Trends for Performance-based Risk-sharing Arrangements

Author: Shuxian Chen

Chen_Shuxian
CHOICE Student Shuxian Chen

When considering the approval of new drugs, devices and diagnostic products, there’s always a tension between making the product’s benefits available to more people and collecting more information in trials. The restrictive design of randomized-controlled trials (RCTs) mean that their indications of effectiveness don’t always hold in the real world. They’re also unlikely to detect long-term adverse events. This uncertainty and risk make it hard for payers to make coverage decisions for new interventions.

Performance-based risk-sharing arrangements (PBRSAs), also known as patient access schemes (PAS), managed entry arrangements, and coverage with evidence development (CED), help to reduce such risk. These are arrangements between a payer and a pharmaceutical, device, or diagnostic manufacturer where the price level and/or nature of reimbursement is related to the actual future performance of the product in either the research or ‘real world’ environment rather than the expected future performance [1].

I recently developed a review paper with CHOICE faculty Josh Carlson and Lou Garrison that gave an update of the trends in PBRSAs both in the US and globally. Using the University of Washington Performance-Based Risk-Sharing Database, we have identified 437 eligible cases between 1993 and 2016 from that contains information obtained by searching Google, PubMed, and government websites. Eighteen cases have been added to the database in 2017 and 2018. Seventy-two cases are from the US.

Figure 1. Eligible cases between 1993-2016 by country

Chen_Bar Graph_March16_2018

Australia, Italy, the US, Sweden and the UK are the five countries that have the largest number of PBRSAs. (Distribution of cases from different countries can be seen in Graph 1.) Except for the US, cases from the other four countries are identified from their government programs: the Pharmaceutical Benefits Scheme (PBS) in Australia, the Italian Medicines Agency (AIFA) in Italy, the Swedish Dental and Pharmaceutical Benefits Agency (TLV) in Sweden, and the National Institute for Health and Care Excellence (NICE) in the UK. These single-payer systems have more power in negotiating drug price with the manufacturer than we do in the US.

Cases in the US are more heterogeneous, with both public (federal/state-level) and private payers involved. The US Centers for Medicare and Medicaid Services (CMS) contributes to 25 (37%) of the 72 US cases. Among these, most arrangements involve medical devices and diagnostic products and originate in the CED program at CMS [2]. This program is used to generate additional data to support national coverage decisions for potentially innovative medical technologies and procedures, as coverage for patients is provided only in the context of approved clinical studies [3]. For pharmaceuticals, there have been few PBRSAs between CMS and manufacturers – no cases established between 2006 and 2016. However, in August 2017, Novartis announced that a first-of-its-kind collaboration with the CMS has been made: a PBRSA for Kymriah™ (tisagenlecleucel), their novel cancer treatment for B-cell acute lymphoblastic leukemia that uses the body’s own T cells to fight cancer [4]. The arrangement allows for payment only when participants respond to Kymriah™ by the end of the first month. It can be categorized as performance-linked reimbursement (PLR), as reimbursement is only provided to the manufacturer if the patient meets the pre-specified measure of clinical outcomes. This recent collaboration may lead to a larger number and more variety of PBRSAs between pharmaceutic manufacturers and the CMS.

Please refer to our article for more detailed analyses regarding the trends in PBRSAs.

References:

[1] 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(3): 179–90. doi:10.1016/j.healthpol.2010.02.005.

[2] CMS. Coverage with Evidence Development. Available at: https://www.cms.gov/Medicare/Coverage/Coverage-with-Evidence-Development/

[3] Neumann PJ, Chambers J. Medicare’s reset on ‘coverage with evidence development’. Health Affairs Blog. 2013 Apr 1. http://healthaffairs.org/blog/2013/04/01/medicares-reset-on-coverage- with-evidence-development/

[4] Novatis. Novartis receives first ever FDA approval for a CAR-T cell therapy, Kymriah(TM) (CTL019), for children and young adults with B-cell ALL that is refractory or has relapsed at least twice. 2017. Available at: https://www.novartis.com/news/media-releases/novartis-receives-first-ever-fda-approval-car-t-cell-therapy-kymriahtm-ctl019

Letter from an Editor

The following emails are real correspondence between myself and an Associate Editor at a prestigious scientific journal, which I have called “Journal X” here, after I published an article in a journal with a very similar title, which I am calling “Journal Y”. I have removed the journal and editor names for privacy.

letter from an editor
Photo by Scott Kidder

Dear Dr. Adamson,

Congratulations on your recent excellent paper. May I ask if you specifically targeted your paper for the “Journal Y” (a relatively new “look-alike” entry to the field in 2013)?  I’m an Associate Editor for the more established “Journal X.” I was curious to what extent authors may be submitting to other “look-alike” journals to intentionally vs. not.

Thx,

Dr. Editor

Associate Editor

“Journal X”


 

Dear Dr. Editor,

Thank you for contacting me about this recently published paper. I am grateful for the opportunity to explain why I chose to submit to the open access “Journal Y” instead of the well-respected “Journal X” and I would like to hear your thoughts. I am cautious about predatory journals, and I consciously wrestled with the pros and cons in journal selection.

I originally prepared this manuscript for submission for “Journal X,” where I thought it would fit nicely in one of the online-only sections. When the manuscript was finalized, I received an invitation to contribute to a special issue of “Journal Y” that included a publication fee waiver.

Having never heard of this look-alike journal before, I read and considered these elements: credibility, cost, ethics, and time to the wide-spread dissemination of the findings.

First, I checked the credibility of “Journal Y” by reading past issues. The quality was good, and they had published a review paper in 2014 by my research heroes Drs. A and B. I thought, “if this journal is good enough for A and B, it is certainly good enough for me.”

I noticed the “Journal Y” had very low or no publication fees for being Open Access, and this signaled a difference from predatory journals with low standards and high profits. Since my current trainee funding does not cover publication fees, the paid Open Access option through your journal was not an option unless I used personal savings.

The University of Washington Biomedical Research Integrity Series lectures on the ethics of responsible publishing changed my opinions about Open Access vs subscription journals. I take seriously my moral responsibility as an HIV researcher to communicate findings in a format accessible to scientists and patients in communities disproportionately affected by this disease. Many of my brilliant economist and mathematician colleagues are based at small institutions and companies with limited funding to purchase articles.

At the end, I am pleased to have chosen “Journal Y” over “Journal X” for several reasons:

  • Less than 8 weeks passed from my submission to publication (including two rounds of revisions)
  • I received rigorous and helpful peer-review comments
  • No cost for Open Access (with invitation to the special issue, or low cost otherwise)
  • Within the first month, the full text has been downloaded more than 200 times in six continents.
  • Last year I submitted a different and very good, in my opinion, paper to your journal. After more than two months, I received a rejection from with peer-review comments that were so mean and personal it was borderline unprofessional. While this look-alike journal does not have the prestige or impact factor of X, I see my younger generation progressively placing more value on the quality of content, free accessibility, dialogues, and citations of individual papers rather than on the sum of journal impact factors on a CV.

Because of these reasons, I am committed to Open Access publishing when I have the opportunity and sufficient funds to do so. I admit there is quite a lot for me to still learn about scientific publications, and so I would greatly appreciate your expert feedback on these considerations.

Thank you for taking the time to read my paper and reach out to me personally with questions.

Sincerely,

Blythe Adamson


 

Dear Dr. Adamson,

Thank you very much for your thoughtful reply.  I’m glad to hear that this was an intentional (vs. accidental) decision. Please also accept my apologies for the “borderline unprofessional” peer review comments you received when submitting to “Journal X”; definitely discouraging to authors.

I will pass on your comments to the other Associate Editors of “Journal X” on our quarterly conference call for discussion on how we can improve and hopefully attract your future papers.

Best regards,

Dr. Editor

Associate Editor

“Journal X”