Rare essentials: drugs for rare diseases as essential medicines
Bulletin of the World Health Organization, 2006 Since 1977, the WHO Model List of Essential Medicines (EML), published by WHO, has provided advice for Member States that struggle to decide which pharmaceutical technologies should be provided to patients within their public health systems.
Pieter Stolk +2 more
doaj
Project Optimus: Is the US Food and Drug Administration Waiving Dose Optimization for Orphan Drugs? [PDF]
JAMA Oncol, 2023 Wesevich A, Ratain MJ.
europepmc +1 more source
Is there a role for cannabidiol in obesity, metabolic syndrome and binge eating?
British Journal of Pharmacology, EarlyView.Cannabidiol (CBD) is one of the most abundant phytocannabinoids isolated from the Cannabis sativa plant. CBD is a lipophilic, non‐intoxicating substance that differently from Δ9‐tetrahydrocannabinol (Δ9‐THC) does not present the typical profile of a drug of abuse.
Luca Botticelli +7 more
wiley +1 more source
Novel approach to decision making for orphan drugs. [PDF]
Int J Technol Assess Health Care, 2023 Decker B +3 more
europepmc +1 more source
Novel approaches for drug development against chronic primary pain: A systematic review
British Journal of Pharmacology, EarlyView.Abstract Chronic primary pain (CPP) persisting for more than 3 months, associated with significant emotional distress without any known underlying cause, is an unmet medical need. Traditional or adjuvant analgesics do not provide satisfactory pain relief for a great proportion of these patients.
Valéria Tékus +5 more
wiley +1 more source
Negotiating Medical Insurance Drug Prices: The Role in Reducing Costs of Orphan Drugs for Rare Diseases. [PDF]
Int J Health Policy Manag, 2023 Lu J, Ying X, Li Z.
europepmc +1 more source
Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review
British Journal of Pharmacology, EarlyView.Abstract In the 2025 novel drug mini‐review, one can take a full measure of the ingenuity that underlies current drug design and development, despite the year's smaller harvest (46 novel drugs) compared to 2024 (53) and 2023 (70). 54% of the novel drugs are first‐in‐class (FIC).
Andreas Papapetropoulos +16 more
wiley +1 more source
Disentangling the Cost of Orphan Drugs Marketed in the United States. [PDF]
Healthcare (Basel), 2023 Althobaiti H +4 more
europepmc +1 more source
Is It Possible to Have Cheaper Drugs and Preserve the Incentive to Innovate: Reforming the Drug Approval Process According to Market Principles [PDF]
This paper argues that drugs are expensive not because of a lack of competition among research-based pharmaceutical companies, but because of a lack of competition in the drug approval process.
Corinne Sauer, Robert Sauer
core
British Journal of Pharmacology, EarlyView.Background and Purpose Intestinal barrier dysfunction caused by mitochondrial stress, oxidative damage and apoptosis, are hallmarks of dysbiosis‐associated gastrointestinal (GI) disorders. Staurosporine causes downstream features of dysbiosis‐induced epithelial damage.
Fatima Maqoud +9 more
wiley +1 more source