Issues And Challenges When Pharma Works With Regulators & HTA Agencies

Published Sep 25, 2013
Canberra, Australia - The pivotal phase III clinical trials for new medicines are designed to support their registration, yet they are also used to support their reimbursement and pricing. The clinical evidence needs of HTA agencies (payers) are often different than those of the regulators. The innovative pharmaceutical industry needs to engage with the major regulators and HTA agencies to determine what clinical evidence can, and should, be developed as part of a phase III clinical trial program to support their timely registration and reimbursement. In the article, “Early Scientific Advice Obtained Simultaneously from Regulators and Payers: Findings from a Pilot Study in Australia,“ the researchers report on early scientific advice of a project conducted by a multinational pharmaceutical company (developer) with representatives of the Therapeutic Goods Administration (regulator) and the PBAC Secretariat (HTA agency/payer). The article provides an important contribution to the international discussion and debate on models of increased engagement and co-operation between technology developers, regulators and HTA agencies. The primary objective of the pilot was to explore the practicality, feasibility and value of obtaining simultaneous scientific advice for a development compound from both a regulatory and reimbursement perspective. The researchers hoped to identify issues that might promote or impede the establishment of a sustainable three-part (HTA agency, regulator, developer) scientific advice process. The representatives from the TGA and PBAC Secretariat provided well-informed, considered and careful advice for both compounds, which was predominantly actionable and practical. "The current development paradigm of the innovative pharmaceutical industry of generating clinical evidence to support registration needs to change to one that supports timely market access. The pharmaceutical industry needs to work more closely with the major regulators and HTA agencies to effect real change. This paradigm shift faces many challenges." said Michael Wonder, BPharm, Director, Wonder Drug Consulting, Sydney Australia, and lead author on the study.

Value in Health (ISSN 1098-3015) publishes papers, concepts, and ideas that advance the field of pharmacoeconomics and outcomes research as well as policy papers to help health care leaders make evidence-based decisions. The journal is published bi-monthly and has over 8,000 subscribers (clinicians, decision-makers, and researchers worldwide).

International Society for Pharmacoeconomics and Outcomes Research (ISPOR) is a nonprofit, international, educational and scientific organization that strives to increase the efficiency, effectiveness, and fairness of health care resource use to improve health.

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