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“ Supporting R&D in rare diseases: barriers and incentives” – Industry’s Views

“ Supporting R&D in rare diseases: barriers and incentives” – Industry’s Views. Charles Waller, PPTA VP Europe EHC Roundtable of Stakeholders, Brussels 7 December, 2011. Agenda. Landscape Uniqueness Safety Novel coagulation treatments Rare coagulation disorders Manufacturing technology

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“ Supporting R&D in rare diseases: barriers and incentives” – Industry’s Views

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  1. “Supporting R&D in rare diseases: barriers and incentives” – Industry’s Views Charles Waller, PPTA VP Europe EHC Roundtable of Stakeholders, Brussels 7 December, 2011

  2. Agenda • Landscape • Uniqueness • Safety • Novel coagulation treatments • Rare coagulation disorders • Manufacturing technology • Clinical trials

  3. Landscape Global Budget pressure Regional approach Collapsing global economies Need to reduce: Costs Utilization Threats to Industry: Decrease in reimbursement Increase in costs Increase in regulatory encroachment Restrictions on access

  4. The Global Landscape • Greece - price cuts averaging 20% • Italy generic price cuts and new NHS spending caps • Spain a 7.5% mandatory discount implemented in 2010, reference price system extended • Germany €1bn rebate in 2010 - 2013 - 6% to 16% • Belgium – PPTs moving out of exemption lists into mainstream hospital budgets • USA – Debt ceiling legislation, super committee

  5. Unique Cost Structure • Consequence = Consolidation

  6. Industry Investment: Safety • Development of large scale NAT testing • Development of viral inactivation methods • No transmissions of any blood borne virus since 1994 • Emerging pathogens and other safety questions • WNV • HEV • Thrombo-embolic Events • ??

  7. Industry Investment: Novel treatments • nVIII • Different generations • nVWF • Inhibitor bypassing agents

  8. Industry Investment: Rare coagulation disorders • Factor XI • Factor XIII • Factor V (underdevelopment )

  9. Industry Investment: Manufacturing Technology • Chromatography purification • Filtration for separation (higher yields) • Plasma processing: • Containers • Thawing

  10. Industry Investment: Clinical trials • Support for prophylaxis studies • Inhibitor Investigation • Better presentation and higher doses

  11. Conclusions • The plasma protein industry has shown considerable commitment to safety and innovation • Coagulation still emerging understanding • Barriers to access still include inappropriate regulatory and reimbursement pathways • Perceptions of product safety are still contributing to non-optimal therapy • More education, communication and collaboration between industry and patients will help address these problems

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