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ADAPTIVE APPROACHES TO LICENSING, HEALTH TECHNOLOGY ASSESSMENT, AND INTRODUCTION OF DRUGS AND DEVICES

Published online by Cambridge University Press:  12 June 2014

Don Husereau
Affiliation:
Institute of Health Economics; Department of Epidemiology and Community Medicine, University of Ottawa; University for Health Sciences, Medical Informatics, and Technology
Chris Henshall
Affiliation:
Economics Research Group, Brunel University; Health Technology Assessment International Policy Forum
Jamil Jivraj
Affiliation:
Health Technology Assessment International

Abstract

Background: Adaptive approaches to the introduction of drugs and medical devices involve the use of an evolving evidence base rather than conventional single-point-in-time evaluations as a proposed means to promote patient access to innovation, reduce clinical uncertainty, ensure effectiveness, and improve the health technology development process.

Methods: This report summarizes a Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations from invited experts, discussions among attendees about real-world case examples, and background paper.

Results: For adaptive approaches to be understood, accepted, and implemented, the Forum identified several key issues that must be addressed. These include the need to define the goals of and to set priorities for adaptive approaches; to examine evidence collection approaches; to clarify the roles and responsibilities of stakeholders; to understand the implications of adaptive approaches on current legal and ethical standards; to determine costs of such approaches and how they will be met; and to identify differences in applying adaptive approaches to drugs versus medical devices. The Forum also explored the different implications of adaptive approaches for various stakeholders, including patients, regulators, HTA/coverage bodies, health systems, clinicians, and industry.

Conclusions: A key outcome of the meeting was a clearer understanding of the opportunities and challenges adaptive approaches present. Furthermore, the Forum brought to light the critical importance of recognizing and including a full range of stakeholders as contributors to a shared decision-making model implicit in adaptive pathways in future discussions on, and implementation of, adaptive approaches.

Type
Policies
Copyright
Copyright © Cambridge University Press 2014 

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References

1. Baird, L, Teagarden, R, Unger, T, Hirsch, G. New medicines eight years faster to patients: Blazing a new trail in drug development with adaptive licensing. 2013. http://goo.gl/RL1dfZ (accessed March 31, 2014).Google Scholar
2. Baird, LG, Trusheim, MR, Eichler, H-G, Berndt, ER, Hirsch, G. Comparison of stakeholder metrics for traditional and adaptive development and licensing approaches to drug development. Ther Innov Regul Sci. 2013;47:474483.CrossRefGoogle Scholar
3. Boon, WPC, Moors, EHM, Meijer, A, Schellekens, H. Conditional approval and approval under exceptional circumstances as regulatory instruments for stimulating responsible drug innovation in Europe. Clin Pharmacol Ther. 2010 Dec;88:848853.CrossRefGoogle Scholar
4. Carlson, JJ, Sullivan, SD, Garrison, LP, Neumann, PJ, Veenstra, DL. Linking payment to health outcomes: A taxonomy and examination of performance-based reimbursement schemes between healthcare payers and manufacturers. Health Policy. 2010;96:179190.CrossRefGoogle ScholarPubMed
5. Chang, M, Chow, S-C, Pong, A. Adaptive design in clinical research: Issues, opportunities, and recommendations. J Biopharm Stat. 2006;16:299309; discussion 311-312.CrossRefGoogle ScholarPubMed
6. Department of Health and Human Services. Speeding access to important new therapies - fast track, breakthrough therapy, accelerated approval and priority review. http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm (accessed March 31, 2014).Google Scholar
7. Dreyer, NA, Tunis, SR, Berger, M, et al. Why observational studies should be among the tools used in comparative effectiveness research. Health Aff (Millwood). 2010;29:18181825.CrossRefGoogle ScholarPubMed
8. Eichler, H-G, Oye, K, Baird, LG, et al. Adaptive licensing: Taking the next step in the evolution of drug approval. Clin Pharmacol Ther. 2012;91:426437.CrossRefGoogle ScholarPubMed
9. Eijkenaar, F, Emmert, M, Scheppach, M, Schöffski, O. Effects of pay for performance in health care: A systematic review of systematic reviews. Health Policy. 2013;110:115130.CrossRefGoogle ScholarPubMed
10. EMA. European Medicines Agency launches adaptive licensing pilot project. http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2014/03/news_detail_002046.jsp&mid=WC0b01ac058004d5c1 (accessed March 31, 2014).Google Scholar
11. Faulkner, E, Annemans, L, Garrison, L, et al. Challenges in the development and reimbursement of personalized medicine-payer and manufacturer perspectives and implications for health economics and outcomes research: A report of the ISPOR Personalized Medicine Special Interest Group. Value Health. 2012;15:11621171.CrossRefGoogle ScholarPubMed
12. Gerber, A, Stock, S, Dintsios, CM. Reflections on the changing face of German pharmaceutical policy: How far is Germany from value-based pricing? Pharmacoeconomics. 2011;29:549553.CrossRefGoogle ScholarPubMed
13. Green Park Collaborative - Alzheimer's disease pilot. http://www.greenparkcollaborative.org/alzheimers-disease-pilot/ (accessed May 9, 2014).Google Scholar
14. Henshall, C, Mardhani-Bayne, L, Frønsdal, KB, Klemp, M. Interactions between health technology assessment, coverage, and regulatory processes: Emerging issues, goals, and opportunities. Int J Technol Assess Health Care. 2011;27:253260.CrossRefGoogle ScholarPubMed
15. Henshall, C, Schuller, T, HTAi Policy Forum. Health technology assessment, value-based decision making, and innovation. Int J Technol Assess Health Care. 2013;29:353359.CrossRefGoogle ScholarPubMed
16. Henshall, C, Schuller, T, Mardhani-Bayne, L. Using health technology assessment to support optimal use of technologies in current practice: The challenge of “disinvestment.” Int J Technol Assess Health Care. 2012;28:203210.CrossRefGoogle ScholarPubMed
17. HTAi. About the HTAi Policy Forum. http://www.htai.org/index.php?id=643 (accessed March 31, 2014).Google Scholar
18. HTAi. What is HTAi?. http://www.htai.org/index.php?id=420 (accessed March 31, 2014).Google Scholar
19. Husereau, D, Goeree, R, Tsoi, B, Masucci, L, Campbell, K. WHO Pan American Health Organization/Organización Panamericana de la Salud (PAHO/OPS): Synthesis of payer (HTA bodies) / regulator interactions for drugs and medical devices. 2013. http://dl.dropboxusercontent.com/u/27573264/DRAFT_18_Sep_Husereau_v2_Clean.pdf (accessed March 31, 2014).Google Scholar
20. Hutton, J, Trueman, P, Facey, K. Harmonization of evidence requirements for health technology assessment in reimbursement decision making. Int J Technol Assess Health Care. 2008;24:511517.CrossRefGoogle ScholarPubMed
21. Innovative Medicines Initiative 2. The right prevention and treatment for the right patient at the right time. http://www.eibir.org/wp_live_eibir12_km21s/wp-content/uploads/2013/07/IMI2-Strategic_Research_Agenda_v-8-July-2013.pdf (accessed March 31, 2014).Google Scholar
22. Kanavos, P, Reinhardt, U. Reference pricing for drugs: Is it compatible with U.S. health care? Health Aff (Millwood). 2003;22:1630.CrossRefGoogle ScholarPubMed
23. Klemp, M, Frønsdal, KB, Facey, K, HTAi Policy Forum. What principles should govern the use of managed entry agreements? Int J Technol Assess Health Care. 2011;27:7783.CrossRefGoogle Scholar
24. Kreis, J, Schmidt, H. Public engagement in health technology assessment and coverage decisions: A study of experiences in France, Germany, and the United Kingdom. J Health Polit Policy Law. 2013;38:89122.CrossRefGoogle ScholarPubMed
25. Mello, MM, Goodman, SN, Faden, RR. Ethical considerations in studying drug safety–The Institute of Medicine report. N Engl J Med. 2012;367:959964.CrossRefGoogle ScholarPubMed
26. Miller, P. Role of pharmacoeconomic analysis in R&D decision making - When, where, how? Pharmacoeconomics. 2005;23:112.CrossRefGoogle Scholar
27. Musch, G. Early access and market authorization. Brussels; 2013. http://thepharmaceuticalconference.com/_docs/Greet%20Musch%20-%20Early%20access%20and%20market%20authorization.pdf (accessed December 11, 2013.Google Scholar
28. Norman, G. Commentary: Breaking the mold of normative clinical decision making: Is it adaptive, suboptimal, or somewhere in between? Acad Med J Assoc Am Med Coll. 2010;85:393394.CrossRefGoogle ScholarPubMed
29. Rabinovich, M, Wood, F, Shemer, J. Impact of new medical technologies on health expenditures in Israel 2000-07. Int J Technol Assess Health Care. 2007;23:443448.CrossRefGoogle ScholarPubMed
30. Rawlins, M. De testimonio: On the evidence for decisions about the use of therapeutic interventions. Lancet. 2008;372:21522161.CrossRefGoogle Scholar
31. Registri Farmaci sottoposti a monitoraggio [Internet]. Registri Farmaci sottoposti a monitoraggio | AIFA Agenzia Italiana del Farmaco. 2014. http://www.agenziafarmaco.gov.it/it/content/registri-farmaci-sottoposti-monitoraggio (accessed March 31, 2014).Google Scholar
32. Salvatore, D, Buzzetti, R, Baldo, E, Furnari, ML, Lucidi, V, Manunza, D, et al. An overview of international literature from cystic fibrosis registries. Part 4: Update 2011. J Cyst Fibros. 2012;11:480493.CrossRefGoogle ScholarPubMed
33. Tsoi, B, Masucci, L, Campbell, K, et al. Harmonization of reimbursement and regulatory approval processes: A systematic review of international experiences. Expert Rev Pharmacoecon Outcomes Res. 2013;13:497511.CrossRefGoogle ScholarPubMed
34. U.S. FDA. FDA's Sentinel Initiative - Deliverables from completed contracts. http://www.fda.gov/Safety/FDAsSentinelInitiative/ucm149343.htm (accessed March 31, 2014).Google Scholar
35. Walker, S, Sculpher, M, Claxton, K, Palmer, S. Coverage with evidence development, only in research, risk sharing, or patient access scheme? A framework for coverage decisions. Value Health. 2012;15:570579.CrossRefGoogle ScholarPubMed
36. Woodcock, J. Evidence vs. access: Can twenty-first-century drug regulation refine the tradeoffs? Clin Pharmacol Ther. 2012;91:378380.CrossRefGoogle ScholarPubMed

Husereau Supplementary Material

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ADAPTIVE APPROACHES TO LICENSING, HEALTH TECHNOLOGY ASSESSMENT, AND INTRODUCTION OF DRUGS AND DEVICES
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