BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Severely Debilitating or Life-Threatening Hematologic Disorders: Nonclinical Development of Pharmaceuticals. The draft guidance is a positive step toward articulating a streamlined, science-based approach for nonclinical development of pharmaceuticals. BIO supports the FDA’s goal of getting safe, effective biopharmaceutical products to patients while avoiding the unnecessary use of animals. On the whole, BIO supports the draft guidance. While BIO understands the draft guidance is specific to severely debilitating or life-threatening hematologic disorders (SDLTHDs), BIO believes it could be used as a model for other disease areas. In the comments, BIO provides high-level comments to strengthen and clarify areas including examples of severely debilitating or life-threatening hematologic disorders, incorporation of ICH S9 guideline, and nonclinical studies vs. nonclinical assessments.

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Pregnant Women: Scientific and Ethical Considerations for Inclusion in Clinical Trials Guidance for Industry. This guidance is an important step towards providing information to patients and providers about the safety and efficacy of therapies for pregnant women. The guidance provides insight to sponsors on how and when to include pregnant women in drug development clinical trials. BIO provided general and detailed comments to clarify the draft guidance, including what types of studies it applies to, terminology such as the meaning of "nonpregnant women," and how to analyze and interpret data obtained from pregnant women in clinical trials that are not for medical products specifically indicated for pregnant women.

BIO submitted comments to the Food and Drug Administration’s (FDA) on Tissue Agnostic Therapies in Oncology: Regulatory Considerations for Orphan Drug Designation, following the Public Workshop on May 9, 2018. While advances in genomics and precision medicine have significantly improved the ability to develop medicines aimed at specific molecular targets that may be present in multiple diseases and/or cancers, there is much to be learned through continued research and development. BIO appreciates the FDA taking a forward-looking approach and having early discussions about how these advances may affect other aspects of drug development and review, such as orphan drug designations. In comments following the public workshop, BIO provided several items for the FDA’s consideration, including on the importance of the Orphan Drug Act as well as how to encourage innovation for novel therapies to treat rare diseases. BIO encourages the FDA to consider how to apply a tissue agnostic approach outside of oncology. BIO also asks the FDA to partner with stakeholders to discuss the impacts of tissue agnostic therapies on drug development and review.