scholarly journals Does the orphan medicinal product regulation assist or hinder access to innovative haemophilia treatment in Europe ?

Haemophilia ◽  
2014 ◽  
Vol 20 (4) ◽  
pp. 455-458 ◽  
Author(s):  
B. O'Mahony ◽  
F. Peyvandi ◽  
A. Bok
Keyword(s):  
Medicinal Product ◽  
Orphan Medicinal Product ◽  
Product Regulation

Medicinal Product Regulation: Portugal׳s Framework

2016 ◽  
Vol 38 (9) ◽  
pp. 2118-2126.e2
Author(s):  
Maria Teresa Herdeiro ◽  
Paulo D. Bastos ◽  
António Teixeira-Rodrigues ◽  
Fátima Roque
Keyword(s):  
Medicinal Product ◽  
Product Regulation

scholarly journals Orphan Dugs in EU / Lieky na zriedkavé choroby v EÚ

2013 ◽  
Vol 60 (Supplementum-VIII) ◽  
pp. 26-31 ◽  
Author(s):  
T. Foltánová ◽  
M. Mazág
Keyword(s):  
Medicinal Product ◽  
Small And Medium Enterprises ◽  
Orphan Drugs ◽  
Added Value ◽  
Medicinal Products ◽  
Orphan Medicinal Product ◽  
Whole Process ◽  
Orphan Medicinal Products ◽  
Commission Regulation ◽  
Medium Enterprises

Medical and scientific knowledge about rare diseases is minimal or lacking, thus making research difficulties for pharmaceutical industry. Orphan drugs in EU are under supervision of European Commission, European medical agency (EMA) and Committee for orphan medicinal products (COMP). The presentation provides a brief review of all supportive incentives in the field of orphan medicinal products as: the European orphan medicinal product (OMP) regulation, Guideline on Clinical Trials in Small Populations and Commission Regulation (EC) No 2049/2005 / support of small and medium enterprises (SMEs). It also introduces the concept of Clinical added value of orphan medicinal products, as one of the key instruments to increase the availability of orphan medicinal products in the member states. Separately it stresses the necessity of Health technology assessment implementation in whole process of orphan medicinal product development as well as the implementation of the Europlan indicators into the Slovak National plan

scholarly journals Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases

2020 ◽  
Vol 25 (2) ◽  
pp. 274-291 ◽  
Author(s):  
Maria E. Sheean ◽  
Eva Malikova ◽  
Dinah Duarte ◽  
Giuseppe Capovilla ◽  
Laura Fregonese ◽  
...  
Keyword(s):  
Infectious Diseases ◽  
Medicinal Product ◽  
Orphan Medicinal Product ◽  
Rare Infectious Diseases

scholarly journals Repurposing of Plasminogen: An Orphan Medicinal Product Suitable for SARS-CoV-2 Inhalable Therapeutics

2020 ◽  
Vol 13 (12) ◽  
pp. 425
Author(s):  
Anna Maria Piras ◽  
Ylenia Zambito ◽  
Maurizio Lugli ◽  
Baldassare Ferro ◽  
Paolo Roncucci ◽  
...  
Keyword(s):  
Medicinal Product ◽  
Specific Activity ◽  
Lung Parenchyma ◽  
Fibrinolytic Therapy ◽  
Lung Damage ◽  
Eye Drops ◽  
Orphan Medicinal Product ◽  
Lung Lesions ◽  
Ultrasonic Nebulization ◽  
Ligneous Conjunctivitis

The SARS-CoV-2 infection is associated with pulmonary coagulopathy, which determines the deposition of fibrin in the air spaces and lung parenchyma. The resulting lung lesions compromise patient pulmonary function and increase mortality, or end in permanent lung damage for those who have recovered from the COVID-19 disease. Therefore, local pulmonary fibrinolysis can be efficacious in degrading pre-existing fibrin clots and reducing the conversion of lung lesions into lasting scars. Plasminogen is considered a key player in fibrinolysis processes, and in view of a bench-to-bedside translation, we focused on the aerosolization of an orphan medicinal product (OMP) for ligneous conjunctivitis: human plasminogen (PLG-OMP) eye drops. As such, the sterile and preservative-free solution guarantees the pharmaceutical quality of GMP production and meets the Ph. Eur. requirements of liquid preparations for nebulization. PLG-OMP aerosolization was evaluated both from technological and stability viewpoints, after being submitted to either jet or ultrasonic nebulization. Jet nebulization resulted in a more efficient delivery of an aerosol suitable for pulmonary deposition. The biochemical investigation highlighted substantial protein integrity maintenance with the percentage of native plasminogen band > 90%, in accordance with the quality specifications of PLG-OMP. In a coherent way, the specific activity of plasminogen is maintained within the range 4.8–5.6 IU/mg (PLG-OMP pre-nebulization: 5.0 IU/mg). This is the first study that focuses on the technological and biochemical aspects of aerosolized plasminogen, which could affect both treatment efficacy and clinical dosage delivery. Increasing evidence for the need of local fibrinolytic therapy could merge with the availability of PLG-OMP as an easy handling solution, readily aerosolizable for a fast translation into an extended clinical efficacy assessment in COVID-19 patients.

Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions

2018 ◽  
Vol 23 (1) ◽  
pp. 26-48 ◽  
Author(s):  
Maria E. Sheean ◽  
Violeta Stoyanova-Beninska ◽  
Giuseppe Capovilla ◽  
Dinah Duarte ◽  
Matthias P. Hofer ◽  
...  
Keyword(s):  
Medicinal Product ◽  
Orphan Medicinal Product ◽  
Neurological Conditions
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