EVIDENCE BASED HAEMOPHILIA CARE CURRENT STATUS AND WHERE
EVIDENCE BASED HAEMOPHILIA CARE – CURRENT STATUS AND WHERE TO GO? Alfonso Iorio Health Information Research Unit & Hamilton-Niagara Hemophilia Program Mc. Master University
In compliance with COI policy, EAHAD requires the following disclosures to the session audience: Shareholder No relevant conflicts of interest to declare Grant / Research Support Bayer, Baxalta, Biogen, Novo Nordisk, Pfizer Consultant Bayer, Baxalta, Novo Nordisk Employee No relevant conflicts of interest to declare Paid Instructor No relevant conflicts of interest to declare Speaker bureau No relevant conflicts of interest to declare Honoraria No relevant conflicts of interest to declare All funding administered via Mc. Master University – no personal compensation received
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? • •
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? • •
FDA/EMA HTA
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? • •
The “concept” of comparison Dimension Country EQ 5 D-5 L A (some prophy) B (no prophy) Chronic pain A (some prophy) B (no prophy) Control Mild Mod Severe . 897. 894 . 788. 801 . 676. 590 28% 30% 53% 56% 68% 70% 78% 86% Investigators – Unpublished data * % patient reporting
The “concept” of comparison Dimension Country EQ 5 D-5 L A (some prophy) B (no prophy) Chronic pain* A (some prophy) B (no prophy) Control Mild Mod Severe . 897. 894 . 788. 801 28% 30% 53% 56% 68% 70% . 676. 590. 086 78% 86% Investigators – Unpublished data * % patient reporting
The “concept” of comparison Dimension Country EQ 5 D-5 L A (some prophy) B (no prophy) Chronic pain A (some prophy) B (no prophy) Control Mild Mod Severe . 897. 894 . 788. 801 28% 30% 53% 56% 68% 70% . 676. 590. 086 78% 86% +56% Investigators – Unpublished data * % patient reporting
Components of a comparison • Comparator – Historical – Inactive – Active • Study design – Randomized – Observational • Comparator – Registration – HTA/reimbursement – Adoption in care • Study design – Registration – HTA/pricing – Adoption in care
HTA
The concept of comparable sample
The concept of comparable sample • Multivariable analysis • Propensity score • Stratification • RCT
MV P S RCT Wedge shape design Interrupted time series
FDA/EMA
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? • •
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 ‘ 60 es 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
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
• Mc Carthy M. New drug for hepatitis C contributes to 13% rise in spending on prescription drugs in US. BMJ. 2015; 350: h 2055. • Cost effective, but also would cost an additional $65 billion over the next 5 years – Chatwal J et al. Ann Intern Med. 2015; 162: 397 -406. By JOSEPH WALKER April 8, 2015 4: 37 p. m. ET
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? • •
Cochrane Collaboration data Trials (CENTRAL) SR (Cochrane) Completed Ongoing CF 2497 79 7 CF = Cystic Fibrosis IRBCD = inherited red blood cell disorders CBD = Congenital bleeding disorders IRBCD 838 CBD 425 36 16 11 4
List of Cochrane Reviews and trials 11 completed reviews 6 reviews included 28 RCTs 1007 patients were randomized
Morf ini Arons tam Aron stam 0 1971 1981 + 467 1991 2001 Publication year Pow vale ell Man ntino co-J vale ohns o Mal ntino n a Kav nghu akli RCTs on secondary prophylaxis 1 2011 2021
HTA in hemophilia • Swedish Council on Health Technology Assessment. Treatment of Hemophilia A and B and von Willebrand Disease (2011). – http: //sbu. se/upload/Publikationer/Content 0/1/Blodarsjuka/Treatment%20 of%2 0 Hemophilia%20 A%20 and%20 B. pdf – Accessed February 2, 2016. • IQWIG Rapid Report – https: //www. iqwig. de/download/A 13 -07_Executive-Summary_Treatment-of-patientswith-haemophilia. pdf – Accessed February 2, 2016.
SBU report IQWIG Rapid Report • 27 studies / 8 reviews Domain A+A C Mortality • Firm conclusions cannot be drawn for – Different regiments for acute bleed and surgery – Optimum time to start, dose interval, discontinuation – [Need for registries] • 16 studies EQ 5 D ✔ Chronic pain ✔ Acute pain Joint function Severe bleeds Life threatening bleeds Inhibitor HR-Qo. L ✔ ✔
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? • •
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, e 1–e 47. • 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”
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, e 1058463.
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? • •
Take home message Item Robust study design Comparative effectiveness Rigorous EBP guidelines Adopted Feasible Value Potential
Take home message Item Robust study design Comparative effectiveness Rigorous EBP guidelines Adopted Feasible Value Potential Sometimes Moderate High
Take home message Item Robust study design Comparative effectiveness Rigorous EBP guidelines Adopted Feasible Value Potential Sometimes Moderate High Rarely Yes High
Take home message Item Robust study design Adopted Feasible Value Potential Sometimes Moderate High Comparative effectiveness Rarely Yes High Rigorous EBP guidelines Rarely YES High
Take home message Item Robust study design Examples Registry based CCT Comparative effectiveness PROBE Rigorous EBP guidelines GRADE / RARE Best. Practice
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