Early Engagement: One Stop Shop

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1 Early Engagement: One Stop Shop Indranil Bagchi, Ph.D. Vice President, Payer Insights & Access April 7, 2014 Insert presentation title, GH&V tagline, you and your groups name and date

2 What is Early Scientific Advice? European Medicines Agency: Scientific advice is when the Agency gives advice to a company on the appropriate tests and studies in the development of a medicine. This is designed to facilitate the development and availability of high-quality, effective and acceptably safe medicines, for the benefit of patients. NICE: NICE Scientific Advice (SA) provides fee-for-service consultation to pharmaceutical companies. By reviewing early product development plans we can advise companies on whether these will generate relevant evidence for future submissions to NICE. Based on a briefing book, written questions submitted by the company and a face-to-face meeting, NICE SA provides an advice report. There is a fee for this service. More recently NICE SA has expanded the range of services we offer to give advice alongside regulators.

3 Early Scientific Advice (ESA): An Industry View What is ESA and what are its goals? Reduce delays in access for patients Reduce unintended/inexplicable differentials in access Reduce uncertainty in biopharmaceutical development by incorporating views on a variety of issues, including trial design trial length patient population and subgroups patient burden comparators primary and surrogate endpoints economic model Help develop the most useful evidence more efficiently Understand decision maker perspectives

4 Early Scientific Advice: An Industry View What are some of the current obstacles and issues in the way? Balancing full participation with different groups goals, structures, and preferences E.g., each agency has its legislative history, authority and consequently unique viewpoint We need to account for the potential of variation across therapeutic areas and diseases Can any current inconsistencies be harmonized for betterment of the system When aggregated, are the evidence requirements in an area practical for the perceived value, or are they likely to just cause innovation to dry up Without all relevant stakeholders, there is a danger of a consensus that fails to meet the goals We need to include physicians and patients Consistency on terms that maybe detrimental to long-term access for patients Beyond the most restrictive and risk-averse Building trust and consistency of advice through time, as development takes time and resources and circumstances change through the lifecycle of a product

5 Requirements for Ideal Parallel Advice Strategically Focus on areas of issue / uncertainty to company Bridging of different requirements from Regulatory and HTA agencies Aim of alignment on realistic, achievable, requirements to optimise the development plan Timing ideally pre-phase III with option for pre-phase II Practically Common procedures and timelines across Regulatory & HTA bodies Flexibility in choice of HTA bodies Simultaneous submission of common briefing documents Knowledgeable experts from HTA bodies Representative number of HTA participants (3-5?) Equality of voice between stakeholders Source: EFPIA

6 Stakeholder involvement: One Stop Patients Prescribers Industry Payers Regulatory

7 Payer Customer Consultations for Tofacitinib Including scientific advice for HTA 1Q 10 2Q 10 3Q 10 4Q 10 1Q 11 2Q 11 3Q 11 4Q 11 1Q 12 2Q 12 3Q 12 4Q 12 Caremark US NICE UK MPA/TLV Sweden NICE UK egence US MoH Spain Italy KOL MDK Germany HAS France CADTH Advisors Canada RA 1 st Launch PBAC Australia PsO RA RA+PsO PsA

8 Lessons Learned: Tofacitinib

9 Considerations for ESA Pricing & Reimbursement decisions and criteria for appraisal are and will remain country-specific Cost of care, medical care continuum, preference for site of care, clinical comparators, etc. are highly variable across national jurisdictions Perspective of budget holders is highly dependent on funding sources for health care and differs across national jurisdictions in EU But significant actions are possible to reduce differences between local HTA reports/guidance Transparent guidance and definition of incremental medical benefit that rewards innovation; sustains incentives for maintaining future investment in innovation and is applicable across a range of jurisdictions Alignment in recommendation of regulatory and HTA agencies vis-à-vis comparators and endpoints for development program Standardization of briefing book and consultation format