March 18, 2020 FDA Docket No: FDA-2019-D-4964: FDA Draft Guidance on Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products Dear Sir/Madam: BIO thanks FDA for developing this additional guidance on demonstrating substantial evidence of effectiveness. BIO understands that the new guidance is intended to be complementary to the 1998 guidance entitled Providing Clinical Evidence of Effectiveness for Human Drug and Biological Products[1] by reflecting FDA’s current thinking on trial designs, endpoints, and analysis, and the Agency’s longstanding flexibility when considering the types and amount of evidence for demonstrating effectiveness. In this letter we provide key areas that we request FDA to consider when finalizing the Guidance as well as redline edits that we hope will help clarify some of FDA’s points. The Guidance Should be Combined with Existing FDA Guidance on Effectiveness. BIO believes that the new Draft Guidance provides important information for drug developers, especially given the advances in science and regulatory science since the publication of the 1998 Guidance.[2] However, BIO strongly encourages FDA to consolidate the content of the 1998 Guidance and the new content introduced in the 2019 Draft Guidance into a new, single, revised draft guidance. In several parts of the new Draft Guidance, FDA refers to sections of the 1998 Guidance on the same topic and currently two guidance documents on the topic requires Sponsors, reviewers, and other stakeholders to refer across two documents for the totality of FDA’s thinking on a given issue. Furthermore, FDA will soon be adding to the Agency’s overall guidance on substantial evidence of effectiveness when it publishes the forthcoming Draft Guidance on use of real-world evidence to support safety and effectiveness determinations.[3] At that time, there will be three guidances on this topic when arguably there should be, at most, two (one within the context of…

March 15, 2020 Re: Docket No. FDA–2019-D-4752: FDA Draft Guidance, Pediatric Study Plans for Oncology Drugs: Questions and Answers BIO appreciates FDA’s efforts to develop a document that outlines questions and answers pertaining to Pediatric Study Plans (PSPs) for oncology products especially in light of the upcoming implementation date for the new pediatric oncology requirements. However, it is unclear whether FDA plans to develop a final version of the draft Q&A guidance, given that many of the questions and answers provide information to drug developers regarding how to prepare and submit PSPs for FDA depending upon whether the marketing application is submitted prior to or after August 18, 2020. BIO sees value in FDA finalizing the guidance and making it available even after August 18, 2020, therefore we request FDA update the Draft Guidance based upon feedback from stakeholders to this docket. BIO also notes that as written the Draft Guidance is geared more towards drug developers who may be less familiar with the new pediatric oncology requirements. While BIO recognizes value in ensuring that drug developers who are less familiar with the new requirements have resources needed to prepare for implementation, BIO has identified additional areas that FDA may consider including in a updated version of the Guidance that will also make the guidance a helpful resource for drug developers who are more familiar with the new requirements, including:     In the Draft Guidance FDARA Implementation Guidance for Pediatric Studies of Molecularly Targeted Oncology Drugs: Amendments to Sec. 505B of FD&C Act, FDA indicates that “More definite evaluation of a product, if warranted based upon the initial pediatric evaluation described in the iPSP, may be the subject of a Proposed Pediatric Study request (PPSR). Following review of the PPSR and discussions with the sponsor, FDA may issue a Written Request, if appropriate.” BIO requests that FDA…

In the wake of the COVID-19 outbreak, the Biotechnology Innovation Organization (BIO) joins 111 industry groups across sectors in a letter to federal, state and local lawmakers supporting uniform definitions of "critical infrastructure" as outlined by the Department of Homeland Security and it's Cybersecurity and Infrastructure Security Agency.  Complete and total adoption of these definitions as a floor across the country will ensure much-needed manufacturers can continue to operate, transport products and have a workforce available. If not adopted widely, curfews that do not consider transportation and workforce needs could quickly become significant barriers to not only supply chains, but also actual supplies.  To help in the exchange of resources and manufacturing capacity as companies focus on addressing the pandemic, BIO has created the Coronavirus Hub. The hub allows researchers and companies to share their extra capacity and resources with companies that need them. Visit the Coronavirus Hub at hub.bio.org/landing-page/community-home.