Rare Diseases and Difficulty Accessing Orphan Drugs: A Topic That Needs Discussion

Rare Diseases and Difficulty Accessing Orphan Drugs: A Topic That Needs Discussion

Bibliographic Details
Main Author:	José Pedro Vieira
Format:	Article
Language:	English
Published:	Sociedade Portuguesa de Pediatria 2021-04-01
Series:	Portuguese Journal of Pediatrics
Online Access:	https://pjp.spp.pt//article/view/23985

Similar Items

Rare diseases and orphan drugs
by: Domenica Taruscio, et al.
Published: (2011-01-01)

Pricing of orphan drugs in oncology and rare diseases
by: Mark Nuijten, et al.
Published: (2020-01-01)

Rare or orphan diseases: more orphan than rare in Peru
by: Frank Lizaraso Caparó, et al.
Published: (2018-08-01)

Rare pulmonary disease and orphan drugs: a path to the future
by: Sergio Harari, et al.
Published: (2019-10-01)

Patient access to orphan drugs in France
by: Marion Bourdoncle, et al.
Published: (2019-02-01)

Mechanism of coordinated access to orphan drugs
by: DeRidder R, et al.
Published: (2012-11-01)

REASONS FOR DIFFICULTY OF THE PHARMACOECONOMIC ANALYSIS FOR ORPHAN DRUGS. WAYS FOR SOLUTIONS
by: Andrey Vladislavovich Pavlysh, et al.
Published: (2015-09-01)

Orphan diseases — rare but important!
by: article Editorial
Published: (2019-06-01)

Orphan diseases — rare but important!
by: article Editorial
Published: (2019-06-01)

Financing rare or orphan diseases
by: Alfredo Oyola-García, et al.
Published: (2014-12-01)

EVALUATION OF PHARMACISTS’ KNOWLEDGE AND ATTITUDES REGARDING RARE DISEASES AND ORPHAN DRUGS
by: Dušanka Krajnović, et al.
Published: (2013-01-01)

Analysis of patient access to orphan drugs in Turkey
by: Güvenç Koçkaya, et al.
Published: (2021-02-01)

Evaluating health policy and legal responses : how to reduce barriers and improve access to orphan drugs for rare diseases in Canada
by: Blais, Catherine-Marie
Published: (2016)

Ethical questions linked to rare diseases and orphan drugs - A systematic review
by: Kacet, Jaroslav, et al.
Published: (2020)

Pricing and reimbursement of orphan drugs: the need for more transparency
by: Simoens Steven
Published: (2011-06-01)

Financing drug discovery for orphan diseases
by: Gromatzky, Austin A., et al.
Published: (2014)

Ethical Questions Linked to Rare Diseases and Orphan Drugs – A Systematic Review
by: Kacetl J, et al.
Published: (2020-10-01)

Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab.
by: Rosângela Caetano, et al.
Published: (2021-01-01)

Global effort against rare and orphan diseases
by: Marc Humbert, et al.
Published: (2012-09-01)

Educational aspects of rare and orphan lung diseases
by: Tiago M. Alfaro, et al.
Published: (2021-03-01)

Financing for rare or orphan diseases, a thing to do
by: Akram Hernández-Vásquez, et al.
Published: (2014-07-01)

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries
by: Marcin Czech, et al.
Published: (2020-01-01)

Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China
by: Xiao-Qiao Li, et al.
Published: (2018-01-01)

Rare pulmonary diseases and orphan drugs: where do we stand and where are we going to?
by: Sergio Harari, et al.
Published: (2015-09-01)

From Rare to Neglected Diseases: A Sustainable and Inclusive Healthcare Perspective for Reframing the Orphan Drugs Issue
by: Marialuisa Saviano, et al.
Published: (2019-03-01)

Market access of orphan drugs and the role of multi-criteria decision making
by: Simoens Steven
Published: (2012-11-01)

DRUG FLURENISID AS AN IMMUNOMODULATOR FOR THE TREATMENT OF ORPHAN DISEASES
by: Мaria Kovalenko
Published: (2017-12-01)

Similarity in the context of the orphan drug legislation
by: Pereira Franco, Joao Pedro
Published: (2015)

Estimating the clinical cost of drug development for orphan versus non-orphan drugs
by: Kavisha Jayasundara, et al.
Published: (2019-01-01)

To what degree are orphan drugs patient-centered? A review of the current state of clinical research in rare diseases
by: Sally Lanar, et al.
Published: (2020-06-01)

Psychological difficulties encountered by HIV/AIDS orphans in Limpopo Province
by: Nemadondoni, Ndivhudzannyi
Published: (2012)

Correlation of social anhedonia and social anxiety with difficulties in mentalization orphans
by: Volikova S.V., et al.
Published: (2014-10-01)

The difficulties experienced by caregivers of AIDS orphans / Elizabeth Qaliwe Motaung
by: Motaung, Elizabeth Qaliwe
Published: (2009)

The difficulties experienced by caregivers of AIDS orphans / Elizabeth Qaliwe Motaung
by: Motaung, Elizabeth Qaliwe
Published: (2009)

Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen
by: Viviana Giannuzzi, et al.
Published: (2017-04-01)

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
by: Kathleen L. Miller, et al.
Published: (2021-06-01)

New functionalities in Orphanet for orphan drugs, R&D and marketing authorisations to better serve the rare diseases community
by: Aymé Ségolène, et al.
Published: (2010-10-01)

Rare Genetic Diseases with Defects in DNA Repair: Opportunities and Challenges in Orphan Drug Development for Targeted Cancer Therapy
by: Sonali Bhattacharjee, et al.
Published: (2018-09-01)

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries.
by: Todd Gammie, et al.
Published: (2015-01-01)

Patients with rare diseases: from therapeutic orphans to pioneers of personalized treatments
by: Christoph Klein, et al.
Published: (2018-01-01)