Description
The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled “Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals.” This guidance outlines nonclinical studies recommended for the development of pharmaceuticals used to treat patients with severely debilitating or life-threatening hematologic disorders (SDLTHDs) and addresses comments received to the docket. This guidance is intended to streamline the development of pharmaceuticals used to treat patients with SDLTHDs, other than cancer, while protecting patients' safety and avoiding unnecessary use of animals, in accordance with the 3R (reduce, refine, replace) principles. This guidance applies to pharmaceuticals used both to treat the active disease and to prevent the recurrence of a life-threatening or debilitating event.
Scope & Applicability
Product Classes
7Guidance does not specifically provide recommendations addressing dosage optimization for radiopharmaceuticals
Use of multiple pharmaceuticals together for treatment
Products for which batch/lot information is particularly important
Products requiring specific discussion with CBER; pharmacokinetic/pharmacodynamic considerations are different for these products
Not within the scope of this guidance
dose selection principles described in ICH S6
EFD evaluation typically in two species for small molecules
Stakeholders
1Entity responsible for submitting applications under section 524B
Regulatory Context
Attributes
2Property used to determine maintenance dose intervals and safety monitoring
covariate associated with sex affecting device performance
Related CFR Sections (1)
- 21CFR312.81§ 312.81 Scope.
This section applies to new drug and biological products that are being studied for their safety and effectiveness in treating life-threatening or severely-debilitating diseases.Read full regulation →
See Also (5)
- Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products: Draft Guidance for Industry (Status: Draft)
- IND Submissions for Individualized Antisense Oligonucleotide Drug Products: Administrative and Procedural Recommendations Guidance for Sponsor-Investigators: Draft Guidance for Sponsor-Investigators (Status: Draft)
- Investigational New Drug Application Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Chemistry, Manufacturing, and Controls Recommendations, Guidance for Sponsor-Investigators : Draft Guidance for Industry (Status: Draft)
- IND Submissions for Individualized Antisense Oligonucleotide Drug Products for Severely Debilitating or Life-Threatening Diseases: Clinical Recommendations: Draft Guidance for Industry (Status: Draft)
- Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics (Status: Draft)