1. Regenerative Therapies KTN Meeting, Sheffield, April 2013 Paul Trueman, Smith & Nephew Securing Market Access for Regenerative Therapies

2. Congratulations! Successful regulatory approval

3. What now? Market Access Requirements

4. Market Access Market Access: a term used to describe activities necessary to secure reimbursement/coverage and adoption of a new technology, following regulatory approval.

5. Regulatory Approval vs Market Access Regulatory Approval requirements Well-defined Objective (Partial) international harmonisation Understood by industry High degree of interaction with mfrs Market Access requirements Often poorly defined and opaque Subjective – perceptions of value National or sub-national Low levels of industry understanding Few opportunities for negotiation

6. Market Access Roadmap: a Gordian Knot

7. Commonalities of Market Access Systems Typically, all market access functions want to address the following: 1. Does the product have regulatory approval? 2. Is the product clinically effective? 3. Is the product cost effective? 4. Is the price justifiable? 5. How can we minimise risk of inappropriate adoption?

8. Challenges for regenerative therapies 1. Does the product have regulatory approval? – Expectations and funding challenges dependent on regulatory route 2. Is the product clinically effective? – Is it feasible to conduct large scale studies of the technology? 3. Is the product cost effective? – T his is reliant on having good data on comparative effectiveness 4. Is the price justifiable? – Does the cost effectiveness analysis support the price? – What are the additional procedure costs that need to be taken into account? 5. How can we minimise risk of inappropriate adoption? – What is the target indication? How can we avoid unregulated adoption?

9. Understanding payment mechanisms Most major markets have adopted a DRG/case payment system covering surgical and medical procedures Provides a lump sum for a procedure, including all medical supplies, labour and sundries Innovative technologies often can not be accommodated into the case payment, due to their cost or complexity (e.g. require multiple episodes) Options are: – Seek to change the existing case payment – Develop a new case payment – Identify other ‘workaround’ payment mechanisms which might be available

10. Specialist funding routes Most healthcare systems have some exemptions from traditional funding routes, such as top-up payments or exclusions for high-cost technologies or pharmaceuticals These can be an appropriate route to securing initial adoption for innovative technologies Often less demanding in terms of the evidence required to support use However, criteria in place to restrict adoption and these are typically not routes to mass-market or long-term coverage

11. Conditional funding mechanisms Many markets are looking at novel ways to manage the adoption of innovative technologies, recognising the difficulties inherent in generating E.g. CMS ‘Coverage with Evidence Development’ or NICE ‘Only in Research’

12. Regenerative Therapies case studies: UK specialist funding Regenerative technologies have sought market access using a number of different approaches in the United Kingdom Specialised services top-up fees: Autologous Chondrocyte Implantation of Knee joints Devices excluded from the National Tariff: Biological Meshes All the examples identified highlight that these are regarded as ‘experimental’ or ‘non-routine’ interventions in a UK setting This can provide a foundation for market access but inevitably limits widespread adoption

13. Regenerative Therapies case studies: US funding Dermagraft (Human fibroblast derived dermal substitute) Device approval in the United States Reasonable evidence base on clinical and cost effectiveness Widespread coverage in physician office and hospital OP department Access limited by patient characteristics (e.g. duration of ulcer, treatment history) Adoption limited by number of dressings/treatment duration and response Crucially, associated with a procedure code, which allows physicians to bill for the application

14. Regenerative Therapies case studies: US funding Dermagraft (Human fibroblast derived dermal substitute) Critical success factors: Evidence to support coverage Clarity of positioning to minimise risk of unregulated adoption Physican fee to incentivise adoption by healthcare professionals

15. Summary  Regenerative Therapies face challenges to their adoption, following regulatory approval  Market Access requirements are disparate and manifold and may involve national and sub-national decision making bodies  There are relatively few examples of regenerative therapies that have secured widespread market access in the United Kingdom to date  To succeed, it is vital that manufacturers consider market access early in development, invest and plan accordingly  Agencies involved in Market Access need to be educated about the challenges faced by manufacturers of Regenerative Therapies