ISPOR 2020: how may we enable faster access to multi-indication orphan products? Rare disease and orphan drug abstract highlights

What research was due to have been presented at the 2020 ISPOR Annual Meeting (18–20 May)? We summarize our abstract highlights, available to view online, focusing on rare diseases and orphan drugs.

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A central aspect of ISPOR meetings is daily poster presentations, during which thousands of international researchers present their poster abstracts of recent research pursuits on topics ranging from cost–effectiveness analyses of treatment protocols for specific disease indications, to reviews of treatment landscapes and potential applications of advanced software to improve patient. Though poster presentations have had to be forgone this year, all abstracts are available to view online>>

Here, we share our abstract highlights from the meeting pertaining to rare diseases and orphan drugs!


Abstract highlights:

How may we enable faster access to multi-indication orphan products?
Why is cost–effectiveness modeling for rare disease treatments so challenging?
The importance of extended and effective payer engagement in orphan drug launches


How may we enable faster access to multi-indication orphan products?

Understanding how a drug’s value evolves across indications throughout its life cycle is an essential aspect of making price, product position and reimbursement decisions for the product. Lack of long-term data on the clinical effectiveness of drug products for various indications, as well as poor market visibility, can create challenges for estimating the value of multi-indication orphan drugs and hence pricing decision making that impacts patient access.

In this study, researchers from Global Pricing Innovations (London, UK) developed a novel tool – comprised of a scenario generator that models likely launch sequences across the USA, UK, France and Germany, built on top of a proprietary value framework – to help support pricing and effective launch planning of early multi-indication orphan products and facilitate early, optimal market access.

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Why is cost–effectiveness modeling for rare disease treatments so challenging?

Compared with the clinical evidence supporting treatments for relatively common diseases, studies for orphan drugs for rare diseases tend to have inferior supporting clinical evidence, which can lead to challenges for payers in determining a treatment’s value for money and in modeling its cost–effectiveness. In this study, researchers from Parexel International (London, UK) reviewed Highly Specialised Technologies appraisals from the National Institute for Health and Care Excellence (NICE; London, UK) and value assessments by the Institute for Clinical and Economic Review (ICER; MA, USA) to identify the most common challenges in cost–effectiveness modeling of rare disease treatments within the UK and USA.

Across 12 eligible Highly Specialised Technologies appraisals and three ICER value assessments evaluated in the study, limited and uncertain clinical data was the most prevalent issue, with challenges associated with assessment of quality of life being the second most common issue. Less common challenges included the capturing of multiple important aspects of a rare disease within the model structure and difficulties with statistical analysis of trial evidence.

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The importance of extended and effective payer engagement in orphan drug launches

There are various challenges associated with launching an orphan drug in the USA, including limited clinical trial data and the clarification of relevant outcomes to payers. To help solve issues arising from a lack of payer engagement, in this study, investigators from ICON plc (London, UK and Nanterre, France) designed and implemented a four-step value communications plan in support of an orphan drug launch of ‘Product X’, which called for early, effective and prolonged payer engagement.

The plan called for a panel of payers and clinicians to validate an evidence generation plan for Product X, a payers’ advisory board to provide their insights into the clinical data and draft value story for Product X following the trial data readout, value story testing via payer interviews and completion of a payer toolkit, value pack, dossier in the Academy of Managed Care Pharmacy (VA, USA) format and global value dossier following US FDA approval of Product X.

The results of the study demonstrate that importance of early and continued payer engagement throughout orphan drug product launch for enhancing communication that promotes the advancement of evidence to action.

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The Evidence Base is proud to be partnering with ISPOR to bring you coverage of the event, including plenary summaries, exclusive interviews with speakers and more! Check out our dedicated channel to view the latest coverage>>

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