Managing High-Cost, Innovative Pharmaceuticals in Asia: Is Something Lost When Translating Theory Into Practice?

Transcription

1 Managing High-Cost, Innovative Pharmaceuticals in Asia: Is Something Lost When Translating Theory Into Practice? The Potential for using Managed Entry Agreements Adrian Towse ISPOR 6 th Asia-Pacific Conference Beijing 6-9 th September 2014 Agenda What are the issues with high-cost innovative pharmaceuticals? Value for Money What are Managed Entry Agreements? The same as PBRSAs? Three or four types of scheme? ISPOR PBRSA Task Force Report What do we know about what works and what doesn t work Summary 2 1

2 What are the issues with high-cost innovative pharmaceuticals? Value for Money Given health effects and the price, is it a good use of resources? Uncertainty. Is there a lot of uncertainty: about the expected health and related effects? as to whether the drug will get to the right sub group of patients? Budget Impact Given the price and potential size of the patient population, is there a large budget impact 3 What elements of value?* Usually recognised Health effects that are well captured Cost offsets Uncertainty Less frequently / consistently recognised Health effects that are less well captured Wider societal impacts Severity /unmet need Process issues Information Innovation *Towse and Barnsley (2013). IJTAHC. 29(4),

3 How aggregated and judged? A decision on value Two types of challenge for decision makers: Scientific uncertainty Value judgements They are weighting multiple criteria relevant to the decision using: Deliberative processes Algorithms How structured could /should this become? 5 Differential pricing* Price transparency plus reference pricing links markets together This makes companies less willing to offer discounts Local value assessment and reference pricing are inconsistent tools Need for confidentiality to get discounts reflecting local value e.g. UK Patient Access Schemes *Danzon, P., Towse, A. and Mestre-Ferrandiz, J.(2013). Health Economics. Epub. doi: /hec

4 Managed Entry Agreements* MEAs are used to give access to new technologies where traditional reimbursement is deemed inappropriate. Three different forms of MEAs have been identified: management of uncertainty relating to clinical and/or costeffectiveness; management of utilization to optimize performance. management of budget impact; The rationale for using these approaches and their advantages and disadvantages differ. All forms of MEA should take the form of a formal written agreement among stakeholders, clearly identifying the rationale for the agreement, aspects to be assessed, methods of data collection and review, and the criteria for ending the agreement. 7 *Klemp, Frønsdal and Facey on behalf of the HTAi Policy Forum (2011) IJTAHC 27:

5 Performance-Based Risk-Sharing Arrangements Good Practices for Design, Implementation, and Evaluation Garrison, L.P., Towse, A., Briggs, A., de Pouvourville, G., Grueger, J., Mohr, P.E., Severens, J.L., Siviero, P. and Sleeper, M. (2013 Report of the ISPOR Good Practices for Performance-based Risk-sharing Task Force. Value in Health. 16(5), Cost sharing arrangement MEA: Payerproducer/provider arrangement Performance based risk sharing arrangements (PBRSA) To manage utilization in the real world To provide evidence regarding decision uncertainty Performance linked reimbursement Coverage with evidence development - Budget capping - Utilization capping - Discounts - Price/volume - Outcomes guarantees - Money back guarantees - Conditional treatment continuation - Process of care - Only with research - Only in research Intermediate endpoint Clinical endpoint Pre-specified agreement No pre-specified agreement 5

6 Performance based risk sharing arrangements To manage utilization in the real world To provide evidence regarding decision uncertainty Performance linked reimbursement Coverage with evidence development - Budget capping - Utilization capping - Discounts - Price/volume - Outcomes guarantees - Money back guarantees - Conditional treatment continuation - Process of care Italy: oncology schemes UK: Velcade, Lucentis etc. - Only with research UK: MS RSS Aus: Bosentan UK: Votrient France: DPP4; risperidone - Only in research US Medicare: LVRS, PET, PTAS Intermediate endpoint Clinical endpoint Pre-specified agreement No pre-specified agreement 11 Possible Uncertainties That Might Addressed by Data Collection with PBRSA Source: ISPOR PBRSA Task Force 1. Efficacy or effectiveness in the tested population as compared to current standard of care; 2. The efficacy or effectiveness in a broader, more heterogeneous population than used in registration trials or in pre-licensing testing; 3. The effects on long-term or more clinically-significant endpoints than those included in registration trials (which in the case of a drug may have used surrogate markers) or in pre-licensing studies (e.g., for procedures or devices); 4. Any adverse effects and adherence issues; 5. Whether health care providers management of the patient will change the relative benefits and harms under conditions of usual care; 6. The size and value of cost-offsets, such as due to fewer hospital visits; 7. The proportion of patients who will respond, i.e., achieve a pre-set (minimum) outcome which may be an intermediate/surrogate endpoint; 8. The numbers and types of patients likely in real-world practice to be treated with the new therapy; 9. Whether the patients treated are the right ones, i.e., they have attributes matching those patients which, on the basis of current evidence, the payer is willing to fund (which may or may not include off-label use). 12 6

7 Pueg-Peiro et al. (2011) conducted a systematic literature review to identify existing knowledge about the costs and benefits, assessed either quantitatively or qualitatively, of PBRSAs. Found little quantitative evidence. Neumann et al. (2011) reviewed five PBRSAs in the US and UK and conclude that they are hard to implement in practice. The results from Italy and other EU countries are also unclear and the schemes are in evolution. Overall, the literature suggests there is an important gap in structured ex post evaluation of PBRSAs. Utilisation schemes appear to have been more successful to date than CED schemes. However, the evidence is limited, mixed, qualitative, and partial. 7

8 Summary High cost innovative pharmaceuticals are one of the challenges facing health systems Assessing local value is key MEAs can be used to address issues of price, uncertainty and budget impact Implementation is difficult, particularly for PBRSAs The alternatives to using these schemes is not an easy option either! 15 Thank you