Detalhe da pesquisa
1.
Model consent clauses for rare disease research.
BMC Med Ethics
; 20(1): 55, 2019 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31370847
2.
ASPECTS OF PATIENT REPORTED OUTCOMES IN RARE DISEASES: A DISCUSSION PAPER.
Int J Technol Assess Health Care
; 32(3): 126-30, 2016 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-27523091
3.
Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major.
Br J Haematol
; 164(3): 431-7, 2014 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-24180641
4.
Returning incidental findings from genetic research to children: views of parents of children affected by rare diseases.
J Med Ethics
; 40(10): 691-6, 2014 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-24356209
5.
Generating health technology assessment evidence for rare diseases.
Int J Technol Assess Health Care
; 30(4): 416-22, 2014 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-25407328
6.
Personalizing health care: feasibility and future implications.
BMC Med
; 11: 179, 2013 Aug 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-23941275
7.
How can we deliver on the promise of precision medicine in oncology and beyond? A practical roadmap for action.
Health Sci Rep
; 6(6): e1349, 2023 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-37359405
8.
Developing a Framework of Cost Elements of Socioeconomic Burden of Rare Disease: A Scoping Review.
Pharmacoeconomics
; 41(7): 803-818, 2023 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-37029233
9.
The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies.
Ther Adv Rare Dis
; 4: 26330040231188979, 2023.
Artigo
em Inglês
| MEDLINE | ID: mdl-37529076
10.
An international comparative analysis of public reimbursement of orphan drugs in Canadian provinces compared to European countries.
Orphanet J Rare Dis
; 17(1): 113, 2022 03 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-35246200
11.
Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group.
Orphanet J Rare Dis
; 16(1): 308, 2021 07 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-34256816
12.
Potential approaches for the pricing of cancer medicines across Europe to enhance the sustainability of healthcare systems and the implications.
Expert Rev Pharmacoecon Outcomes Res
; 21(4): 527-540, 2021 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-33535841
13.
Strengthening health systems for access to gene therapy in rare genetic disorders.
Mol Ther Methods Clin Dev
; 32(2): 101220, 2024 Jun 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-38516694
14.
Familial hypercholesterolaemia patient support groups and advocacy: A multinational perspective.
Atherosclerosis
; 277: 377-382, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-30270074
15.
Moving from Patient Advocacy to Partnership: A Long and Bumpy Road.
Patient
; 10(3): 271-276, 2017 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-28097637
16.
Review of 11 national policies for rare diseases in the context of key patient needs.
Orphanet J Rare Dis
; 12(1): 63, 2017 03 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-28359278
17.
Research on rare diseases: ten years of progress and challenges at IRDiRC.
Nat Rev Drug Discov
; 21(5): 319-320, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-35079160
18.
Can a health professional represent patient view: patient organization response.
Int J Technol Assess Health Care
; 27(4): 401-2, 2011 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-22004785
19.
Developing a patient-directed policy framework for managing orphan and ultra-orphan drugs throughout their lifecycle.
Patient
; 8(1): 103-17, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25559762
20.
Quality indicators as a tool in improving the introduction of new medicines.
Basic Clin Pharmacol Toxicol
; 116(2): 146-57, 2015 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-25052464