scholarly journals European stakeholder perspectives on challenges to rare disease drug development – a qualitative study

2021 ◽  
Author(s):  
Sarala Joshi ◽  
Jessica Chen ◽  
Mustafa Sultan ◽  
Shefali Singh ◽  
Syed Abedi ◽  
...  
Keyword(s):  
Qualitative Study ◽  
Drug Development ◽  
Rare Disease ◽  
Stakeholder Perspectives

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.

Individuals With the Rare Disease Pemphigus: A Quest for Diagnostic

2018 ◽  
Vol 29 (6) ◽  
pp. 889-899 ◽  
Author(s):  
Yannick Le Hénaff ◽  
Stéphane Héas ◽  
Pascal Joly
Keyword(s):  
Qualitative Study ◽  
Autoimmune Diseases ◽  
Rare Disease ◽  
Medical Professionals ◽  
Medical Work ◽  
Medical Tests

This qualitative study conducted in France of “individuals living with a pemphigus” (ILPs; N = 54) highlights the taxing diagnostic trajectory of those suffering from these rare autoimmune diseases. Beyond enduring a diagnostic period that may prove long, during their numerous medical appointments, these individuals internalize the expectations of the medical professionals who are treating them. In some cases, numerous inconclusive medical tests and, at times, a doctor’s condescension may push the patient toward a process of renunciation. This article relates the ILPs’ critiques of the medical work conducted during the trying diagnostic period.

Effective provider-patient communication of a rare disease diagnosis: A qualitative study of people diagnosed with schwannomatosis

2020 ◽  
Author(s):  
Vanessa L. Merker ◽  
Scott R. Plotkin ◽  
Martin P. Charns ◽  
Mark Meterko ◽  
Justin T. Jordan ◽  
...  
Keyword(s):  
Qualitative Study ◽  
Rare Disease ◽  
Disease Diagnosis ◽  
Patient Communication

scholarly journals Implications of aligning full registration of doctors with medical school graduation: a qualitative study of stakeholder perspectives

BMJ Open ◽  
2016 ◽  
Vol 6 (2) ◽  
pp. e010246 ◽  
Author(s):  
K L Mattick ◽  
K Kaufhold ◽  
N Kelly ◽  
J A Cole ◽  
G Scheffler ◽  
...  
Keyword(s):  
Qualitative Study ◽  
Medical School ◽  
Stakeholder Perspectives ◽  
Medical School Graduation ◽  
School Graduation
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