Description
Prometheus has established through a research In Residence Scheme project with Smartline (Knowledge exchange project of University of Exeter) that the pharmaceutical industry has an unmet need for high quality observational data from people living with rare diseases, which may then be used to support approvals for new pharmaceuticals, and get treatments into the hands of patients with unmet needs, far faster. The outcome of our In Residence Scheme project with Smartline identified a considerable opportunity to conduct a “discrete-choice experiment”, in charged by Dr. Gengyang Tu, with representatives from the pharmaceutical industry to understand their willingness-to-pay for real world data in rare disease. This in turn would act as a proxy for market-level willingness-to-pay for any data collected by Prometheus (details of how we will collect this data are provided below). By undertaking a discrete-choice experiment in this setting, Prometheus will be far more informed about the demand for real-world evidence within the pharmaceutical sector, the potential revenue associated with the provision of different ‘types’ of real-world data, and will subsequently inform the proposed revenue model and income potential necessary when applying for developmental grant funding or Series A investment. The results of the choice experiment in itself would yield not only a co-authored high impact scientific publication in a high impact area, but also a proprietary statistical model which could act as a tool to inform others considering developing similar offers in rare diseases, of the likely economic value of their offering. In short this would act as a return-on-investment calculator and would represent a new product which has never been made available previously. This tool (the Prometheus Rare Disease Information Demand Simulator or PRIDeS tool) could open doors for further activity in rare diseases for Prometheus and for others operating in this space, making clear, academic and transparent estimations of the revenue streams different products may be able to generate, thereby reducing risk and uncertainty for subsequent larger scale bids with the aim of leading to commercial success.Furthermore, through our wider research and development activity we have established (and will soon have three publications to suggest that) those living with rare diseases have a willingness to share health data. However, patients have only reported a willingness to share real world health data under the right conditions, and with the necessary incentivisation to do so. We therefore now need to develop our first prototype survey-based approach into a working prototype known herein as the Prometheus Rare disease Insights and Metrics toolkit (PRIME), and conduct a pilot of the PRIME platform with three cohort(s) of rare disease patients, with vastly different conditions, namely, Sickle Cell Disease, Lupus and Fibromyalgia.
The aim of this follow-on research is therefore five-fold:
• Firstly, we aim to determine empirically, through a representative sample of pharmaceutical executives, whether the willingness-to-pay estimates obtained in the research in residence scheme, are generalisable across the sector and represent feasible estimates of potential revenue streams for those collecting health data in rare diseases.
• Secondly, we wish to publish this research in a peer-reviewed journal co-authored with the University of Exeter, and in doing so, create and disseminate the PRIDeS tool, from the resulting analysis.
• Thirdly, we aim to determine the feasibility and acceptability of collecting real-world health data among those living with rare diseases day-to-day. In doing so, a prototype (or MVP) will be created, named previously as the PRIME tool, which will represent a novel product and service available to those with rare diseases, beyond the scope of this proposal.
• Fourthly, we aim to publish our findings from the implementation of the PRIME tool in a peer-reviewed journal, co-authored with the University of Exeter, highlighting not only the successes (and areas for improvement), but also the resultant value of the data collected, with reference to the PRIDeS tool. This will serve as a first-of-its-kind study where those living with rare diseases are empowered to collect and share their personal health data and be remunerated at a fair market rate.
• Finally, the combination of the prior four aims will result in a situation where Prometheus are in a position to seek significant development funding opportunities from SBRI or Innovate UK, in the knowledge that key risks associated with the project have been assuaged in previous projects coordinated by Smartline. Once this project is completed, Prometheus will be able to objectively (and with reference to published studies) highlight that (1) those living with rare diseases are willing to share personal health data under the right conditions, (2), that these conditions have been explored and built into the PRIME tool, (3) that the pharmaceutical industry is willing to pay for this type of data to support their efforts in achieving medicines approval, with reference to the PRIDeS tool, (4) that a prototype of the PRIME tool was capable of collecting real-world data across three different rare diseases, and (5) that learning has been achieved in terms of how to improve the PRIME tool for future commercial success.
| Period | 17 Aug 2022 → 20 Feb 2023 |
|---|---|
| Work for | University of Exeter, United Kingdom |
| Degree of Recognition | International |