1 Comparator in HTA Some methodological challenges Preliminary - PDF document

Drug Relative efficacy and relative development effectiveness – do regulatory Does a new drug have a agencies and reimbursement Regulatory clinical effect under agencies have the same needs? approval controlled conditions? How effective is a new drug An HTA perspective HTA compared with existing Dr Elisabeth George treatments in routine clinical practice? NICE and EUnetHTA partner Use in healthcare system 1 2 “Comparative/relative effectiveness” Drug for the purpose of our discussions … development Relative efficacy  Establishing clinical value in routine practice Regulatory  HTA definition of ‘added therapeutic value’ approval  …. in routine practice, a new drug offers better ‘…based on …based on p , g clinical outcome, and/or better safety and/or simpler identical data’? administration, than existing alternatives HTA  Exploration and quantification of the magnitude of difference in health outcomes between new treatment and existing treatments Relative effectiveness Use in  To help decide if a new drug is made available healthcare system within a (national) health care system 3 4 Question 1 Question 2 Can decisions on the clinical value of a new Can REA be carried out across countries on medicine and its place in routine practice be an international basis/ EU basis? made on an international basis/ EU basis? • Not easily • Routine practice is not identical across • Information could be shared across countries countries countries • Duplication of effort in information gathering and D li ti f ff t i i f ti th i d evidence collection could be avoided • National responsibility for configuration of • Methodological approaches could be shared health services and medical care – Could they be standardised? – Diverse funding of health care and organisation of service delivery … there are challenges 5 6 1

Comparator in HTA Some methodological challenges • Preliminary results from EUnetHTA survey • 16 European countries/ Canada/ Australia/New Zealand • Comparators • Multiple entries possible – differ between EMA/FDA and HTA 12 – differ between countries 10 • Different organisational structures and 8 6 pathways of care 4 • Quantifying and valuing health effects 2 0 • Acceptance of methodological approaches Whatever Best possible Best other across countries was in the care standard registration care 7 trials 8 Definitions Some methodological challenges ‘Best standard care’ and ‘Other’ • usually the treatment(s) used in current clinical practice • Comparators • Most frequently used therapy – differ between EMA/FDA and HTA • ‘routine care,’ that is, the technology or technologies – differ between countries most widely used in clinical practice • Most frequently used pharmaceutical in practice • Different organisational structures and • 'Currently accepted therapy' which is defined as the pathways of care single most prevalent clinical practice • Quantifying and valuing health effects • Most commonly used alternative pharmaceutical • Actually reimbursed treatments with the same • Acceptance of methodological approaches therapeutic indication across countries 9 10 Acceptance of methodological Quantifying different health effects – a hypothetical example approaches across countries Treatment A Treatment B • Approaches to single arm studies Diarrhoea moderate absent • Acceptance of observational data or other non-RCT data other non RCT data Hot flushes Hot flushes mild mild moderate moderate • Acceptance of secondary end points Breast swelling no problems severe • Indirect / mixed treatment comparisons Physical energy severe fatigue no problems • Inclusion of qualitative evidence Life expectancy 12 months 11 months 11 12 Adapted from M Sculpher 2

Question 3: In conclusion If so, how should this be done and who should be involved? It is not helpful to blur boundaries between relative • Identification of evidence and information efficacy and relative effectiveness assessment that can be shared across countries • Developing agreed scientific and Developing agreed scientific and It is helpful to build a strong bridge between It is helpful to build a strong bridge between methodological standards for REA and other relative efficacy and relative effectiveness assessments evidence requirements • Changes to the EPARs to allow clinical data We need to develop consistent evidence to be more easily accessible for HTA standards for relative effectiveness assessment evaluation 13 14 3