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    Review of 'Using Interventional Pharmacoeconomic Clinical Trials and Outcomes-Based Contracts to Repurpose Generic Drugs with Cost-Savings'

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    Using Interventional Pharmacoeconomic Clinical Trials and Outcomes-Based Contracts to Repurpose Generic Drugs with Cost-SavingsCrossref
    a proposal for using interventional pharmacoeconomics to help fund the repurposing of generics
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    Using Interventional Pharmacoeconomic Clinical Trials and Outcomes-Based Contracts to Repurpose Generic Drugs with Cost-Savings

    One of the greatest barriers to the repurposing of generic drugs is the lack of a financial model to recover the costs of clinical trials. Academics have acknowledged this “problem of new uses” for many years.[1] And so despite their great medical and cost-saving potential, repurposed generic drugs are referred to as “financial orphans” [2], “highly non-excludable therapies” [3], or “unmonopolisable therapies” [4], that are extremely unlikely to receive the funding needed for regulatory approval. One proposed solution to the problem of new uses is to restrict off-label use and allow reimbursement of the repurposed generic at a higher price for the new indication.[5] Another option is the increased public funding of clinical trials. However, this has been politically, legally and practically difficult to achieve. A financially-innovative solution to the problem of new uses is to leverage the immediate and future cost savings of health insurers. In particular, the novel discipline of interventional pharmacoeconomics (IVPE) allows for self-funding clinical trials by comparing a low-cost intervention (such as a repurposed generic) to expensive standard of care.[6] The cost savings from patients taking the low-cost intervention in one arm can exceed the cost of the trial itself, even if it fails, which means there is no financial risk. However, an IVPE trial requires the substitution of the low-cost intervention for expensive standard of care, and may not be appropriate for all repurposing opportunities, especially where no expensive comparator intervention is available. For this latter situation, it is possible to use outcomes-based incentives similar to prizes called Pay-For-Success (PFS) contracts, Advance Market Commitments (AMC) [7], or Social Impact Bonds (SIBs).[8] Such “prize-like” incentives have been used to accelerate the development of Covid vaccines [9] and antibiotics [10], but not yet for repurposing generic drugs. The IVPE concept has been called a ‘revolving research fund’ in Europe, where the cost-savings of clinical trials are used to fund additional research.[11] Similar cost-saving trials are also being funded by a consortium of health insurers in the Netherlands on a case-by-case basis.[12] It is hereby proposed that establishment of an IVPE + AMC fund by private or public health insurers can develop repurposed generic drugs, which can address the “financial toxicity” of new patented drugs while improving patient outcomes.[13] There are many known IVPE opportunities, for example, comparing repurposed generic IV ketamine to patented esketamine for treatment resistant depression [14] or off-label bevacizumab (Avastin) to ranibizumab (Lucentis) for macular degeneration.[15] There is an opportunity to discover and medically de-risk additional IVPE repurposing candidates for self-funding trials by conducting retrospective studies and meta-analyses of medical records and clinical data.
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      Review information

      10.14293/S2199-1006.1.SOR-LAW.AYCZEW.v1.RDOHKJ
      This work has been published open access under Creative Commons Attribution License CC BY 4.0, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Conditions, terms of use and publishing policy can be found at www.scienceopen.com.

      Medicine,Intellectual property law,Commercial law & legal protection,Biotechnology,Complementary & Alternative medicine,Public health,Life sciences
      Financial Innovation,Self-Sustaining Fund,Outcomes-Based Contracts,Social Impact Bonds,Generic Drug Repurposing,Interventional Pharmacoeconomics,Advance Market Commitments,Financial Toxicity ,Pay-For-Success Contracts,Generic Drug Repositioning

      Review text

      Review of manuscript entitled “Using Interventional Pharmacoeconomic Clinical Trials and Outcomes-Based Contracts to Repurpose Generic Drugs with Cost-Savings”

       

      The authors present a proposal for using interventional pharmacoeconomics to help fund the development of repurposed generics. The article is well-written and presenting an interesting approach that holds some promise – however, there are a lot of structural challenges that may hinder such an approach in practice that are not discussed here. I make some suggestions for considering them in the points below.

      Major points:

      1. The article is well-written but the structure is a bit difficult to follow. The abstract should not contain any information that is not also in the manuscript itself (e.g. references and relevant background information). I would suggest that the abstract is rather an overview or teaser of what the article contains, including that it will outline four steps to make IVPE work. So this should be re-written.

      Minor points:

      1. I would suggest removing references from the abstract
      2. I would suggest to re-define acronyms in the introduction
      3. The 4 steps, step 1: should “treatment protocol” read “study protocol”? Change throughout.
      4. After reading the 4 steps I was left with some questions. What kind of economic information should be provided to the payer? Can the authors be more specific about the kind of calculations / supporting information that should be provided?
      5. It sounds like there may be ethical concerns with getting approval for these trials, let’s say when there is an unmet need that is now newly met by an expensive medicine and this would be taken away in the intervention arm?
      6. In practice, payers are not likely to be one single institution, e.g. there may be a HTA agency, the Ministry of Health, a national health system and / or health insurers. It is only one of the difficulties in operationalizing this scheme: identifying the right person to contact. Perhaps a discussion can be included that discusses the likely barriers and steps needed to overcome these, with an example? But I realise that this may go beyond the scope of this.

      Comments

      I thank you for your time and very much agree with your points or concerns and will address them in an editorial recommendation. 

      2024-05-01 17:21 UTC
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