POLICY sciencesRARE diseasesHEALTH care rationingMore than 8% of the global population is affected by a rare disease and >50% affected are children (30% of whom will die before 5 years). There are 6,000-8,000 classified rare diseases - 80% are genetically based - ...
In 2021, Shenzhen implemented an outpatient-specific disease policy, which included seven rare diseases like hemophilia, pulmonary hypertension, and multiple sclerosis in outpatient-specific disease treatment. The higher reimbursement...
One of the four priority areas outlined in this policy published in 2021 is 'increasing awareness amongst healthcare professionals'. Medics4RareDiseases (M4RD), a charity based in the UK, has proposed a disease-agnostic approach to educating doctors about rare disease, focusing on the common ...
Dealmaking related to the development of therapies for rare diseases has accelerated significantly over the past decade, particularly in the past few years. With the help of DealForma, here we highlight annual rare disease partnership activity (Fig. 1) and high-value rare disease R&D deals signe...
Join the 14th World Congress on Rare Diseases and Orphan Drugs, June 2-3, 2025, in Amsterdam. Explore breakthroughs in research, treatment, and innovation to transform rare disease care globally.
As China continues to expand its policy support for development, registration review and commercializing of orphan drugs, China might become a more desirable market for orphan drugs. [Note] [1] The 2023 China Rare Disease Observation Report issued by Frost & Sullivan on April 27, 2023. ...
The Rare Diseases Epidemiology book is a comprehensive text focused on the rare disease field where both general epidemiological methods and specific methods for low prevalence diseases are discussed. This is the first book to address rare diseases as a public health problem where the epidemiological ...
rare disease community. As we look to the year ahead, we continue to hope and strive for long-awaited breakthroughs in diagnosis, treatment, and policy, while recognizing the critical role of expanding access to rare disease care to all who need it. Wishing you all a healthy and safe 2024...
WEBWIRE –Friday, February 26, 202140 per cent of Swedes with a rare condition, or their family members, state that a relationship has ended due to a rare condition, according to a study by Swedish biopharmaceutical company Sobi. In addition, 63 per cent say that the disease has prevented...
Leveraging data from the Global Burden of Disease Study 2021, the study reported both numbers and age-standardized rates of prevalence, mortality, disability-adjusted life-years (DALYs), years lived with disability, and years of life lost of rIDPs with corresponding 95% uncertainty intervals (UIs...