Real World Evidence and Artificial Intelligence

RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”

2018 HTAi Annual Meeting – Vancouver

RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”

2018 HTAi Annual Meeting – Vancouver

Innovative Approaches to Evidence Generation – Nancy Dreyerlides

A Primer on RICC: How to Generate Real World Evidence? A Modern Approach – Massoud Toussi

EUnetHTA-EMA Collaboration, RICC Workshop – François Meyer

HTAi Workshop: Risk Sharing Agreements – a company perspective – Mark Godfrey

Publications

Current update: July 2017

The RICC Interest Group has developed the following repository of publically available information. The primary objective of this repository is to provide a regularly updated bibliography of peer-reviewed literature on the key topics in scope of the Interest Group, namely:

Adaptive pathways
Early dialogue / scientific advice involving both regulators and HTA bodies
HTA-Regulatory Interactions relating to evidence development
Managed entry agreements (focussed on outcomes-based agreements)
Performance-linked Risk Sharing Agreements (PBRSAs)
Performance-linked Risk Sharing Agreements (PBRSAs)
Conditional treatment continuation (CTC)
Post-marketing additional data collection

Post-marketing additional data collection

Repository Manager: Michelle Mujoomdar (CADTH)

Information Specialist: David Kaunelis (CADTH)

Detail of the literature search methodology

The literature search was performed by an information specialist. Published literature was identified by searching the following bibliographic database: MEDLINE (1946- ) with in-process records & daily updates via Ovid. The search strategy was comprised of both controlled vocabulary, such as the National Library of Medicine’s MeSH (Medical Subject Headings), and keywords. The main search parameters were health technology assessment and regulatory concepts. Additional terms specific to the RICC key topics were also included, such as: coverage with evidence development, scientific advice, adaptive licensing, adaptive pathways, managed entry and conditional approval. A search of specific agencies contributing to this work (TLV, PBAC, NICE, HAS, G-BA and ZIN) was also incorporated into the strategy. No methodological filters were applied to limit retrieval. Retrieval was limited to English-language citations, published from January 1, 2015 to the present. The initial search was completed on February 4, 2016. A monthly alert has been established to update the search results.

2018

January 2018 – May 2018

Berger ML. The joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making: an interview with Marc Berger. J.2018 Jan;comp. eff. res.. 7(1):11-3. PubMed: PM29052427

Bouvy JC, Sapede C, Garner S. Managed Entry Agreements for Pharmaceuticals in the Context of Adaptive Pathways in Europe. Front Pharmacol. 2018;9:280, 2018. PubMed: PM29636692

Dal-Re R. Could phase 3 medicine trials be tagged as pragmatic? A case study: The Salford COPD trial. J Eval Clin Pract. 2018 Feb;24(1):258-61. PubMed: PM28685913

Dal-Re R, Janiaud P, Ioannidis JPA. Real-world evidence: How pragmatic are randomized controlled trials labeled as pragmatic? BMC Med. 2018 Apr 3;16(1):49, 2018. PubMed: PM29615035

Dubromel A, Geffroy L, Aulagner G, Dussart C. Assessment and diffusion of medical innovations in France: an overview. J Mark Access Health Policy. 2018;6(1):1458575, 2018. PubMed: PM29686802

Garrison LP, Jr., Neumann PJ, Willke RJ, Basu A, Danzon PM, Doshi JA, et al. A Health Economics Approach to US Value Assessment Frameworks-Summary and Recommendations of the ISPOR Special Task Force Report [7. Value Health. 2018 Feb;21(2):161-5. PubMed: PM29477394

Goldberg KB, Blumenthal GM, McKee AE, Pazdur R. The FDA Oncology Center of Excellence and precision medicine. Exp Biol Med (Maywood). 2018 Feb;243(3):308-12. PubMed: PM29105511

de GS, van der LN, Franken MG, Blommestein HM, Leeneman B, van RE, et al. Balancing the Optimal and the Feasible:

A Practical Guide for Setting Up Patient Registries for the Collection of Real-World Data for Health Care Decision Making Based on Dutch Experiences. Value Health. 2017 Apr;20(4):627-36. PubMed: PM28408005

Hanna E, Toumi M, Dussart C, Borissov B, Dabbous O, Badora K, et al. Funding breakthrough therapies: A systematic review and recommendation. Health Policy. 2018 Mar;122(3):217-29. PubMed: PM29223847 Hofer MP, Hedman H, Mavris M, Koenig F, Vetter T, Posch M, et al. Marketing authorisation of orphan medicines in Europe from 2000 to 2013. Drug Discov Today. 2018 Feb;23(2):424-33. PubMed: PM29074441

Jokura Y, Yano K, Yamato M. Comparison of the new Japanese legislation for expedited approval of regenerative medicine products with the existing systems in the USA and European Union. J Tissue Eng Regen Med. 2018 Feb;12(2):e1056-e1062. PubMed: PM28211195

Kondo H, Saint-Raymond A, Yasuda N. What to Know About Medicines With New Active Ingredients Approved in FY 2016 / 2016 in Japan and EU: A Brief Comparison of New Medicines Approved in Japan and the EU in 2016. Ther. 2018 Mar;innov. regul. sci.. 52(2):214-9. PubMed: PM29714521 Lexchin J. Quality of evidence considered by Health Canada in granting full market authorisation to new drugs with a conditional approval: a retrospective cohort study. BMJ Open. 2018 Apr 28;8(4):e020377, 2018. PubMed: PM29705760

Makady A, van VA, Jonsson P, Moseley O, d’Andon A, De BA, et al. Using Real-World Data in Health Technology Assessment (HTA) Practice: A Comparative Study of Five HTA Agencies. Pharmacoeconomics. 2018 Mar;36(3):359-68. PubMed: PM29214389

Malone DC, Brown M, Hurwitz JT, Peters L, Graff JS. Real-World Evidence: Useful in the Real World of US Payer Decision Making? How? When? And What Studies? Value Health. 2018 Mar;21(3):326-33. PubMed: PM29566840

Marsh KD, Sculpher M, Caro JJ, Tervonen T. The Use of MCDA in HTA: Great Potential, but More Effort Needed. Value Health. 2018 Apr;21(4):394-7. PubMed: PM29680094

Olberg B, Fuchs S, Panteli D, Perleth M, Busse R. Scientific Evidence in Health Technology Assessment Reports: An In-Depth Analysis of European Assessments on High-Risk Medical Devices. Value Health.2017 Dec;20(10):1420-6. PubMed: PM29241902

Oude RK, Kalkman S, Collier S, Ciaglia A, Worsley SD, Lightbourne A, et al. Series: Pragmatic trials and real world evidence: Paper 3. Patient selection challenges and consequences. J Clin Epidemiol.2017 Sep;89:173-180, 2017 Sep:-180. PubMed: PM28502808

Robinson MF, Mihalopoulos C, Merlin T, Roughead E. CHARACTERISTICS OF MANAGED ENTRY AGREEMENTS IN AUSTRALIA. Int J Technol Assess Health Care. 2018 Jan;34(1):46-55. PubMed: PM29277174

Sun J, Hu CJ, Stuntz M, Hogerzeil H, Liu Y. A review of promoting access to medicines in China – problems and recommendations. BMC Health Serv Res. 2018 Feb 20;18(1):125, 2018. PubMed: PM29458428 Sun X, Tan J, Tang L, Guo JJ, Li X. Real world evidence: experience and lessons from China. BMJ. 2018 Feb 5;360:j5262, 2018 02 05. PubMed: PM29437644

Tafuri G, Lucas I, Estevao S, Moseley J, d’Andon A, Bruehl H, et al. The impact of parallel regulatory-health technology assessment scientific advice on clinical development. Assessing the uptake of regulatory and health technology assessment recommendations. Br J Clin Pharmacol. 2018 May;84(5):1013-9. PubMed: PM29370449

Trifiro G, Marciano I, Ingrasciotta Y. Interchangeability of biosimilar and biological reference product: updated regulatory positions and pre- and post-marketing evidence. Expert Opin Biol Ther. 2018 Mar;18(3):309-15. PubMed: PM29186988

Tuffaha HW, Scuffham PA. The Australian Managed Entry Scheme: Are We Getting it Right? Pharmacoeconomics.2018 May;36(5):555-65. PubMed: PM29478116

2017

(Annemans L, Ayme S, Le CY, Facey K, Gunther P, Nicod E, et al. Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL). Orphanet J Rare Dis. 2017 Mar 10;12(1):50, 2017. PubMed: PM28283046

Banzi R, Gerardi C, Bertele’ V, Garattini S. Conditional approval of medicines by the EMA. BMJ. 2017 May 2;357:j2062, 2017 May 02. PubMed: PM28465401

Berger ML, Sox H, Willke RJ, Brixner DL, Eichler HG, Goettsch W, et al. Good Practices for Real-World Data Studies of Treatment and/or Comparative Effectiveness: Recommendations from the Joint ISPOR-ISPE Special Task Force on Real-World Evidence in Health Care Decision Making. Value Health. 2017 Sep;20(8):1003-8. PubMed: PM28964430

Berger ML, Sox H, Willke RJ, Brixner DL, Eichler HG, Goettsch W, et al. Good practices for real-world data studies of treatment and/or comparative effectiveness: Recommendations from the joint ISPOR-ISPE Special Task Force on real-world evidence in health care decision making. Pharmacoepidemiol Drug Saf. 2017 Sep;26(9):1033-9. PubMed: PM28913966

Bucek PM, Visnansky M, Mackovicova S, Tomek D. Drug Policy in Slovakia. Value Health Reg Issues. 2017 Sep;13:44-49, 2017 Sep:-49. PubMed: PM29073987

Brooks SS. Regulation and Device Development: Tips for Optimizing Your Experience With the Food and Drug Administration. Tech Vasc Interv Radiol. 2017 Jun;20(2):109-15. PubMed: PM28673647

Carbonneil C. New French Coverage with Evidence Development for Innovative Medical Devices: Improvements and Unresolved Issues. Value Health. 2017 Jan;20(1):178-9. PubMed: PM28212960

Carlson JJ, Chen S, Garrison LP, Jr. Performance-Based Risk-Sharing Arrangements: An Updated International Review. Pharmacoeconomics. 2017 Oct;35(10):1063-72. PubMed: PM28695544

Chen YC, Cheng HF, Yeh MK. Cell Therapy Regulation in Taiwan. Cell Transplant. 2017 Mar 13;26(3):483-92. PubMed: PM27697103

Ciani O, Wilcher B, van GA, Taylor RS. Linking the Regulatory and Reimbursement Processes for Medical Devices: The Need for Integrated Assessments. Health Econ. 2017 Feb;26 Suppl 1:13-29, 2017 Feb:-29.

Culig J, Antolic S, Szkultecka-Debek M. Drug Policy in Croatia. Value Health Reg Issues. 2017 Sep;13:27-30, 2017 Sep:-30. PubMed: PM29073984

Fuchs S, Olberg B, Panteli D, Perleth M, Busse R. HTA of medical devices: Challenges and ideas for the future from a European perspective. Health Policy. 2017 Mar;121(3):215-29. PubMed: PM27751533

Degtiar I. A review of international coverage and pricing strategies for personalized medicine and orphan drugs. Health Policy. 2017 Dec;121(12):1240-8. PubMed: PM29033060

De Groot S, van der LN, Franken MG, Blommestein HM, Leeneman B, van RE, et al. Balancing the Optimal and the Feasible: A Practical Guide for Setting Up Patient Registries for the Collection of Real-World Data for Health Care Decision Making Based on Dutch Experiences. Value Health. 2017 Apr;20(4):627-36. PubMed: PM28408005

Ferrario A, Araja D, Bochenek T, Catic T, Danko D, Dimitrova M, et al. The Implementation of Managed Entry Agreements in Central and Eastern Europe: Findings and Implications. Pharmacoeconomics. 2017 Dec;35(12):1271-85. PubMed: PM28836222

Goble JA, Ung B, van Boemmel-Wegmann S, Navarro RP, Parece A. Performance-Based Risk-Sharing Arrangements: U.S. Payer Experience. J Manag Care Spec Pharm.2017 Oct;23(10):1042-52. PubMed: PM28944728

Greenhalgh T, Fahy N, Shaw S. The Bright Elusive Butterfly of Value in Health Technology Development Comment on “Providing Value to New Health Technology: The Early Contribution of Entrepreneurs, Investors, and Regulatory Agencies”. Int.2017 May 29;j. health policy manag.. 7(1):81-5. PubMed: PM29325407

Gronning N. Data Management in a Regulatory Context. Front Med (Lausanne).2017;4:114, 2017. PubMed: PM28785556

Grossmann N, Del Paggio JC, Wolf S, Sullivan R, Booth CM, Rosian K, et al. Five years of EMA-approved systemic cancer therapies for solid tumours-a comparison of two thresholds for meaningful clinical benefit. Eur J Cancer. 2017 Sep;82:66-71, 2017 Sep:-71. PubMed: PM28648700

Inokuma Y. Pharmacovigilance of Regenerative Medicine Under the Amended Pharmaceutical Affairs Act in Japan. Drug Saf. 2017 Jun;40(6):475-82. PubMed: PM28299610

Irving E, van den BR, Welsing P, Walsh V, fonso-Cristancho R, Harvey C, et al. Series: Pragmatic trials and real world evidence: Paper 7. Safety, quality and monitoring. J Clin Epidemiol. 2017 Nov;91:6-12, 2017 Nov:-12. PubMed: PM28502812

Kalkman S, van Thiel GJMW, Zuidgeest MGP, Goetz I, Pfeiffer BM, Grobbee DE, et al. Series: Pragmatic trials and real world evidence: Paper 4. Informed consent. J Clin Epidemiol. 2017 Sep;89:181-187, 2017 Sep:-187. PubMed: PM28502809

Kawalec P, Tesar T, Vostalova L, Draganic P, Manova M, Savova A, et al. Pharmaceutical Regulation in Central and Eastern European Countries: A Current Review. Front Pharmacol.2017;8:892, 2017. PubMed: PM29326583

Kempf E, Bogaerts J, Lacombe D, Liu L. ‘Mind the gap’ between the development of therapeutic innovations and the clinical practice in oncology: A proposal of the European Organisation for Research and Treatment of Cancer (EORTC) to optimise cancer clinical research. Eur J Cancer. 2017 Nov;86:143-149, 2017 Nov:-149. PubMed: PM28987771

Khozin S, Blumenthal GM, Pazdur R. Real-world Data for Clinical Evidence Generation in Oncology. J Natl Cancer Inst. 2017 Nov 1;109(11), 2017 Nov 01). PubMed: PM29059439

Kristensen FB, Lampe K, Wild C, Cerbo M, Goettsch W, Becla L. The HTA Core Model-10 Years of Developing an International Framework to Share Multidimensional Value Assessment. Value Health. 2017 Feb;20(2):244-50. PubMed: PM28237203

Lach K, Dziwisz M, Remuzat C, Toumi M. Towards a more transparent HTA process in Poland: new Polish HTA methodological guidelines. J Mark Access Health Policy. 2017;5(1):1355202, 2017. PubMed: PM28804603

Lehoux P, Miller FA, Daudelin G, Denis JL. Providing Value to New Health Technology: The Early Contribution of Entrepreneurs, Investors, and Regulatory Agencies. Int. 2017 Jan 25;j. health policy manag.. 6(9):509-18. PubMed: PM28949463

Maignen F, Osipenko L, Pinilla-Dominguez P, Crowe E. Regulatory watch: Outcomes of early health technology assessment dialogues in medicinal product development. Nat Rev Drug Discov. 2017 Feb 2;16(2):79, 2017. PubMed: PM28148947

Maignen F, Osipenko L, Pinilla-Dominguez P, Crowe E. Integrating health technology assessment requirements in the clinical development of medicines: the experience from NICE scientific advice. Eur J Clin Pharmacol. 2017 Mar;73(3):297-305. PubMed: PM27942759

Makady A, De BA, Hillege H, Klungel O, Goettsch W, (on behalf of GetReal Work Package. What Is Real-World Data? A Review of Definitions Based on Literature and Stakeholder Interviews. Value Health. 2017 Jul;20(7):858-65. PubMed: PM28712614

Makady A, Ham RT, De BA, Hillege H, Klungel O, Goettsch W, et al. Policies for Use of Real-World Data in Health Technology Assessment (HTA): A Comparative Study of Six HTA Agencies. Value Health. 2017 Apr;20(4):520-32. PubMed: PM28407993

Meinecke AK, Welsing P, Kafatos G, Burke D, Trelle S, Kubin M, et al. Series: Pragmatic trials and real world evidence: Paper 8. Data collection and management. J Clin Epidemiol. 2017 Nov;91:13-22, 2017 Nov:-22. PubMed:

Miller KL, Woodcock J. Value Assessment in the Regulatory Context. Value Health. 2017 Feb;20(2):296-8. PubMed: PM28237213

Nazareth T, Ko JJ, Sasane R Frois C, Carpenter S, Demean S, et al. Outcomes-Based Contracting Experience: Research Findings from U.S. and European Stakeholders. J Manag Care Spec Pharm. 2017 Oct;23(10):1018-26. PubMed: PM28944734

Neyt M, Baeyens H, Pouppez C, Slegers P, Hulstaert F, Stordeur S, et al. Introduction of high-risk medical devices: national measures that can be taken under the current European legislation to put the patient interest central. Expert Rev Med Devices. 2017 Mar;14(3):181-8. PubMed: PM28128008

Novakovic T, Martin AP, Parker M, Ferrario A, Vukovic S, Landa K, et al. The value of innovation in decision-making in health care in Central Eastern Europe – The Sixth International Conference, 2 June 2017, Belgrade, Serbia. Expert rev. 2017 Dec;pharmacoecon. outcomes res. 17(6):519-21. PubMed: PM28946800

Oude RK, Kalkman S, Collier S, Ciaglia A, Worsley SD, Lightborne A, et al. Series: Pragmatic trials and real world evidence: Paper 3. Patient selection challenges and consequences. J Clin Epidemoil. 2017 Sep;89:173-180. PubMed: PM28502808

Pace J, Ghinea N, Kerridge I, Lipworth W. Caution needed in introduction of provisional approvals for medicines. Intern Med J. 2017 Nov;47(11):1321-4. PubMed: PM29105267

Parvizi N, Parvizi S. New Health Technologies: A UK Perspective Comment on “Providing Value to New Health Technology: The Early Contribution of Entrepreneurs, Investors, and Regulatory Agencies”. Int. 2017 May 9;j. health policy manag.. 6(12):721-2. PubMed: PM29172379

Schwartz JL. Real-World Evidence, Public Participation, and the FDA. Hastings Cent Rep. 2017 Nov;47(6):7-8. PubMed: PM29171057

Pauwels K, Huys I, Vogler S, Casteels M, Simoens S. Managed Entry Agreements for Oncology Drugs: Lessons from the European Experience to Inform the Future. Front Pharmacol.2017;8:171, 2017. PubMed: PM28420990

Shiroiwa T, Fukuda T, Ikeda S, Takura T. New decision-making processes for the pricing of health technologies in Japan: The FY 2016/2017 pilot phase for the introduction of economic evaluations. Health Policy. 2017 Aug;121(8):836-41. PubMed: PM28687183

Smith JA, Brindley DA. Conditional Approval Pathways: The “Special” Case of Global Regenerative Medicine Regulation. Rejuvenation Res. 2017 Feb;20(1):1-3. PubMed: PM28125919

Tarricone R, Torbica A, Drummond M, MedtecHTA Project Group. Key Recommendations from the MedtecHTA Project. Health Econ. 2017 Feb;26 Suppl 1:145-152, 2017 Feb:-152. PubMed: PM28139086

Toumi M, Jaroslawski S, Sawada T, Kornfeld A. The Use of Surrogate and Patient-Relevant Endpoints in Outcomes-Based Market Access Agreements : Current Debate. Appl Health Econ Health Policy. 2017 Feb;15(1):5-11. PubMed: PM27581118

Vella BP, Ermisch M, Godman B, Martin AP, Van Den BJ, Bezmelnitsyna L, et al. Adaptive Pathways: Possible Next Steps for Payers in Preparation for Their Potential Implementation. Front Pharmacol. 2017;8:497, 2017. PubMed: PM28878667

PubMed: PM29105267

Watson R. EU agrees tighter standards for medical devices. BMJ. 2016 Jun 1;353:i3058, 2016 Jun 01. PubMed: PM27251309

van de Wetering EJ, van EJ, Brouwer WB. The Challenge of Conditional Reimbursement: Stopping Reimbursement Can Be More Difficult Than Not Starting in the First Place! Value Health. 2017 Jan;20(1):118-25. PubMed: PM28212952

Wilk N, Wierzbicka N, Skrzekowska-Baran I, Mocko P, Tomassy J, Kloc K. Study types and reliability of Real World Evidence compared with experimental evidence used in Polish reimbursement decision-making processes. Public Health. 2017 Apr;145:51-58, 2017 Apr:-58. PubMed: PM28359391

Woolacott N, Corbett M, Jones-Diette J, Hodgson R. Methodological challenges for the evaluation of clinical effectiveness in the context of accelerated regulatory approval: an overview. J Clin Epidemiol. 2017 Oct;90:108-118, 2017 Oct:-118. PubMed: PM28709997

Worsley SD, Oude RK, Irving E, Lejeune S, Mol K, Collier S, et al. Series: Pragmatic trials and real world evidence: Paper 2. Setting, sites, and investigator selection. J Clin Epidemiol. 2017 Aug;88:14-20, 2017 Aug:-20. PubMed: PM28502811

Yao X, Ding J, Liu Y, Li P. The New Drug Conditional Approval Process in China: Challenges and Opportunities. Clin Ther. 2017 May;39(5):1040-51. PubMed: PM28431767

Yeung K, Li M, Carlson JJ. Using Performance-Based Risk-Sharing Arrangements to Address Uncertainty in Indication-Based Pricing. J Manag Care Spec Pharm. 2017 Oct;23(10):1010-5. PubMed: PM28944729

Yu JS, Chin L, Oh J, Farias J. Performance-Based Risk-Sharing Arrangements for Pharmaceutical Products in the United States: A Systematic Review. J Manag Care Spec Pharm.2017 Oct;23(10):1028-40. PubMed: PM28944733

Zuidgeest MGP, Goetz I, Groenwold RHH, Irving E, van Thiel GJMW, Grobbee DE, et al. Series: Pragmatic trials and real world evidence: Paper 1. Introduction. J Clin Epidemiol. 2017 Aug;88:7-13, 2017 Aug:-13. PubMed: PM28549929

Zuidgeest MGP, Welsing PMJ, van Thiel GJMW, Ciaglia A, fonso-Cristancho R, Eckert L, et al. Series: Pragmatic trials and real world evidence: Paper 5. Usual care and real life comparators. J Clin Epidemiol. 2017 Oct;90:92-98, 2017 Oct:-98. PubMed: PM28694123

2016

Ali J, Califf R, Sugarman J. Anticipated Ethics and Regulatory Challenges in PCORnet: The National Patient-Centered Clinical Research Network. Account Res. 2016;23(2):79-96. PubMed: PM26192996

Basu A, Axelsen K, Grabowski DC, Meltzer DO, Polsky D, Ridley DB, et al. Real-World Data: Policy Issues Regarding their Access and Use. Med Care. 2016 Dec;54(12):1038-44. PubMed: PM27489028

Boucaud-Maitre D, Altman JJ. Do the EMA accelerated assessment procedure and the FDA priority review ensure a therapeutic added value? 2006-2015: a cohort study. Eur J Clin Pharmacol. 2016 Oct;72(10):1275-81. PubMed: PM27473682

Bouvy JC, Jonsson P, Longson C, Crabb N, Garner S. Health Technology Assessment in the Context of Adaptive Pathways for Medicines in Europe: Challenges and Opportunities. Clin Pharmacol Ther. 2016 Dec;100(6):594-7. PubMed: PM27530105

Brandes A, Schwarzkopf L, Rogowski WH. USING CLAIMS DATA FOR EVIDENCE GENERATION IN MANAGED ENTRY AGREEMENTS. Int J Technol Assess Health Care. 2016 Jan;32(1-2):69-77. PubMed: PM26975757

Ermisch M, Bucsics A, Vella BP, Arickx F, Bybau A, Bochenek T, et al. Payers’ Views of the Changes Arising through the Possible Adoption of Adaptive Pathways. Front Pharmacol. 2016;7:305, 2016. PubMed: PM27733828:

Faulkner SD, Lee M, Qin D, Morrell L, Xoxi E, Sammarco A, et al. Pricing and reimbursement experiences and insights in the European Union and the United States: Lessons learned to approach adaptive payer pathways. Clin Pharmacol Ther. 2016 Dec;100(6):730-42. PubMed: PM27626221

Gad M, Kriza C, Fidler A, Kolominsky-Rabas P. Accessing the medical devices market in Egypt and Saudi Arabia: a systematic review of policies and regulations. Expert Rev Med Devices. 2016 Jul;13(7):683-96. PubMed: PM27268662

Husereau D, Henshall C, Sampietro-Colom L, Thomas S. CHANGING HEALTH TECHNOLOGY ASSESSMENT PARADIGMS? Int J Technol Assess Health Care. 2016 Jan;32(4):191-9. PubMed: PM27766998

Leverkus F, Chuang-Stein C. Implementation of AMNOG: An industry perspective. Biom J. 2016 Jan;58(1):76-88. PubMed: PM26332597

Martelli N, van den BH, Borget I. New French Coverage with evidence Development for Innovative Medical Devices: Improvements and Unresolved Issues. Value Health. 2016 Jan;19(1):17-9. PubMed: PM26797231

Martinalbo J, Bowen D, Camarero J, Chapelin M, Demolis P, Foggi P, et al. Early market access of cancer drugs in the EU. Ann Oncol. 2016 Jan;27(1):96-105. PubMed: PM26487583

McDonald L, Lambrelli D, Wasiak R, Ramagopalan SV. Real-world data in the United Kingdom: opportunities and challenges. BMC Med. 2016;14(1):97, 2016. Available from: PubMed: PM4921013

Meier A, Faulkner SD, Schoonderbeek C, Jong B, Kung J, Brindley D, et al. An assessment of implications of adaptive licensing for pharmaceutical intellectual property and regulatory exclusivity rights in the European Union. Clin Pharmacol Ther. 2016 Dec;100(6):743-53. PubMed: PM27626890

Oye KA, Eichler HG, Hoos A, Mori Y, Mullin TM, Pearson M. Pharmaceuticals Licensing and Reimbursement in the European Union, United States, and Japan. Clin Pharmacol Ther. 2016 Dec;100(6):626-32. PubMed: PM27618128 Pita R, Ehmann F, Papaluca M. Nanomedicines in the EU-Regulatory Overview. AAPS J. 2016 Nov;18(6):1576-82. PubMed: PM27527889

Rey-Ares L, Hernandez-Vasquez A, Garay OU, Pichon RA, Garcia MS, Gilardino R, et al. Medical devices: from licensing to coverage. highlights from Argentina, Brazil, Colombia and Mexico. Expert Rev Med Devices. 2016 Nov;13(11):1053-65. PubMed: PM27732123 Ruof J, Staab T, Dintsios CM, Schroter J, Schwartz FW. Comparison of post-authorisation measures from regulatory authorities with additional evidence requirements from the HTA body in Germany – are additional data requirements by the Federal Joint Committee justified? Health Econ Rev. 2016 Dec;6(1):46, 2016. PubMed: PM27687714 Salas-Vega S, Bertling A, Mossialos E. A comparative study of drug listing recommendations and the decision-making process in Australia, the Netherlands, Sweden, and the UK. Health Policy. 2016 Oct;120(10):1104-14. PubMed: PM27665497 Schneeweiss S, Eichler HG, Garcia-Altes A, Chinn C, Eggimann AV, Garner S, et al. Real World Data in Adaptive Biomedical Innovation: A Framework for Generating Evidence Fit for Decision-Making. Clin Pharmacol Ther. 2016 Dec;100(6):633-46. PubMed: PM27627027

Tafuri G, Pagnini M, Moseley J, Massari M, Petavy F, Behring A, et al. How aligned are the perspectives of EU regulators and HTA bodies? A comparative analysis of regulatory-HTA parallel scientific advice. Br J Clin Pharmacol. 2016 Oct;82(4):965-73. PubMed: PM27245362

Thompson M, Henshall C, Garrison LP, Griffin AD, Coyle D, Long S, et al. Targeting improved patient outcomes using innovative product listing agreements: a survey of Canadian and international key opinion leaders. ClinicoEcon. 2016;outcomes res.. 8:427-33, 2016:-33. PubMed: PM27616892 Yue LQ, Campbell G, Lu N, Xu Y, Zuckerman B. Utilizing national and international registries to enhance pre-market medical device regulatory evaluation. J Biopharm Stat. 2016;26(6):1136-45. PubMed: PM27540636 The U.S. FDA focuses on next-generation sequencing: Draft guidance proposals address potential standards for demonstrating analytical validity and criteria for FDA-recognized genetic variant databases. Am J Med Genet A. 2016 Oct;170(10):2496-7. PubMed: PM27643967

2015

Banzi R, Gerardi C, Bertele’ V, Garattini S. Approvals of drugs with uncertain benefit-risk profiles in Europe. Eur. 2015 Oct;J. INTERN. MED.. 26(8):572-84. PubMed: PM26342723

Barker RW, Garner S. Realising the potential of adaptive development of medicines. Rev Recent Clin Trials. 2015;10(1):19-24. PubMed: PM25925883

Brugger U, Horisberger B, Ruckstuhl A, Plessow R, Eichler K, Gratwohl A. Health technology assessment in Switzerland: a descriptive analysis of “Coverage with evidence Development” decisions from 1996 to 2013. BMJ Open. 2015;5(3):e007021, 2015. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4386218PubMed: PM25818273

Bubela T, McCabe C, Archibald P, Atkins H, Bradshaw S, Kefalas P, et al. Bringing regenerative medicines to the clinic: the future for regulation and reimbursement. Regen Med. 2015 Oct;10(7):897-911. PubMed: PM26565607

Capkun G, Lahoz R, Verdun E, Song X, Chen W, Korn JR, et al. Expanding the use of administrative claims databases in conducting clinical real-world evidence studies in multiple sclerosis. Curr Med Res Opin. 2015 May;31(5):1029-39. PubMed: PM25661016

Clarke et al. Toward a Functional Definition of a “Rare Disease” for Regulatory Authorities and Funding Agencies. Value in Health. 2015; 17:757-761 Claxton K, Martin S, Soares M, Rice N, Spackman E, Hinde S, et al. Methods for the estimation of the National Institute for Health and Care Excellence cost-effectiveness threshold. Health Technol Assess. 2015 Feb;19(14):1-503. PubMed: PM25692211

De Lusignan S, Crawford L, Munro N. Creating and using real-world evidence to answer questions about clinical effectiveness. J Innov Health Inform. 2015;22(3):368-73. PubMed: PM26577427

Drummond M. When do performance-based risk-sharing arrangements make sense? Eur J Health Econ. 2015 Jul;16(6):569-71. PubMed: PM25783211

Drummond M. Health technology assessment and its interface with regulation, policy and management. In: Llano-Searis JE, Campillo-Artero C, editors. Health technology assessment and health policy today: a multifaceted view of their unstable crossroads. New York: Springer; 2015. p. 3-14. Eichler HG, Baird LG, Barker R, Bloechl-Daum B, Borlum-Kristensen F, Brown J, et al. From adaptive licensing to adaptive pathways: delivering a flexible life-span approach to bring new drugs to patients. Clin Pharmacol Ther. 2015 Mar;97(3):234-46. PubMed: PM25669457

Facey K, Henshall C, Sampietro-Colom L, Thomas S. IMPROVING THE EFFECTIVENESS AND EFFICIENCY OF EVIDENCE PRODUCTION FOR HEALTH TECHNOLOGY ASSESSMENT. Int J Technol Assess Health Care. 2015 Jan;31(4):201-6. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4697336PubMed: PM26646858

Leverkus F, Chuang-Stein C. Implementation of AMNOG: An industry perspective. Biom J. 2016 Jan;58(1):76-88. PubMed: PM26332597

Fernandez-Lopez S. FDA Draft Guidance on the Naming of Biosimilars. BioDrugs. 2015 Oct;29(5):323-5. PubMed: PM26481940

Gammie T, Lu CY, Babar ZU. Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. PLoS One. 2015;10(10):e0140002, 2015. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4599885 PubMed: PM26451948

Godman B, Malmstrom RE, Diogene E, Gray A, Jayathissa S, Timoney A, et al. Are new models needed to optimize the utilization of new medicines to sustain healthcare systems? Expert Rev Clin Pharmacol. 2015 Jan;8(1):77-94. PubMed: PM25487078

Hofer MP, Jakobsson C, Zafiropoulos N, Vamvakas S, Vetter T, Regnstrom J, et al. Regulatory watch: Impact of scientific advice from the European Medicines Agency. Nat Rev Drug Discov. 2015 May;14(5):302-3. PubMed: PM25881970

Hsu JC, Lu CY. The evolution of Taiwan’s National Health Insurance drug reimbursement scheme. Daru. 2015;23:15, 2015. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4333892PubMed: PM25889754

Hwang TJ and Ciolino JB. Retinal Implants and Medicare Reimbursement Policies for Breakthrough Treatments in Ophthalmology. JAMA Ophthalmology. 2015; 133: 373-374 Hwang TJ, Lehmann LS & Kesselheim AS. Precision medicine and the FDA’s draft guidance on laboratory-developed tests. Nature Biotechnology. 2015; 33 449-451 Jonsson B. Bringing in health technology assessment and cost-effectiveness considerations at an early stage of drug development. Mol Oncol. 2015 May;9(5):1025-33. PubMed: PM25454346

EARLY EVALUATION OF NEW HEALTH TECHNOLOGIES: THE CASE FOR PREMARKET STUDIES THAT HARMONIZE REGULATORY AND COVERAGE PERSPECTIVES. Int J Technol Assess Health Care. 2015 Jan;31(4):207-9. PubMed: PM26560412

Levin L. EARLY EVALUATION OF NEW HEALTH TECHNOLOGIES: THE CASE FOR PREMARKET STUDIES THAT HARMONIZE REGULATORY AND COVERAGE PERSPECTIVES. Int J Technol Assess Health Care. 2015 Jan;31(4):207-9. PubMed: PM26560412

Liberti L, Stolk P, McAuslane N, Somauroo A, Breckenridge AM, Leufkens HG. Adaptive licensing and facilitated regulatory pathways: A survey of stakeholder perceptions. Clin Pharmacol Ther. 2015 Nov;98(5):477-9. PubMed: PM25877216

Lipska I, Hoekman J, McAuslane N, Leufkens HG, Hovels AM. Does conditional approval for new oncology drugs in Europe lead to differences in health technology assessment decisions? Clin Pharmacol Ther. 2015 Nov;98(5):489-91. PubMed: PM26250656

Lu CY, Lupton C, Rakowsky S, Babar ZU, Ross-Degnan D, Wagner AK. Patient access schemes in Asia-pacific markets: current experience and future potential. J. 2015;pharm. policy pract.. 8(1):6, 2015. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4359387PubMed: PM25815200

McCauley JL. Guidelines and Value-Based Decision Making: An Evolving Role for Payers. N c med j. 2015 Sep;76(4):243-6. PubMed: PM26509518

Messner DA, Towse A, Mohr P, Garau M. The future of comparative effectiveness and relative efficacy of drugs: an international perspective. J Comp Eff Res. 2015 4(4):419-27. PubMed: PM25730094

Milne CP, Cohen JP, Felix A, Chakravarthy R. Impact of Postapproval Evidence Generation on the Biopharmaceutical Industry. Clin Ther. 2015 Aug;37(8):1852-8. PubMed: PM26143223

Moloney R, Mohr P, Hawe E, Shah K, Garau M, Towse A. PAYER PERSPECTIVES ON FUTURE ACCEPTABILITY OF COMPARATIVE EFFECTIVENESS AND RELATIVE EFFECTIVENESS RESEARCH. Int J Technol Assess Health Care. 2015 Jan;31(1-2):90-8. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4505735PubMed: PM26168804

Montilla S, Xoxi E, Russo P, Cicchetti A, Pani L. MONITORING REGISTRIES AT ITALIAN MEDICINES AGENCY: FOSTERING ACCESS, GUARANTEEING SUSTAINABILITY. Int J Technol Assess Health Care. 2015 Jan;31(4):210-3. PubMed: PM26646859

Parkinson B, Sermet C, Clement F, Crausaz S, Godman B, Garner S, et al. Disinvestment and Value-Based Purchasing Strategies for Pharmaceuticals: An International Review. Pharmacoeconomics. 2015 Sep;33(9):905-24. PubMed: PM26048353

Rasi G, Bonini S. Innovative medicines: new regulatory procedures for the third millennium. Expert Opin Biol Ther. 2015;15 Suppl 1:S5-8. PubMed: PM25777774

Rawlins MD. National Institute for Clinical Excellence: NICE works. J R Soc Med. 2015 Jun;108(6):211-9. PubMed: PM26085559

Vitry A, Nguyen T, Entwistle V, Roughead E. Regulatory withdrawal of medicines marketed with uncertain benefits: the bevacizumab case study. J. 2015;pharm. policy pract.. 8:25, 2015. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4610052PubMed: PM26483954

Rosano GM, Anker SD, Marrocco W, Coats AJ. Adaptive licensing – A way forward in the approval process of new therapeutic agents in Europe. Int J Cardiol. 2015 Apr 1;184:568-9, PubMed: PM25767018

Sipp D. Conditional approval: Japan lowers the bar for regenerative medicine products. Cell Stem Cell. 2015 Apr 2;16(4):353-6. PubMed: PM25842975

Salmikangas P, Schuessler-Lenz M, Ruiz S, Celis P, Reischl I, Menezes-Ferreira M, et al. Marketing Regulatory Oversight of Advanced Therapy Medicinal Products (ATMPs) in Europe: The EMA/CAT Perspective. Adv Exp Med Biol. 2015;871:103-30. PubMed: PM26374215

Scannell JW, Hinds S, Evans R. Financial returns on R&D: looking back at history, looking forward to adaptive licensing. Rev Recent Clin Trials. 2015;10(1):28-43. PubMed: PM25925881

Simon R, Blumenthal GM, Rothenberg ML, Sommer J, Roberts SA, Armstrong DK, et al. The role of nonrandomized trials in the evaluation of oncology drugs. Clin Pharmacol Ther. 2015 May;97(5):502-7. PubMed: PM25676488

Tsoi B, O’Reilly D, Masucci L, Drummond M, Goeree R. Harmonization of HTA–based reimbursement and regulatory approval activities: a qualitative study. J Popul Ther Clin Pharmacol. 2015;22(1):e78-e89. PubMed: PM25715384

2014

2012-13

2013

Baird et al. Comparison of Stakeholder Metrics for Traditional and Adaptive Development and Licensing Approaches to Drug Development. Ther. Inn. Reg. Sci. 2013; 47:474-483
De Jong et al. Appropriate evidence for adaptive marketing authorization. Nat. Rev. Drug Discov. 2013; 12:647-648
Forda et al. Priorities for improving drug research, development and regulation. Nat. Rev. Drug Discov. 2013; 12:247-248 Technology Assessment. Value in Health. 2014 [Epub ahead of print]
Henshall et al. Understanding the Role and Evidence Expectations of Health Technology Assessment and Coverage/Payer Bodies: What Are They Looking for, and How and Why Does This Differ From What Regulators Require? Ther. Inn. Reg. Sci. 2013; 48:341-346
Tsoi et al. Harmonization of reimbursement and regulatory approval processes: a systematic review of international experiences, Expert Rev Pharmacoecon. Outcomes Res. 2013; 13:497-511
Wonder et al. Early scientific advice obtained simultaneously from regulators and payers: findings from a pilot study in Australia, Value in Health. 2013; 16:1067-1073

2012

Barker & Garner. Adaptive drug development and licensing. Regulatory Rapporteur. 2012; 9:13-14
Development. Ther. Inn. Reg. Sci. 2013; 47:474-483
Brekenridge et al. Medicines Regulation and Health Technology Assessment. Clin. Phar. Ther. 2012; 87: 152-154
Eichler et al. Adaptive Licensing: Taking the Next Step in the Evolution of Drug Approval. Nature. 2012; 91: 426-437
Frnsdal et al. Interaction initiatives between regulatory, health technology assessment and coverage bodies, and industry. Int. J. Technol. Assess. Health Care. 2012; 28:374-81
Liberti et al. Preparing for regulatory review and reimbursement decisions: a case for cooperation between regulatory authorities and health technology assessment agencies. Pharm. Med. 2012; 23:263-267
Messner & Tunis. Current and future state of FDA-CMS parallel reviews. Clin. Pharmacol. Ther. 2012; 91:383-385

1999-2011

2011

Backhouse et al. Early dialogue between the developers of new technologies and pricing and reimbursement agencies: a pilot study. Value in Health. 2011; 14:608-615
Barlas S. Pitting the FDA against CMS: the good, the bad and the ugly about “parallel review”. Biotechnol Health. 2011; 8:29-30

Eichler et al. Bridging the efficacy-effectiveness gap: a regulator’s perspective on addressing variability of drug response. Nat. Rev. Drug Discov. 2011; 10:495-506
Henshall et al. Interactions between HTA, coverage and regulatory processes: emerging issues, goals and opportunities. Int. J. Technol. Assess. Health Care; 2011; 27:253-60.

Pignatti et al. Regulators, payers and prescribers: can we fill the gaps? The Lancet Oncology. 2011; 12: 930-931

2010

Eichler et al. Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers. Nat. Rev. Drug Discov. 2010; 9:277-91.

1999 – 2009

Battista et al. Health technology assessment and the regulation of medical devices and procedures in Quebec. Int. J. Technol. Assess. Health Care. 1999; 15:593-601

Wang S and Smith J. Potential legal barriers to increasing CMS/FDA collaboration: the law of trade secrets and related considerations. Food and Drug Law Journal. 2003; 58:613-627

Governmental and non-governmental activities of interest to the RICC

The Governmental and non-governmental activities of interest to the RICC provides a list of website links to government and non-government activities that are relevant to the RICC.

At this stage, a simple title and hyperlink is provided although it is intended in the future that a short description will be developed for each of these links. While we endeavour to keep the list current and complete, the information has necessarily been compiled in a non-systematic fashion. If you are aware of activities that are relevant but have not been included in this list we would be grateful if you could let the RICC Repository Manager know so that the appropriate information can be uploaded. If your organisation has been included on the list but you would prefer alternative information presented please also contact us.

WEBINARS

Compare and Contrast RWE Guidelines Across Decision – Makers Including HTAs

VIEW THE RECORDING

April 28, 2021

7:00 a.m. MDT

Panelists:
Ashley Jaksa, Aetion (USA)

Session Details:
The main goal of this webinar is to promote integration and collaboration among RWEAI IG members, who are invited to speak about their projects and work.

Resources & Materials

RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”

2018 HTAi Annual Meeting – Vancouver

RICC workshop (Saturday 15 June 2019) – “A Primer in Regulatory Interactions and Conditional Coverage – How to Generate Real World Evidence – A Modern Approach”