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A uniQue platform enabling faster development of treatments for Rare Diseases

Periodic Reporting for period 1 - QRD (A uniQue platform enabling faster development of treatments forRare Diseases)

Reporting period: 2017-01-01 to 2017-06-30

Over the last six months Decodeon has actively worked on a unique solution that helps to enable faster development of rare disease treatments. It is our ambition to help people with a rare disease to understand their condition better by offering new disease insights and enabling them to be part of medical research. Medical research and hence drug development is currently slowed down by the challenges of finding matching study participants. This issue is addressed by bridging the gap between patients and research. The platform allows patients to connect to local clinical trials for their disease and simultaneously facilitates the sharing of knowledge and experiences to advance patients understanding of their condition. Hence, connecting patients and research, ultimately supports the availability of rare disease treatments.

By offering this solution, Decodeon is able to address two problems:
Firstly, currently 95% of all rare diseases have no approved drug treatment. With our solution, drug development can be speed up, hence lowering the respective costs and making it more attractive to initiate research on rare diseases. Secondly, we identified that people with a rare disease feel there is too little disease information available including the real-life consequences of having a rare disease. This lack of information is a key factor in experiencing anxiety caused by uncertainty, as many people simply do not know what to expect from the future. By building platforms that facilitate the exchange of experiences among patients generating new disease insights that help to tackle this uncertainty.
There are approximately 7,000 rare diseases, with new ones being discovered every day, affecting 350 million to 600 million people worldwide. However, due to several factors, such as patients being unaware of research opportunities and potential participants being located far away from each other, researchers face challenges to develop effective treatments for rare diseases. Decodeon solves this problem by fostering the exchange of information and knowledge and helping people to find research studies. The result is being one step closer to speeding up the development of rare disease medicine.

The overall objectives were the fostering of the connection of patients and research, to define the commercial and marketing strategy and to validate and refine the business model.
All objectives are achieved through the work performed in this feasibility study.
To create a platform that fosters this connection, we applied lessons learned from a previous project to a new platform for narcolepsy ( as there is a clear need for improving the Quality of Life for people with narcolepsy. By using the platform patients have the opportunity to (1) share their story with other patients, (2) exchange knowledge and experience with each other in a structured manner and (3) check whether they are eligible to participate in a trial. As a result of our platform patients now have access to unique disease insights and can get connected to trials easily, therefore achieving the objective to connect patients and research.

Another objective was to define the commercial and marketing strategy. We developed a strategy which has created an active community both through our platform, social media and other online channels. By creating a strong value identification and delivering this value to patients we are able to continuously realize the described business purpose.

The result of this feasibility study is a validated and refined business model which addresses the existing issue of delayed clinical trials leading to higher cost of drug development and consequently later access to medication for patients. Our project shows not only the pressing relevance, but offers a validated solution that uniquely combines the needs for both researchers and patients. Additionally, four strategies to leverage the platform and to enhance value creation further, were developed. The feasibility study thus has proven that the platform is sustainable and delivers value to both patients and (clinical) researchers.
The progress made within this project becomes obvious when looking at the achieved results. Currently, there is no existing solution that is people centered, focusing on the needs of people with rare diseases and at the same time aiming to speed up the development of medicine. The validation of our business model shows that there is a market need and that our solution addresses this need. The impact this platform has on the lives of people with a rare disease is subjective, but the connections to research and the respective costs saved, can be measured. With phase III clinical trial costs being as high as $103 million for orphan drugs, there is a large socio-economic benefit to be achieved by reducing clinical trial time, hence lowering respectively the costs and supporting earlier availability of treatments.
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