Optimise Your Regulatory & HTA Submissions with a Comprehensive Health Economic & Epidemiologic Evidence Strategy

Key Insights

Join us to learn about the best practices for developing a comprehensive health economic and epidemiologic evidence strategy and evidence generation plan that is targeted to meet the needs of regulators and payers.

Get insights on the different types of health economic and epidemiologic evidence that can be used to optimise the success of regulatory and HTA submissions, the typical timeframes for generating each piece of evidence, and how to address challenging HTA submission scenarios, such as when:

• Sponsors only submit Phase II data for accelerated regulatory approval
• Clinical trials did not demonstrate clinical/statistical significance in their primary endpoint
• Clinical trials effect sizes did not meet the existing cost-effectiveness thresholds

Overview

Regulators and payers have placed new demands on the pharmaceutical and biotechnology industry to provide health economic and epidemiologic evidence, in addition to clinical trials data, to demonstrate the value and safety of a new product. A well-developed evidence strategy is an important part of the clinical development program, and is a key component of regulatory and HTA submission planning.

Planning for regulatory and HTA submissions requires strategic foresight into what regulators and payers will consider most influential in their respective reviews. An important step is to identify the health economic and epidemiology evidence that needs to be collected during the clinical development program, and what evidence may already exist. A landscape assessment, done when the investigational product is in early clinical development, can identify evidence sources that may already exist in the published literature, and the availability of:

• Background epidemiologic data on the incidence/prevalence of the disease
• Characteristics and comorbidities of the target patient population , and the burden of disease
• Current treatment guidelines for the disease
• HTA approaches used for other therapies with the same indication
• Current and future market access and pricing considerations
• Data on the humanistic and economic impacts on the disease

Also in early clinical development, it is important to define the aspirational value message for the investigational product in terms of unmet medical need, clinical value, human value, or economic value. By aligning the health economic and epidemiologic evidence strategy with the aspirational value message, the relevance of each piece of evidence becomes clear.

Best Practices and Challenges

Identify Evidence Gaps Early

Identify any gaps in the health economic and epidemiologic evidence strategy as early as possible so the necessary research can be completed for inclusion in the regulatory and HTA submissions. When this is developed early enough, the health economic and epidemiologic evidence generation plan can be executed, in part in the Phase II trials to collect data on target adverse events, healthcare resource use, and health state preference utilities.

Clinical development programs without a comprehensive health economic and epidemiologic evidence generation plan prior to Phase III are less likely to have the appropriate evidence needed to optimise the success of regulatory and HTA submissions.

Solicit Early Stakeholder Feedback

Over the past 5-10 years, payers and reimbursement agencies have been moving to standardise the evidence requirements for HTA submissions. Many payers are willing to meet with a sponsor during Phase II to comment on the sponsor’s evidence strategy in relation to the product’s proposed labelling claims and value proposition.

Soliciting this early feedback is critical for understanding payers’ decision-making processes and their respective weighting of health economic and epidemiologic evidence in their reviews.

Be Ready for Challenging HTA Submissions

It is also important to consider challenging situations for preparing HTA submissions, including when:

• Sponsors only submit Phase II data for accelerated regulatory approval
• Clinical trials did not demonstrate clinical/statistical significance in their primary endpoint
• Cost-effectiveness thresholds were not met

Register today to learn how having a comprehensive health economic and epidemiologic evidence strategy and evidence generation plan can optimise your regulatory and HTA submissions.

Speakers

Michael Cook, MSc, MSPH, PhD, Principal, Epidemiology, ICON Commercialisation & Outcomes

Dr. Cook has 15-years of pharmaceutical company experience holding positions of increasing responsibility at Merck, Pfizer and Shire. His area of methodological expertise is in the analysis of large health care databases to conduct real world evidence (RWE) studies.

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Dhvani Shah, MS, Lead Health Economist, ICON Commercialisation & Outcomes

Dhvani Shah has over 8 years of experience in HEOR, and has led numerous evidence generation activities to demonstrate and communicate product value. She is responsible for preparing HEOR research and value strategies, developing economic models for HTA submissions and conducting literature reviews and evidence synthesis activities.

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