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Current Approaches in European             Drug related Health Care Policy                 relative effectiveness and real...
New evidence for decisions aboutmarket access                              Market                               Page 3
Regulatory changes• Drugs have been withdrawn from the  market due to in-appropriate use   – Based on risk benefit assessm...
Page 5
Regulatory decisions, uncertainty andopportunity cost• More stringent criteria for effectiveness and  safety, i.e. net hea...
Health technology Assessment(HTA)• Shift in focus from established therapies to new  technologies, particularly drugs• Lim...
New drug introductions 1940-2009                                   Page 8
Reduced productivity in R&D                              Page 9
Mean cost of taking a NME to the    market• Di Masi 2003              USD 1 billion• Paul et al 2010           USD 1.8 bil...
Increasing role for public payment for drugs• Chronic therapy for RA and MS cost  1000 Euro per month• New cancer drugs pr...
The major problem for HTA – Lack of data• Regulatory decisions based on clinical trials aimed  at investigating if a new d...
Re-engineering drug development tomeet payer demands• Early HTA advice on development  strategies  – May help directing R&...
Payer decisions, uncertainty andopportunity cost• More stringent criteria for relative effectiveness  and cost-effectivene...
Methods for the Estimation of the NICE CostEffectiveness ThresholdKarl Claxton, Steve Martin, Marta Soares, Nigel Rice,Eld...
Patients and real life data• Potential patients are interested in access to new  medicines regard of their costs• Variatio...
All decision makers andstakeholders demand real lifedata                                Page 17
Sir richard            doll            Smoking causes            cancer           Bradford Hill criteria for           cau...
Collecting real life data• Data never speaks for themselves   – Data versus evidence   – Evidence depends on the circumsta...
Collecting real life data• Choice of outcome measure   –   Mortality   –   Age adjusted mortality   –   Survival   –   Qua...
Analysing real life data• The problems with “causation” must  always be considered• The need for improvement in  statistic...
real life data and policy• Coverage by evidence development  (CED)  – Requirement to collect data part of the    reimburse...
Issues related to CED and P4P•   How common is this is different countries?    – Are there provisions for this in legislat...
United Kingdom (NICE)•   Experience: MS risk sharing scheme in 2002    –   Price linked to outcome in clinical practice   ...
France•   Experience    – Long tradition of follow up studies    – Mainly to control that indications are followed    – No...
Germany•   Experience    – Possible for hospital drugs but not used•   Today    – Federal Joint Commission and a pharmaceu...
Sweden•   Experience    – CED is used in at least      20 cases by TLV 2002-      12                        Evidence on ef...
CED, P4P and CER in the US – Growing importance forEurope                                                      Page 28
Why CED and P4P are going to be the newstandard in health care – Outcome matters                                       Pag...
Conclusions• Regulators and HTA/reimbursement  agencies/payers have a common objective   – Improvements in public health• ...
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Current Approaches in European Drug related Health Care Policy: Relative Effectiveness and Real Life Studies

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Key note speech held by Prof Bengt Jönsson from Stockholm School of Economics and Chairman of the NDA HTA Advisory Board, at the ZonMw Congres Goed Gebruik Geneesmiddelen, 31st January 2013, on the topic of dealing with decision making under uncertainty for the reimbursement of medicines

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Current Approaches in European Drug related Health Care Policy: Relative Effectiveness and Real Life Studies

  1. 1. Current Approaches in European Drug related Health Care Policy relative effectiveness and real life studies Bengt Jönsson Stockholm School of EconomicsKey note lecture ZonMw Congres Goed Gebruik Geneesmiddelen,31 January 2013 Page 1
  2. 2. New evidence for decisions aboutmarket access Market Page 3
  3. 3. Regulatory changes• Drugs have been withdrawn from the market due to in-appropriate use – Based on risk benefit assessment – Data on side effects only one side of the assessment• Post-authorisation efficacy studies (PAESs) a new tool for regulatory decisions – Conditional market authorization (CMA) or “adaptive licensing” – Data on “relative efficacy” also relevant for HTA Page 4
  4. 4. Page 5
  5. 5. Regulatory decisions, uncertainty andopportunity cost• More stringent criteria for effectiveness and safety, i.e. net health benefit, may lead to delay of access to valuable drugs and increasing costs for drug development• Relaxed criteria may lead to the introduction of drugs with a negative net health benefit• Decisions must be made on incomplete data – A system for follow-up and re-assessment of ADRs is in place based on “real life data” – Increasingly recognized that net health benefit depends on how the drugs are used in clinical practice Page 6
  6. 6. Health technology Assessment(HTA)• Shift in focus from established therapies to new technologies, particularly drugs• Limited data for assessment of effectiveness – Uncertainty about clinical outcome• Payers are increasingly using HTA, including relative effectiveness assessment (REA) and cost- effectiveness analysis (CEA) to inform decisions• Economic aspects, including price, plays a larger role for reimbursement decisions – Specific market access agreements Page 7
  7. 7. New drug introductions 1940-2009 Page 8
  8. 8. Reduced productivity in R&D Page 9
  9. 9. Mean cost of taking a NME to the market• Di Masi 2003 USD 1 billion• Paul et al 2010 USD 1.8 billion• OHE 2011 USD 1.5 billion (all in 2011 prices)• Increasing relative costs of new drugs• New cancer drugs priced at USD 10 000 per month of treatment• Equal to monthly salary for an oncologist in Sweden Page 10
  10. 10. Increasing role for public payment for drugs• Chronic therapy for RA and MS cost 1000 Euro per month• New cancer drugs priced at 10 000 USD per month• Reimbursement is key to market access – And for efficiency and equity• HTA is developing as a major policy instrument to assess value for money Page 11
  11. 11. The major problem for HTA – Lack of data• Regulatory decisions based on clinical trials aimed at investigating if a new drug works as intended – Balancing benefits and risks• HTA aims at investigating how a new drug works in clinical practice compared to relevant alternatives – Population (indication) – Comparator – Outcome• Clinical trials do not provide the necessary data for an assessment of relative effectiveness Page 12
  12. 12. Re-engineering drug development tomeet payer demands• Early HTA advice on development strategies – May help directing R&D towards development of drugs meeting payer objectives• Joint regulatory and HTA advice – May help reducing costs and improving value of clinical trials• But there are limits to produce evidence of relative and cost-effectiveness before the drug comes to the market Page 13
  13. 13. Payer decisions, uncertainty andopportunity cost• More stringent criteria for relative effectiveness and cost-effectiveness, i.e. value for money, may lead to delay of access to valuable drugs and increasing costs for drug development• Relaxed criteria may lead to the introduction of drugs with a negative net health benefit compared to using the resources otherwise• Decisions must be made on incomplete data – A system for follow-up and re-assessment based on “real life data” must be introduced – Value for money depends on how the drugs are used in clinical practice Page 14
  14. 14. Methods for the Estimation of the NICE CostEffectiveness ThresholdKarl Claxton, Steve Martin, Marta Soares, Nigel Rice,Eldon Spackman, SebastianHinde, Nancy Devlin, Peter C Smith, Mark Sculpher CHE January 2013 • “The central or best threshold is estimated to be £18,317 per QALY” • “It is crucial that the cost effectiveness threshold is seen as representing health forgone as the additional costs of new technologies are imposed on the fixed budgets of local commissioners” (MS) • “This study also starts to make the other NHS patients, who ultimately bear the opportunity cost, less abstract so they can be properly taken into account when decisions about new health technologies are being made.” (KC) Page 15
  15. 15. Patients and real life data• Potential patients are interested in access to new medicines regard of their costs• Variations in use of new technologies is seen as unfair and inefficient – NICE was introduced to solve the problem with “post- code” prescribing• Patients are increasingly involved as a stakeholder in decisions about use of drugs – Regulatory decisions about safety – Development of clinical guidelines – Reimbursement decisions Page 16
  16. 16. All decision makers andstakeholders demand real lifedata Page 17
  17. 17. Sir richard doll Smoking causes cancer Bradford Hill criteria for causation Strength of association Consistency Specificity Temporal relationship Biological gradient Plausibility Coherence Experiment (reversibility) Analogy13-02-08 18
  18. 18. Collecting real life data• Data never speaks for themselves – Data versus evidence – Evidence depends on the circumstances• Augmenting clinical trial data – Inclusion of additional variables (PRO, resource utilization) – Parallel data collection outside the clinical trial in an identical population• Registries – prospective, observational cohort studies of patients who have a particular disease and/or are receiving a particular treatment or intervention Page 19
  19. 19. Collecting real life data• Choice of outcome measure – Mortality – Age adjusted mortality – Survival – Quality of life – Other relevant outcome• Work performance – Working versus not working – Reduced productivity at work• Resource utilization – Inside and outside health care Page 20
  20. 20. Analysing real life data• The problems with “causation” must always be considered• The need for improvement in statistical methods for analysing registry data• The need for modelling• Opportunities for randomization in clinical practice should be investigated Page 21
  21. 21. real life data and policy• Coverage by evidence development (CED) – Requirement to collect data part of the reimbursement decision – Data will be used for a revision of the reimbursement decision• Pay for performance (P4P) – Reimbursement is linked to collection of data on outcome Page 22
  22. 22. Issues related to CED and P4P• How common is this is different countries? – Are there provisions for this in legislation• For which types of drugs is it required? – Expensive drugs – Uncertainty about effects – Uncertainty about their use• Who is undertaken and paying for the studies? – What type of studies• How is the link between study result and decision? – Open-ended (CED) – Decided up-front (P4P) Page 23
  23. 23. United Kingdom (NICE)• Experience: MS risk sharing scheme in 2002 – Price linked to outcome in clinical practice – Initiated to solve a “political” problem – Study complicated and inconclusive – Not used for revision of decision• Today – No enthusiasm for CED – Preference for market access agreements which mainly consists of price discounts (Tarceva,Sutent) – Pay for responders only (Velcade, Erbitux)• Tomorrow – Depends on the design of the VBP scheme Page 24
  24. 24. France• Experience – Long tradition of follow up studies – Mainly to control that indications are followed – No transparent link to decision making• Today – Studies to assess relative effectiveness and cost savings (Risperidol, Januvia) – Secret agreement with the company• Tomorrow – CEA will be mandatory at launch and at revision after five years – More CED studies but no commitment to specific decision Page 25
  25. 25. Germany• Experience – Possible for hospital drugs but not used• Today – Federal Joint Commission and a pharmaceutical company can agree on performing a (real life) study to evaluate the effects of a new drug – The company has to pay for this – Only one example so far• Tomorrow – CED may be more used in the future to follow up results of price negotiations Page 26
  26. 26. Sweden• Experience – CED is used in at least 20 cases by TLV 2002- 12 Evidence on effectiveness and cost-effectivness. – Over-representation Evaluating if the use in clinical praxis follows the of “life style drugs” specified indication. Combination – Follow up data on restricted indications – Follow-up data on cost-effectiveness• Tomorrow – To be used more at the county level Page 27
  27. 27. CED, P4P and CER in the US – Growing importance forEurope Page 28
  28. 28. Why CED and P4P are going to be the newstandard in health care – Outcome matters Page 29
  29. 29. Conclusions• Regulators and HTA/reimbursement agencies/payers have a common objective – Improvements in public health• They have different roles and are guided by different legislation – But closer collaboration can overcome many of the present problems• Real life data is a common interest – For assessment of risk-benefit – For assessment of cost-benefit – For assuring patients that resources are used in an efficient and equitable manner

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