E-labeling: BIO Comments on Safety Labeling Changes-Implementation of Section 505(o)(4) of the Federal Food, Drug, and Cosmetic Act

April 15, 2019


BIO Submits Comments on FDA Draft Guidance, Rare Diseases: Common Issues in Drug Development

The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the Draft Guidance on Rare Diseases: Common Issues in Drug Development. BIO is the world’s largest trade association representing biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO’s members develop medical products and technologies to treat patients afflicted with serious diseases, to delay the onset of diseases, and to prevent diseases in the first place. BIO commends the FDA for its efforts to update the 2015 Draft Guidance on Rare Diseases: Common Issues in Drug Development. The updated Draft Guidance serves as an important communication tool between the FDA and Sponsors on issues pertaining to rare disease drug development. Such guidance ensures that Sponsors have appropriate information for developing new therapies for rare disease patients, especially given that many rare diseases still do not have an FDA approved treatment. In the following pages of this letter, BIO has included general comments as well as line edits that we believe will make the Draft Guidance more useful for rare disease drug developers.

ICH: BIO Submits Comments on S11 Nonclinical Safety Testing in Support of Development of Pediatric Medicines

One area where the guideline is of particular relevance to BIO, and is thus appreciated, is in establishing both clarity and flexibility around the need and value of studies conducted in nonhuman primates. The language in Section 3.3 that acknowledges the limited value of dedicated studies in postweaning juvenile NHP is supported, as is the final paragraph in Section 4 which specifically addresses the situation of “pediatric first” clinical development in neonates.

REMS: BIO Submits Comments on REMS Assessment: Planning and Reporting

April 2, 2019 Dockets Management Branch (HFA-305) Food and Drug Administration 5630 Fishers Lane, Rm. 1061 Rockville, MD 20852 Re: Docket No. FDA-2018-D-4628-0001: REMS Assessment: Planning and Reporting Dear Sir/Madam, The Biotechnology Innovation Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments to the Draft Guidance on REMS Assessment: Planning and Reporting.

New Drug Cost Poll Shows Top Concern for Voters is Lowering Out-of-Pocket Costs

As policymakers continue to examine ways to make prescription drugs more affordable, the Biotechnology Innovation Organization (BIO), in conjunction with Morning Consult, has released new polling data that show voters’ views on prescription drug costs. Results will be shared with Washington D.C. lawmakers today as BIO hosts its annual fly-in on Capitol Hill.

Senator Roberts Presented Lifetime Achievement Award

Washington, D.C. (April 3, 2019) – The Biotechnology Innovation Organization (BIO) today announced Senator Pat Roberts (R-KS) will be presented with a Lifetime Achievement Award. Senator Roberts...

Siddhartha Mukherjee, author of “The Emperor of All Maladies: A Biography of Cancer” to Open on Keynote Stage at BIO 2019

Pulitzer Prize-winning author, physician and oncologist, Siddhartha Mukherjee with headline BIO 2019 in a keynote speach on Tuesday, June 4.  He is the author of The Emperor of All Maladies: A Biography of Cancer, and the New York Times Best Seller, The Gene: An Intimate History.

BIO's 2019 Special 301 Submission

Section 182 of the Trade Act of 1974, known as the “Special 301” provisions, requires the United States Trade Representative to identify countries that deny adequate and effective intellectual...

BIO Comments on Earnings Releases and Quarterly Reports

The Biotechnology Innovation Organization (BIO) responded to the SEC’s request for comments on the nature and timing of disclosures that reporting companies are required to provide in their quarterly reports. BIO believes that the current quarterly reporting framework places an unhealthy emphasis on meeting or exceeding short-term forecasts, which engenders an inefficient outlook on short-term results. Due to the lengthy timeline for potentially life-saving drug discovery, which averages 10-15 years, biotech companies and their investors would be better served by a less frequent (e.g. semiannual) reporting regime that prioritizes long-term value creation. Biotech companies have unique business models and would benefit from scaled disclosure whereby smaller reporting companies (SRCs) and emerging growth companies (EGCs) report on a less frequent (e.g. semiannual) basis while preserving the flexibility to adopt more frequent (e.g. quarterly) reporting as they advance toward commercial stage.

PFDD: BIO Comments on FDA Draft Guidance Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data

March 21, 2019