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
Novel approach to decision making for orphan drugs. [PDF]
Int J Technol Assess Health Care, 2023 Decker B +3 more
europepmc +1 more source
British Journal of Haematology, EarlyView.Left panel: Scheme of the XPAG‐immune thrombocytopenia trial. The dexamethasone (DEX) arm consisted of DEX 40 mg/day for days 1–4 for one to three cycles every 28 days to a maximum of 12 weeks, cycles 2 + 3 were optional. Patients randomised to eltrombopag (ETB) + DEX received eltrombopag in combination with a short course of high‐dose DEX beginning on
Paul Schmidt‐Barbo +24 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
Artificial intelligence streamlines scientific discovery of drug–target interactions
British Journal of Pharmacology, EarlyView.Abstract Drug discovery is a complicated process through which new therapeutics are identified to prevent and treat specific diseases. Identification of drug–target interactions (DTIs) stands as a pivotal aspect within the realm of drug discovery and development. The traditional process of drug discovery, especially identification of DTIs, is marked by
Yuxin Yang, Feixiong Cheng
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
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
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 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
Does Misery Love Company? Evidence from pharmaceutical markets before and after the Orphan Drug Act [PDF]
With substantial fixed costs of drug development, more common conditions can support more products. If additional pharmaceutical products are beneficial, they will attract greater consumption and promote better health, e.g. greater longevity.
Frank R. Lichtenberg, Joel Waldfogel
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