEA?
- Methods overview: Estimation of health distributions and assessment of equity impacts
- Presentation of published examples of DCEAs
- Discussion on:
– Data requirements and challenges of building DCEA
– Implementation in HTA
Audience
- Health economists and market access teams with an interest in health equity and valuation of interventions beyond cost-effectiveness
Integrated evidence generation planning: Frameworks and approaches to optimize value-based strategies
Tutors
Joanne Evason
Partner, Putnam
Eric Auger
Partner, Value, Pricing & Access Practice Lead
Content
- The increasing importance of cross-functionally developed early value narratives
– The interplay between value narrative and strategic evidence planning and generation
– Strategic frameworks to support development of the narrative and prioritization of evidence
– Practical tips and ways of working to facilitate alignment, develop impactful narratives and optimization of evidence generation activities, and how to ensure it is evolutionary in nature
Audience
- Access, HEOR and Evidence teams seeking to understand how to build strategic value narratives and the role of evidence generation to support impactful demonstration of value
