Publications by authors named "M Dooms"
Objectives: The aim of the study was to assess the reimbursement policy for orphan drugs (ODs) in selected European countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedures and reimbursement decision-making.
Materials And Methods: A list of authorized ODs was extracted from a web-based registry of the European Medicines Agency, including information on active substance, Anatomical Therapeutic Chemical (ATC) classification code, and therapeutic area. A country-based questionnaire survey was conducted between September 2022 and September 2023 among selected experts from 12 European countries.
Article Synopsis
- The growth of rare disease therapeutics is leading to various innovative treatment options, including orphan medicinal products, medical devices, rehabilitative therapies, and digital therapeutics, all addressing unique patient needs.
- The paper discusses insights from the RE(ACT)-IRDiRC Conference 2023, focusing on orphan medical device development, the associated challenges, and the opportunities presented in this expanding field.
- Examples of groundbreaking devices include an exoskeleton for Duchenne Muscular Dystrophy and a seizure-detecting EEG device, both emphasizing the importance of patient-centric design and the need for increased support in research for rare disease therapies.
Introduction: The Medical Device Regulation (EU)745/2017, increased the regulatory requirements and thus the time and the cost associated with marketing medical devices. For a majority of medical device manufacturers, this has lead to reconsiderations of their product portfolio. The risk of important or essential devices being withdrawn is particularly relevant for pediatric patients and other rare disease patients where limited numbers of devices can be sold and hence the investment needed may not be recovered.
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- - Rare diseases impact over 300 million people globally and are becoming a priority in global health discussions, recognized by the UN and WHO initiatives.
- - Individuals with rare diseases often struggle with accessing essential health services like screening, diagnosis, and treatment, highlighting the importance of awareness and education in primary healthcare.
- - The International Rare Diseases Research Consortium (IRDiRC) is forming a task force to explore ways to enhance the role of primary healthcare providers in overcoming the challenges faced by those with rare diseases.
Rare diseases affect over 400 million people worldwide and less than 5% of rare diseases have an approved treatment. Fortunately, the number of underlying disease etiologies is far less than the number of diseases, because many rare diseases share a common molecular etiology. Moreover, many of these shared molecular etiologies are therapeutically actionable.
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