Value, Access and Regulatory Strategy Workshop

Session Chair(s)

Mira Pavlovic, DrMed, MS

• Founder
• Medicines Development and Training (MDT) Services, France

After having obtained marketing authorisation, 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. An early dialogue/scientific advice between the sponsor and the Health Technology Assessment (HTA) bodies before the start of the pivotal clinical programme is particularly useful to discuss the evidence to provide and ultimately facilitate HTA process, timely reimbursement decision(s) and market access. For this purpose, sponsors may ask for national or multinational, regulatory and/or HTA advice. The experience gathered so far will be discussed as well as ways of possible improvements. When receiving guidance from HTA bodies, national authorities apply country/region-specific policies and legal requirements related to the specificities of their own healthcare system. Therefore, respecting HTA needs for evidence generation does not guarantee that a drug will be reimbursed. A topic of specific interest here is the choice of an adequate comparator when the latter differs in different European countries including the use of non-authorised comparators. Possible implications for trial designs will be discussed.

• Experience so far
• How to improve it (continuous dialogue)
• Early dialogue in PRIME and ADAPTIVE PATHWAYS
• Special topic: choice of comparator and off-label use
  • Differences in acceptance by HTA bodies
  • Implications for trial design

Speaker(s)

HTA Bodies Cooperation on Early Dialogues: Recent Advances

François Meyer, MD

• Advisor to the President, International Affairs
• French National Authority for Health (HAS), France

Enhancing the Dialogue Beyond RCTs

Byron Jones

• Executive Director
• Statistical Methodology and Consulting Group, Novartis Pharma AG, Switzerland

The Imperfect Study Design – How to Deal with It?

Stefan Lange, MD

• Deputy Director
• Institute for Quality and Efficiency in Health Care (IQWiG), Germany