Ethical Drug Development for Rare Childhood Diseases: When There Are Limited But Promising Data in Adults, How to Choose Between Safety or Efficacy Studies?

Author:

Johnson Liza-Marie1ORCID,Duenas Devan M.2ORCID,Wilfond Benjamin S.23

Affiliation:

1. St. Jude Children's Research Hospital;

2. Seattle Children's Research Institute;

3. University of Washington School of Medicine

Publisher

Informa UK Limited

Subject

Health Policy,Issues, ethics and legal aspects

Reference18 articles.

1. Lessons from SLE: children are not little adults

2. Food and Drug Administration (FDA). 2019a. 21 CFR Part 50.52. Clinical investigations involving greater than minimal risk but presenting the prospect of direct benefit to individual subjects. Revised 1 April 2019. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=50&showFR=1&subpartNode=21:1.0.1.1.20.4 (accessed December 24, 2019)

3. Food and Drug Administration (FDA). 2019b. 21 CFR Part 50.53. Clinical investigations involving greater than minimal risk and no prospect of direct benefit to individual subjects, but likely to yield generalizable knowledge about the subjects’ disorder or condition. Revised 1 April 2019. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=50&showFR=1&subpartNode=21:1.0.1.1.20.4 (accessed December 24, 2019)

4. Low-dose interleukin-2 treatment selectively modulates CD4+ T cell subsets in patients with systemic lupus erythematosus

5. Differences in long-term disease activity and treatment of adult patients with childhood- and adult-onset systemic lupus erythematosus

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