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BIO Submits Comments on FDA Draft Guidance, Rare Diseases: Common Issues in Drug Development

Rare Diseases • Letters, Testimony & Comments • April 11, 2019

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Organization (BIO) thanks the Food and Drug Administration (FDA) for the opportunity to submit comments regarding the Draft Guidance on Rare Diseases: Common Issues in Drug Development.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 BIO commends the FDA for its efforts to update the 2015 Draft Guidance on Rare Diseases: Common Issues in Drug Development. The updated Draft Guidance serves as an important communication tool between the FDA and Sponsors on issues pertaining to rare disease drug development.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following pages of this letter, BIO has included general comments as well as line edits that we believe will make the Draft Guidance more useful for rare disease drug developers.

Sustainable Fuels, Renewable Fuel Standard (RFS) • Press Release • April 3, 2019

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 will receive the award tonight in conjunction with BIO’s Legislative Day Fly-In. “I am honored to receive the Lifetime Achievement Award from BIO for our work to promote an industry critical to our economic future, our national security and our well-being. It has been my honor to build Kansas’ bio and life science research infrastructure to establish a research economy for future generations of Kansans, to the benefit of the nation. I am proud of the work we have done on the Senate Agriculture Committee to invest and protect innovation in biotechnology, fulfilling our responsibility to feed a troubled and hungry world while also improving the management of our natural resources. It has been equally important to ensure our nation’s plant and animal health are considered a national security priority. The contributions of the bio industry should not be taken for granted, and this Marine will continue to be its champion.” “Senator Roberts understands the value of America’s biotechnology industry, not just on an economic scale, but through the benefits it delivers to patients facing devastating diseases and consumers of America’s agricultural and industrial innovation," said BIO President and CEO Jim Greenwood. “As a leader on both the Senate Committee on Health, Education, Labor, and Pensions and the Senate Committee on Finance, he has been at the cross-section of every major healthcare debate throughout his distinguished career. At every turn he chose to support policies that promote and protect innovation, including the 21st Century Cures Act, Orphan Drug Tax Credit and countless others.” “As a champion for the thousands of Kansans working in the biotechnology industry, Senator Roberts has demonstrated leadership on issues vitally important to our state and the nation,”…

Intellectual Property, Patents, Human Health • Press Release • April 3, 2019

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.

Human Health • Letters, Testimony & Comments • April 2, 2019

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.

Letters, Testimony & Comments • April 1, 2019

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.

Press Release • March 28, 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

Intellectual Property, Patents • Letters, Testimony & Comments • March 27, 2019

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 property protections or fair and equitable market access to U.S. persons who rely on IP protection. As part of the 2019 Special 301 Review process conducted by USTR, Justin Pine prepared BIO’s formal submission and presented oral testimony to USTR and the interagency Special 301 subcommittee highlighting the IP issues facing our members in key markets around the world. The principal concerns highlighted in BIO’s submission are: i) global compulsory licensing trends; ii) lack of adequate or any regulatory data protection; iii) restrictive patentability frameworks; and iv) market access concerns associated with drug pricing controls.

Letters, Testimony & Comments • March 25, 2019

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.

Human Health • Letters, Testimony & Comments • March 21, 2019

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Developing and Submitting Proposed Draft Guidance Relating to Patient Experience Data. In the comments, BIO thanks FDA for promoting partnership between patients, industry, and the FDA by providing patient groups and other stakeholders with a pathway to submit proposed draft guidance and other resources related to patient experience data. BIO encourages FDA to continue to facilitate the open exchange on patient experience data with all stakeholders, which will lead to more meaningful outcomes. BIO believes strong trilateral communication between the FDA, patients/patient organizations, and Sponsors will help better incorporate patient experience data throughout the product lifecycle, including drug development and review. BIO provides recommendations on how the FDA can better support this trilateral communication, such as the process by which FDA will communicate and provide feedback to stakeholders who submit draft guidance or other data, when dockets are open for stakeholder comments, and how FDA staff will engage with stakeholders around patient experience data.

Human Health, Reimbursement & Access • Letters, Testimony & Comments • March 20, 2019

On January 25, BIO submitted comments in response to the Centers for Medicare and Medicaid Services' (CMS') Modernizing Part D and Medicare Advantage to Lower Drug Prices and Reduce Out-of-Pocket Expenses Proposed Rule. BIO is extremely concerned to see the Agency continue to focus on policy changes that erode the value of the Medicare benefit for seniors, placing patient access at risk. This Proposed Rule is yet another in a series of proposals that prioritize cost containment policies over patient healthcare needs. BIO respectfully requests that the Agency and Administration instead focus on holistic solutions that balance the financial sustainability of the Medicare program, while addressing patient out-of-pocket cost and access to appropriate treatment. Our concerns, detailed further in the balance of this letter, focus on the following: I. CMS should not allow plans to restrict access in the Medicare Part D six protected classes, as plans already have flexibility to manage these drugs in a clinically appropriate manner. II. CMS must go further in protecting beneficiaries enrolled in Medicare Advantage who may be subject to step therapy for their Part B drugs. III. CMS should not move forward with the inclusion of drug pricing information in the explanation of benefits, and instead focus on the updates to e-Prescribing standard to appropriately share cost information. IV. CMS must continue to work to ensure that patients see the benefit of negotiated prices reflected in their out-of-pocket (OOP) costs. V. CMS should move forward with the proposed requirements prohibiting gag clauses.