University of Twente Student Theses

Analyzing the development pipeline of medical devices for rare diseases : A mixed method study towards the development scope of medical devices for rare diseases and the perceptions of Key Opinion Leaders in the field of orphan devices

Potman, Kiki (2024) Analyzing the development pipeline of medical devices for rare diseases : A mixed method study towards the development scope of medical devices for rare diseases and the perceptions of Key Opinion Leaders in the field of orphan devices.

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Abstract:	Background: Medical devices play a crucial role in the early diagnosis and treatment of diseases. While numerous medical devices are developed globally, the focus on developing and ensuring accessibility of medical devices for rare diseases is often lacking. In order to gain an understanding of the orphan device development landscape, this study aimed to identify the current landscape of orphan devices in the EU. Methodology: A mixed method design of quantitative and qualitative data sources was used. First a dataset with clinical trials downloaded from clinicaltrials.gov was analyzed. The dataset consisted of 2004 clinical trials. Several data visualization and analytical techniques were used to provide an overview of the dataset. Second, interviews with Key Opinion Leaders (KOL) in the field of orphan devices were conducted. The interviews took place in April and May 2024, 12 interviews were conducted in this period. Participants with different specialties were interviewed, in total the analysis included 9 different specialties. The interviews were conducted via a semi-structured approach. Results: The dataset consisted of 2004 clinical trials that evaluated medical devices developed for at least one rare disease and some in combination with non-rare diseases. Of the 2004 clinical trials, 736 clinical trials evaluated medical devices developed for only one or more rare diseases. The interviews with KOL indicated that the Medical Device Regulation presents additional challenges in development and accessibility of orphan devices. These challenges occur due to the different market dynamics of orphan devices, which makes them vulnerable to change and the requirement for clinical evidence, which is difficult to maintain due to the small patient populations. Conclusion: The MDR poses additional challenges in the development and accessibility of orphan devices in the EU. To enhance the development of orphan devices in the EU, it is necessary to address the challenges perceived by KOL towards the MDR.
Item Type:	Essay (Master)
Faculty:	TNW: Science and Technology
Subject:	70 social sciences in general
Programme:	Health Sciences MSc (66851)
Link to this item:	https://purl.utwente.nl/essays/101766
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