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1. Slowly progressive, low-prevalence rare diseases with substrate deposition that result from single enzyme defects: providing evidence of effectiveness for replacement or corrective therapies

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, March 2020

Subject(s):

Drug Development -- standards
Drugs, Investigational
Rare Diseases -- drug therapy
Metabolism, Inborn Errors -- drug therapy
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

2. Qualification process for drug development tools: guidance for industry and FDA staff

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, November 2020

Subject(s):

Drug Approval
Drug Development -- methods
Drug Development -- standards
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

3. Pediatric HIV infection: drug product development for treatment

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, March 2019

Subject(s):

Drug Development -- standards
HIV Infections -- drug therapy
Pediatrics
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

4. Delayed graft function in kidney transplantation: developing drugs for prevention

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, July 2019

Subject(s):

Delayed Graft Function -- drug therapy
Drug Development -- standards
Kidney Transplantation
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

5. Drugs for treatment of partial onset seizures: full extrapolation of efficacy from adults to pediatric patients 2 years of age and older

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, September 2019

Subject(s):

Anticonvulsants -- therapeutic use
Clinical Trials as Topic
Drug Development -- standards
Seizures -- drug therapy
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

6. Amyotrophic lateral sclerosis: developing drugs for treatment

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, September 2019

Subject(s):

Amyotrophic Lateral Sclerosis -- drug therapy
Clinical Trials as Topic -- standards
Drug Development -- standards
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

7. Smallpox (variola virus) infection: developing drugs for treatment or prevention

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, November 2019

Subject(s):

Clinical Trials as Topic
Drug Development -- standards
Smallpox -- drug therapy
Smallpox -- prevention & control
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

8. Considerations for the development of dried plasma products intended for transfusion

Publication:

Silver Spring, MD : Center for Biologics Evaluation and Research, December 2019

Subject(s):

Blood Transfusion -- standards
Device Approval
Drug Approval
Drug Development -- standards
Plasma
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration

9. Expansion cohorts: use in first-in-human clinical trials to expedite development of oncology drugs and biologics

Publication:

Silver Spring, MD : Center for Drug Evaluation and Research, March 2022

Subject(s):

Antineoplastic Agents
Biological Products
Clinical Trials as Topic
Drug Development -- legislation & jurisprudence
Drug Development -- standards
Medical Oncology
United States
United States. Department of Health and Human Services
United States. Food and Drug Administration