In 2000, regulation on orphan medicinal products was adopted in the European Union with the aim of benefiting patients who suffer from serious, rare conditions for which there is currently no satisfactory treatment. Since then, more than 850 orphan drug designations have been granted by the European Commission based on a positive opinion from the Committee for Orphan Medicinal Products (COMP), and more than 60 orphan drugs have received marketing authorization in Europe. Here, stimulated by the tenth anniversary of the COMP, we reflect on the outcomes and experience gained in the past decade, and contemplate issues for the future, such as catalysing drug development for the large number of rare diseases that still lack effective treatments.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1038/nrd3445 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Projections of Public Spending on Pharmaceuticals: A Review of Methods.
Pharmacoeconomics
January 2025
Belgian Health Care Knowledge Centre, Brussels, Belgium.
Background: Forecasting future public pharmaceutical expenditure is a challenge for healthcare payers, particularly owing to the unpredictability of new market introductions and their economic impact. No best-practice forecasting methods have been established so far. The literature distinguishes between the top-down approach, based on historical trends, and the bottom-up approach, using a combination of historical and horizon scanning data.
View Article and Find Full Text PDFMenin Inhibitors: New Targeted Therapies for Specific Genetic Subtypes of Difficult-to-Treat Acute Leukemias.
Cancers (Basel)
January 2025
Hematology Unit, S. Eugenio Hospital (ASL Roma 2), 00122 Rome, Italy.
Menin (MEN1) is a well-recognized powerful tumor promoter in acute leukemias (AL) with KMT2A rearrangements (KMT2Ar, also known as MLL) and mutant nucleophosmin 1 (NPM1m) acute myeloid leukemia (AML). MEN1 is essential for sustaining leukemic transformation due to its interaction with wild-type KMT2A and KMT2A fusion proteins, leading to the dysregulation of KMT2A target genes. MEN1 inhibitors (MIs), such as revumenib, ziftomenib, and other active small molecules, represent a promising new class of therapies currently under clinical development.
View Article and Find Full Text PDFAdopting GPR75 in treating obesity: unraveling the knowns and unknowns of this orphan GPCR.
Trends Cell Biol
January 2025
Center for the Genetics of Host Defense, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA; Division of Endocrinology, Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA. Electronic address:
G protein-coupled receptor 75 (GPR75) is emerging as a promising target for obesity treatment, but its exact role in energy regulation remains unclear. This article explores the latest research on GPR75's molecular function, potential ligands, and therapeutic challenges in addressing obesity.
View Article and Find Full Text PDFBeta Blockers and Septic Shock: More Work to Do.
Chest
January 2025
Bloomsbury Institute of Intensive Care Medicine, Division of Medicine, University College London, London, England.
The Population-Based Incidence and Prevalence of Catatonia.
J Neuropsychiatry Clin Neurosci
January 2025
Department of Psychiatry, Massachusetts General Hospital, and Harvard Medical School, Boston (Luccarelli); Division of Child and Adolescent Psychiatry, Department of Psychiatry and Behavioral Sciences, Vanderbilt University Medical Center at Village of Vanderbilt, and Vanderbilt Kennedy Center, Vanderbilt University, Nashville (Smith); Department of Psychiatry, Beth Israel Deaconess Medical Center, and Harvard Medical School, Boston (Kalinich); Lille Neuroscience and Cognition, University of Lille, Lille, France (Amad); Division of Psychiatry, University College London, London (Rogers).
Objective: Catatonia is a neuropsychiatric disorder that is associated with a range of medical and psychiatric illnesses. Although many single-center studies have been conducted, uncertainty over the population-based incidence and prevalence of the disorder remains. This study reports on the incidence and prevalence rates of catatonia extrapolated from two large epidemiologic studies in the United Kingdom and United States.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!