IDEAS home Printed from https://ideas.repec.org/a/spr/pharme/v39y2021i9d10.1007_s40273-021-01050-5.html
   My bibliography  Save this article

Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

Author

Listed:
  • Karen M. Facey

    (Usher Institute, University of Edinburgh, NINE Edinburgh Bioquarter)

  • Jaime Espin

    (Andalusian School of Public Health/Escuela Andaluza de Salud Pública (EASP)
    CIBER of Epidemiology and Public Health (CIBERESP)
    Instituto de Investigación Biosanitaria ibs)

  • Emma Kent

    (National Institute for Health and Care Excellence (NICE))

  • Angèl Link

    (Zorginstituut (ZIN) Nederland)

  • Elena Nicod

    (Bocconi University)

  • Aisling O’Leary

    (Trinity Centre for Health Sciences, St. James’s Hospital)

  • Entela Xoxi

    (Università Cattolica del Sacro Cuore)

  • Inneke Vijver

    (National Institute for Health and Disability Insurance (INAMI))

  • Anna Zaremba

    (Agency for Health Technology Assessment and Tariff System (AOTMiT))

  • Tatyana Benisheva

    (Sofia University)

  • Andrius Vagoras

    (Vilnius University)

  • Sheela Upadhyaya

    (National Institute for Health and Care Excellence (NICE))

Abstract

Background and Objective Enthusiasm for the use of outcomes-based managed entry agreements (OBMEAs) to manage uncertainties apparent at the time of appraisal/pricing and reimbursement of new medicines has waned over the past decade, as challenges in establishment, implementation and re-appraisal have been identified. With the recent advent of innovative treatments for rare diseases that have uncertainties in the clinical evidence base, but which could meet a high unmet need, there has been renewed interest in the potential of OBMEAs. The objective of this research was to review the implementation of OBMEAs for two case studies across countries in the European Union, Australia and Canada, to identify good practices that could inform development of tools to support implementation of OBMEAs. Methods To investigate how OBMEAs are being implemented with rare disease treatments, we collected information from health technology assessment/payer experts in countries that had implemented OBMEAs for either nusinersen in spinal muscular atrophy or tisagenlecleucel in two cancer indications. Operational characteristics of the OBMEAs that were publicly available were documented. Then, the experts discussed issues in implementing these OBMEAs and specific approaches taken to overcome challenges. Results The OBMEAs identified were based on individual outcomes to ensure appropriate use, manage continuation of treatment and in two cases linked to payment schedules, or they were population based, coverage with evidence development. For nusinersen, population-based OBMEAs are documented in Belgium, England and the Netherlands and individual-based schemes in Bulgaria, Ireland, Italy and Lithuania. For tisagenlecleucel, there were population-based schemes in Australia, Belgium, England and France and individual-based schemes in Italy and Spain. Comparison of the OBMEA constructs showed some clear published frameworks and clarity of the uncertainties to be addressed that were similar across countries. Agreements were generally made between the marketing authorisation holder and the payer with involvement of expert physicians. Only England and the Netherlands involved patients. Italy used its long-established, national, web-based, treatment-specific data collection system linked to reimbursement and Spain has just developed such a national treatment registry system. Other countries relied on a variety of data collection systems (including clinical registries) and administrative data. Durations of agreements varied for these treatments as did processes for interim reporting. The processes to ensure data quality, completeness and sufficiency for re-analysis after coverage with evidence development were not always clear, neither were analysis plans. Conclusions These case studies have shown that important information about the constructs of OBMEAs for rare disease treatments are publicly available, and for some jurisdictions, interim reports of progress. Outcomes-based managed entry agreements can play an important role not only in reimbursement, but also in treatment optimisation. However, they are complex to implement and should be the exception and not the rule. More recent OBMEAs have developed document covenants among stakeholders or electronic systems to provide assurances about data sufficiency. For coverage with evidence development, there is an opportunity for greater collaboration among jurisdictions to share processes, develop common data collection agreements, and share interim and final reports. The establishment of an international public portal to host such reports would be particularly valuable for rare disease treatments.

Suggested Citation

  • Karen M. Facey & Jaime Espin & Emma Kent & Angèl Link & Elena Nicod & Aisling O’Leary & Entela Xoxi & Inneke Vijver & Anna Zaremba & Tatyana Benisheva & Andrius Vagoras & Sheela Upadhyaya, 2021. "Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel," PharmacoEconomics, Springer, vol. 39(9), pages 1021-1044, September.
  • Handle: RePEc:spr:pharme:v:39:y:2021:i:9:d:10.1007_s40273-021-01050-5
    DOI: 10.1007/s40273-021-01050-5
    as

    Download full text from publisher

    File URL: http://link.springer.com/10.1007/s40273-021-01050-5
    File Function: Abstract
    Download Restriction: Access to the full text of the articles in this series is restricted.

    File URL: https://libkey.io/10.1007/s40273-021-01050-5?utm_source=ideas
    LibKey link: if access is restricted and if your library uses this service, LibKey will redirect you to where you can use your library subscription to access this item
    ---><---

    As the access to this document is restricted, you may want to search for a different version of it.

    References listed on IDEAS

    as
    1. Amanda Whittal & Michela Meregaglia & Elena Nicod, 2021. "The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment," The Patient: Patient-Centered Outcomes Research, Springer;International Academy of Health Preference Research, vol. 14(5), pages 485-503, September.
    2. Martin Wenzl & Suzannah Chapman, 2019. "Performance-based managed entry agreements for new medicines in OECD countries and EU member states: How they work and possible improvements going forward," OECD Health Working Papers 115, OECD Publishing.
    Full references (including those not matched with items on IDEAS)

    Citations

    Citations are extracted by the CitEc Project, subscribe to its RSS feed for this item.
    as


    Cited by:

    1. Fontrier, Anna-Maria, 2022. "Market access for medicines treating rare diseases: Association between specialised processes for orphan medicines and funding recommendations," Social Science & Medicine, Elsevier, vol. 306(C).
    2. Marcelien H. E. Callenbach & Rick A. Vreman & Aukje K. Mantel-Teeuwisse & Wim G. Goettsch, 2022. "When Reality Does Not Meet Expectations—Experiences and Perceived Attitudes of Dutch Stakeholders Regarding Payment and Reimbursement Models for High-Priced Hospital Drugs," IJERPH, MDPI, vol. 20(1), pages 1-12, December.

    Most related items

    These are the items that most often cite the same works as this one and are cited by the same works as this one.
    1. Marcelien H. E. Callenbach & Rick A. Vreman & Aukje K. Mantel-Teeuwisse & Wim G. Goettsch, 2022. "When Reality Does Not Meet Expectations—Experiences and Perceived Attitudes of Dutch Stakeholders Regarding Payment and Reimbursement Models for High-Priced Hospital Drugs," IJERPH, MDPI, vol. 20(1), pages 1-12, December.
    2. Russo, Pierluigi & Carletto, Angelica & Németh, Gergely & Habl, Claudia, 2021. "Medicine price transparency and confidential managed-entry agreements in Europe: findings from the EURIPID survey," Health Policy, Elsevier, vol. 125(9), pages 1140-1145.
    3. Natalie Bohm & Sarah Bermingham & Frank Grimsey Jones & Daniela C. Gonçalves-Bradley & Alex Diamantopoulos & Jessica R. Burton & Hamish Laing, 2022. "The Challenges of Outcomes-Based Contract Implementation for Medicines in Europe," PharmacoEconomics, Springer, vol. 40(1), pages 13-29, January.
    4. Michela Meregaglia & Elena Nicod & Michael Drummond, 2023. "The estimation of health state utility values in rare diseases: do the approaches in submissions for NICE technology appraisals reflect the existing literature? A scoping review," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(7), pages 1151-1216, September.
    5. Jae Ho Jung & Dae Jung Kim & Kangho Suh & Jaeeun You & Je Ho Lee & Kyung In Joung & Dong Churl Suh, 2021. "International Price Comparisons of Anticancer Drugs: A Scheme for Improving Patient Accessibility," IJERPH, MDPI, vol. 18(2), pages 1-14, January.
    6. Hofmarcher, Thomas & Ericson, Oskar & Lindgren, Peter, 2022. "Cancer in Ireland – Disease Burden, Costs and Access to Medicines," IHE Report / IHE Rapport 2022:4, IHE - The Swedish Institute for Health Economics.
    7. Olina Efthymiadou, 2023. "Health technology assessment criteria as drivers of coverage with managed entry agreements: a case study of cancer medicines in four countries," The European Journal of Health Economics, Springer;Deutsche Gesellschaft für Gesundheitsökonomie (DGGÖ), vol. 24(7), pages 1023-1031, September.
    8. Pierluigi Russo & Matteo Zanuzzi & Angelica Carletto & Annalisa Sammarco & Federica Romano & Andrea Manca, 2023. "Role of Economic Evaluations on Pricing of Medicines Reimbursed by the Italian National Health Service," PharmacoEconomics, Springer, vol. 41(1), pages 107-117, January.

    More about this item

    Statistics

    Access and download statistics

    Corrections

    All material on this site has been provided by the respective publishers and authors. You can help correct errors and omissions. When requesting a correction, please mention this item's handle: RePEc:spr:pharme:v:39:y:2021:i:9:d:10.1007_s40273-021-01050-5. See general information about how to correct material in RePEc.

    If you have authored this item and are not yet registered with RePEc, we encourage you to do it here. This allows to link your profile to this item. It also allows you to accept potential citations to this item that we are uncertain about.

    If CitEc recognized a bibliographic reference but did not link an item in RePEc to it, you can help with this form .

    If you know of missing items citing this one, you can help us creating those links by adding the relevant references in the same way as above, for each refering item. If you are a registered author of this item, you may also want to check the "citations" tab in your RePEc Author Service profile, as there may be some citations waiting for confirmation.

    For technical questions regarding this item, or to correct its authors, title, abstract, bibliographic or download information, contact: Sonal Shukla or Springer Nature Abstracting and Indexing (email available below). General contact details of provider: http://www.springer.com .

    Please note that corrections may take a couple of weeks to filter through the various RePEc services.

    IDEAS is a RePEc service. RePEc uses bibliographic data supplied by the respective publishers.