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.

For more information: www.ispor.org

Related Stories

The Ozempic Paradox: How Spending Billions on Weight-Loss Drug Would Actually Reduce Overall Medicare Costs

Oct 14, 2025

Value in Health, the official journal of ISPOR—The Professional Society for Health Economics and Outcomes Research, announced today the publication of a landmark study by researchers at the University of Washington, Curta, Inc, and the University of North Carolina showing that broad Medicare coverage of semaglutide in diabetes, obesity, and liver disease could generate significant cost savings while delivering substantial health benefits to beneficiaries. The report, “Comprehensive Access to Semaglutide: Clinical and Economic Implications for Medicare,” was published in the October 2025 issue of Value in Health.

ISPOR Launches New Content on Whole Health

Sep 23, 2025

ISPOR announced that it has launched new website content on whole health, a topic of increasing importance as health systems across the globe grapple with providing the best possible healthcare to patients within constrained budgets.
Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×