12 Results Found

Background/Objectives: Orphan medicinal products offer essential treatments for rare diseases, but patient access varies across European Union countries despite a common regulatory framework. In Bulgaria, access is primarily through inclusion in the...

Background: Real-world evidence (RWE) can complement clinical trials to inform health technology assessments (HTAs). This study examined the extent to which RWE is considered in HTAs of non-oncology orphan medicinal products across six agencies globa...

Background: Claims included in package inserts (PIs) for medicinal products approved by the US Food and Drug Administration (FDA) constitute the regulatory definition of drugs’ benefits and risks. Objective: We sought to assess the usage of patient-r...

SARS-CoV-2 replicates efficiently in the upper airways during the prodromal stage, resulting in environmental viral shedding from patients with active COVID-19 as well as from asymptomatic individuals. There is a need to find pharmacological interven...

Traditionally, drug discovery utilises a de novo design approach, which requires high cost and many years of drug development before it reaches the market. Novel drug development does not always account for orphan diseases, which have low demand and...

Background: Pharmaceutical innovation plays a vital role in advancing global health. This study evaluates the landscape of pharmaceutical innovation in the European Union (EU) over the 2011–2020 decade. Methods: A retrospective analysis was per...

Background/Objectives: We conducted this study to develop a generic amiodarone tablet pharmaceutically equivalent to the reference drug. This development is crucial for securing a stable supply chain for this orphan drug, which currently faces domest...

Incentives for the development of Orphan Medicinal Products (OMP) development have been introduced in both the European Union and the US, yet there has been a relatively little investigation of the success of these policies. An in-depth analysis of t...

Pleural mesothelioma is a lung diffuse tumor, whose complete resection is unlikely. Consequently, metastases reappear where the primary tumor was removed. This paper illustrates the orphan medicine designation procedure of an intracavitary cisplatin...

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 transfo...

There is an urgent need for new approaches and strategies for the introduction of antioxidant drugs in medicine. Despite hundreds of clinical trials with potential antioxidants, no antioxidant drugs have so far been developed for clinical use; this i...

Perilla, also termed as purple mint, Chinese basil, or Perilla mint, is a flavoring herb widely used in East Asia. Both crude oil and essential oil are employed for consumption as well as industrial purposes. Fatty acids (FAs) biosynthesis and oil bo...