1 / 11

“ 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

amal
Télécharger la présentation

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

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  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

More Related