Webinar Description

The first step to market access is an assessment of relative efficacy. To be granted regulatory approval, a new drug should have sufficient quality, efficacy, and safety, with benefits outweighing the risks in a predefined patient population with a given disease. The second step is to assess its relative effectiveness, cost effectiveness and usefulness to the healthcare system. This assessment is generally based on the evidence that a new drug does more good than harm (has added benefit) in a target population when compared to one or more therapeutic alternatives (standard of care) in achieving desired results when provided under the usual circumstances of health care practice.

The added benefit is assessed with appropriate endpoints and in comparison to an adequate comparator. The choice of endpoints and of an adequate comparator have a major impact on HTA decisions. Although many sponsors have their focus on regulatory guidelines while planning study design, incorporating HTA requests is essential for market access purposes. Indeed, a product may be refused for reimbursement if its relative effectiveness is considered inferior to existing treatments or if the evidence provided is considered inadequate or insufficient.

As joint European assessment reports will be introduced soon, we shall cover the main characteristics of the new EU HTA regulation, its implementation, and obligations it brings for all stakeholders: pharmaceutical companies (sponsors), HTA assessors, clinicians, and patients.

Why Attend this Training?

Beginning with January 2025, joint European HTA reports will be mandatory for oncology drugs and advanced therapy medicinal products. New structures have been put in place at the European Commission for this task; process, timelines, and content of the dossiers to submit is complex and very demanding.

The aim of this webinar is to revisit the benefit/risk assessment in the regulatory setting, the concept of HTA, and that of added benefit assessment for reimbursement purposes. We shall cover the main pillars of both regulatory and HTA assessments: population, intervention, comparators, and outcomes (endpoints) together with adequate methodology.

By attending this training course, you will:

• Deep Dive into Regulatory Approval Processes: Gain insights into the basis for regulatory approval, focusing on comprehensive benefit/risk assessments and the criteria for relative efficacy. Understand how these factors drive the approval process in the pharmaceutical landscape.
• Holistic Understanding of Health Technology Assessment (HTA): Delve into the definition, aim, and basic principles of HTA. Learn about the relative effectiveness assessment for reimbursement purposes across different European Union countries, enhancing your ability to navigate diverse regulatory environments.
• Mastering the PICO Framework: Get equipped with the skills to accurately identify and analyze the four elements of PICO: Population, Intervention, Comparators, and Outcomes. This knowledge is crucial in designing and evaluating clinical trials and healthcare research.
• Differentiating Outcome Types for Regulatory and HTA Assessments: Develop an understanding of various types of outcomes (endpoints) used in both regulatory and HTA assessments. Learn to select appropriate endpoints based on the specific requirements of each assessment type.
• Strategizing Dossier Development: Explore strategies for creating a comprehensive dossier that meets the criteria for both regulatory approval and HTA assessment. Learn about the commonalities and differences in requirements, streamlining the approval process.
• Elevating Methodology and Evidence Quality: Enhance your ability to evaluate and implement robust methodologies in clinical trials and research. Understand the criteria for high-quality evidence, vital for successful regulatory and HTA submissions.
• Navigating the EU HTA Regulation: Gain expertise in the new EU HTA regulation, understanding its implications, and requirements. Be prepared for the evolving regulatory landscape in the European Union, especially for oncology drugs and advanced therapy medicinal products.
• Collaborative Approaches in Joint Scientific Consultations and Assessments: Learn about the processes and benefits of joint scientific consultations and clinical assessments under the new EU framework, fostering collaborative approaches among stakeholders.
• Understanding Stakeholder Roles in HTA and Regulatory Processes: Develop a comprehensive view of the roles and responsibilities of each stakeholder in the HTA and regulatory processes, including pharmaceutical companies, assessors, clinicians, and patients.
• Anticipating Challenges and Formulating Solutions: Equip yourself with the knowledge to anticipate potential difficulties in the regulatory and HTA landscapes and learn effective strategies for overcoming these challenges, ensuring smoother market access for pharmaceutical products.