federal_register: 2019-00677
Data license: Public Domain (U.S. Government data) · Data source: Federal Register API & Regulations.gov API
This data as json
| document_number | title | type | abstract | publication_date | pub_year | pub_month | html_url | pdf_url | agency_names | agency_ids | excerpts | regulation_id_numbers |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2019-00677 | Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry; Availability | Notice | The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Rare Diseases: Common Issues in Drug Development." This draft guidance assists sponsors of drug and biological products intended to treat or prevent rare diseases in conducting more efficient and successful development programs through discussions of selected issues commonly encountered in rare disease drug development. This draft guidance addresses the following important aspects of drug development: Adequate description and understanding of the disease's natural history, adequate understanding of the pathophysiology of the disease and the drug's proposed mechanism of action, nonclinical pharmacotoxicology considerations to support the proposed clinical investigation or investigations, reliable endpoints and outcome assessment, standard of evidence to establish safety and effectiveness, drug manufacturing considerations during drug development, participation of patients, caretakers, and advocates in development programs, and interactions with the Agency. This guidance revises and replaces the draft guidance of the same name issued on August 17, 2015. | 2019-02-01 | 2019 | 2 | https://www.federalregister.gov/documents/2019/02/01/2019-00677/rare-diseases-common-issues-in-drug-development-draft-guidance-for-industry-availability | https://www.govinfo.gov/content/pkg/FR-2019-02-01/pdf/2019-00677.pdf | Health and Human Services Department; Food and Drug Administration | 221,199 | The Food and Drug Administration (FDA or Agency) is announcing the availability of a draft guidance for industry entitled "Rare Diseases: Common Issues in Drug Development." This draft guidance assists sponsors of drug and biological products intended... |