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Evidence-Based Haemophilia Care: Current Overview and Future Directions

Explore the principles of evidence-based care for hemophilia, key components, guidelines, and the community's journey towards embracing EBC. Understand the evolving scenario, challenges, opportunities, and the relevance of FDA/EMA and HTA in haemophilia care.

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Evidence-Based Haemophilia Care: Current Overview and Future Directions

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  1. EVIDENCE BASED HAEMOPHILIA CARE – CURRENT STATUS AND WHERE TO GO? Alfonso Iorio Health Information Research Unit & Hamilton-Niagara Hemophilia Program McMaster University

  2. In compliance with COI policy, EAHAD requires the following disclosures to the session audience: All funding administered via McMaster University – no personal compensation received

  3. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  4. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  5. FDA/EMA HTA

  6. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  7. The “concept” of comparison * % patient reporting Investigators – Unpublished data

  8. The “concept” of comparison * % patient reporting Investigators – Unpublished data

  9. The “concept” of comparison * % patient reporting Investigators – Unpublished data

  10. Components of a comparison • Comparator • Registration • HTA/reimbursement • Adoption in care • Study design • Registration • HTA/pricing • Adoption in care • Comparator • Historical • Inactive • Active • Study design • Randomized • Observational

  11. HTA

  12. The concept of comparable sample

  13. The concept of comparable sample • Multivariable analysis • Propensity score • Stratification • RCT

  14. MV P RCT S Wedge shape design Interrupted time series

  15. FDA/EMA

  16. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  17. Why would the hemophilia community embrace EBC? • Evolving scenario - challenges • Hemophilia • has been a prototype • has been the betrayal of the dream of a cure for a male’s disease in the roaring ‘60es of the infinite power of progress • Many more rare diseases with more expensive treatment • learning to advocate for their treatment in absence of alternatives • Different economical models landing on the stage

  18. Why would the hemophilia community embrace EBC? • Evolving scenario - opportunities • Hemophilia • Strong and widespread network of HTC, NMO, patients • Rare, but sizeable disease • many less patients studied that one could • Long tradition of collaboration among all relevant stakeholders

  19. McCarthy M. New drug for hepatitis C contributes to 13% rise in spending on prescription drugs in US. BMJ. 2015;350:h2055. • Cost effective, but also would cost an additional $65 billion over the next 5 years • ChatwalJ et al. Ann Intern Med. 2015;162:397-406. By JOSEPH WALKER April 8, 2015 4:37 p.m. ET

  20. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  21. Cochrane Collaboration data CF = Cystic Fibrosis IRBCD = inherited red blood cell disorders CBD = Congenital bleeding disorders

  22. List of Cochrane Reviews and trials

  23. RCTs on secondary prophylaxis Manco-Johnson Powell valentino valentino Aronstam Aronstam + 467 Morfini Malanghu Kavakli Publication year

  24. HTA in hemophilia • Swedish Council on Health Technology Assessment. Treatment of Hemophilia A and B and von WillebrandDisease (2011). • http://http://sbu.se/upload/Publikationer/Content0/1/Blodarsjuka/Treatment%20of%20Hemophilia%20A%20and%20B.pdf • Accessed February 2, 2016. • IQWIG Rapid Report • https://www.iqwig.de/download/A13-07_Executive-Summary_Treatment-of-patients-with-haemophilia.pdf • Accessed February 2, 2016.

  25. SBU report IQWIG Rapid Report • 27 studies / 8 reviews • Firm conclusions cannot be drawn for • Different regiments for acute bleed and surgery • Optimum time to start, dose, dose interval, discontinuation • [Need for registries] • 16 studies

  26. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  27. WFH guidelines • Srivastava, A, Brewer, A, Mauser-Bunschoten, E.P., Key, N.S., Kitchen, S., Llinas, A., Ludlam, C., Mahlangu, J.N., Mulder, K., Poon, M.C. & Street, A. • Haemophilia 2013, 19, e1–e47. • 3. Examination of Guidelines [from the IQWIG Rapid Report] • There are 13 treatment guidelines. Only three are evidence based. But most of the guidelines are in line with the results of the report. • The WFH treatment guidelines from 2012 were considered as “not evidence based”

  28. Guidelines in rare diseases • Developing methodology for the creation of clinical practice guidelines for rare diseases: A report from RARE-Best Practices. • Pai, M., Iorio, A., Meerpohl, J., Taruscio, D., Laricchiuta, P., Mincarone, P., Morciano, C., Leo, C.G., Sabina, S., Akl, E., Treweek, S., Djulbegovic, B. & Schunemann, H. • Rare Diseases, 2015, 3, e1058463.

  29. Overview • What is evidence based care (EBC) ? • What are the key components of EBC? • Is the EBC framework a value for hemophilia? • Can the EBC framework be applied to haemophilia? • Can EBC guidelines be issued in hemophilia? • Is the hemophilia community going toward EBC?

  30. Take home message

  31. Take home message

  32. Take home message

  33. Take home message

  34. Take home message

  35. Thank you !!! Download these slides at: Hemophilia.mcmaster.ca

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