Orphan drugs are medications that are produced for the treatment of rare diseases. As there is less number of patients, the drug manufacturing companies are not keen in producing these drugs. Due to high costs of research and development and low profitability, companies do not want to invest in manufacturing of orphan drugs. Several laws have been passed by Governments of different nations to encourage the development of orphan drugs and make it available to patients. This study explores the interrelation dynamics of factors that has resulted in the greater availability of orphan drugs in recent times. Ten factors: internet technology, legislation, online patient support groups, government subsidiary, biotechnological advancements, corporate social responsibility, awareness and diagnosis of rare diseases and exclusive budgeting by pharmaceutical industries for orphan drugs related research and development and production were taken for the study. With a sample size of 38 experts, the technique of decision-making trial and evaluation laboratory (DEMATEL) was used for the study. It was found that information technology, legislation, support groups, and budget were the causes and the factors awareness, diagnosis, medicine availability, subsidiary, CSR and biotechnology emerged to be the effect.
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http://dx.doi.org/10.1016/j.pharma.2024.12.005 | DOI Listing |
Acta Biomater
December 2024
Lingang Laboratory, Shanghai 200031, China. Electronic address:
Triple-negative breast cancer (TNBC) has been a clinical challenge due to its high recurrence and metastasis rates. Chemotherapy remains the primary treatment for TNBC after surgery ablation, but it lacks targeted specificity and causes side effects in normal tissues. Receptor tyrosine kinase-like orphan receptor 1 (ROR1) is significantly expressed in TNBC cells, and small interference RNA (siRNA) targeting ROR1 can effectively suppress ROR1 gene expression, thereby inhibiting proliferation and metastasis.
View Article and Find Full Text PDFOrphanet J Rare Dis
December 2024
Medicine for Society, Platform at Amsterdam University Medical Center - University of Amsterdam, Amsterdam, The Netherlands.
Background: The combination of high prices and uncertain effectiveness is a growing challenge in the field of orphan medicines, hampering health technology assessments. Hence, new methods for establishing price benchmarks might be necessary to support reimbursement negotiations. In this study, we applied several pricing models containing cost-based elements to the case of lumasiran for treating primary hyperoxaluria type 1.
View Article and Find Full Text PDFValue Health
December 2024
Institute of Clinical Pharmacy and Pharmaceutical Sciences, College of Medicine, National Cheng Kung University, Tainan, Taiwan; Department of Pharmacy, College of Medicine, National Cheng Kung University, Tainan, Taiwan. Electronic address:
Objective: Given the lack of a value framework for assessing health technologies in Asian settings, a value framework incorporating multiple-criteria decision analysis (MCDA) for new drugs under universal healthcare coverage in Taiwan was established.
Methods: The development process included: 1) the adoption of five value domains (i.e.
BMC Med
December 2024
Clinical Research Institute, Institute of Advanced Clinical Medicine, Peking University, Beijing, China.
Background: The Food and Drug Administration (FDA)'s Accelerated Approval (AA) pathway has increasingly used to authorize market approval of new drugs amid controversy. The present study aims to inform the most recent data on the strength of clinical evidence supporting such approvals.
Methods: Evidentiary characteristics of pre-approval pivotal clinical studies and regulator-required post-approval confirmatory studies supporting AAs between 2015 and 2022 were extracted from publicly available FDA documents.
Retinoid-related orphan receptor-γ (RORγ) is a nuclear receptor that plays important roles in the development and activation of T helper type-17 (Th17) cells. In this study, we characterized the pharmacological profile of JTE-151 ((4S)-6-[(2-chloro-4-methylphenyl)amino]-4-{4-cyclopropyl-5-[cis-3-(2,2-dimethylpropyl)cyclobutyl]isoxazol-3yl}-6-oxohexanoic acid), which is a novel RORγ antagonist identified by our group. JTE-151 dissociated co-activator peptide from the human RORγ-ligand binding domain (LBD) and recruited co-repressor peptide into human RORγ-LBD, and potently inhibited the transcriptional activity of RORγ of human, mouse and rat.
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