Early scientific advice obtained simultaneously from regulators and payers: findings from a pilot study in Australia.

Objective: There is scope for better interaction between regulators, payers/HTA agencies, and medicines developers in their common objective of getting new medicines to patients. This paper reports on a tripartite early scientific advice pilot conducted by a pharmaceutical company (developer), the Therapeutic Goods Administration (TGA: regulator) and the Pharmaceutical Benefit Advisory Committee (PBAC) Secretariat (HTA agency) in Australia. The objective was to explore the practicality, feasibility, and sustainability of means of obtaining simultaneous scientific advice from both a regulatory and reimbursement perspective.

Methods: Advice was sought for two development compounds in different disease areas. The focus was on matters of common interest to the TGA and the PBAC (i.e. the clinical evidence). Briefing books were prepared by the developer and supplied eight weeks prior to the meeting and only verbal advice was provided.

Results: The pilot meeting took place in 2009. Each session lasted for approximately two hours and was structured around the questions in the briefing books. The representatives from the TGA and PBAC Secretariat provided well-informed, considered and careful advice for both compounds, which was predominantly actionable and practical.

Discussion: The sessions proved highly informative and permitted better alignment of the possible positioning of new medicines with the clinical evidence that regulators and HTA agencies might subsequently require for favorable assessment. The process provided early and clear signals to inform major development investments and the probability of successful market access. A number of challenges need to be addressed before tripartite scientific advice can be provided on continual basis.