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Recent Amendments to the FDA Laws Attempt to Clarify and Improve Existing Systems

Although the Biden-Harris Administration that assumed control of the Executive Branch on January 20, 2021 immediately ordered a regulatory freeze of new or pending rules while the new administration gets its bearings (as reported by our colleagues in this post), several important changes to the laws enforced by the Food and Drug Administration (FDA) were recently enacted by Congress. As legislative actions, those changes are of course unaffected by President Biden’s regulatory freeze and so we thought worth a summary to ensure our readers are up to speed on the large amount of activity that occurred in the final weeks of the 116th Congress and the Trump Administration. 

First, as most already know, after months of partisan haggling, Congress passed a COVID-19 relief package on December 21, 2020, in conjunction with $1.4 trillion in appropriations legislation to fund the government (it was signed into law by the President on December 27). Officially referred to as the Coronavirus Response and Relief Supplemental Consolidated Appropriations Act of 2021, it represents the long-awaited sequel to the Coronavirus Aid, Relief, and Economic Security Act, or CARES Act, that was enacted last March in the early days of the pandemic. The consolidated appropriations package is one of the longest bills in recent memory and is chock full of provisions addressing a myriad of topics pertaining to pandemic relief and the governmental response. It included $55 million for FDA’s COVID-19 response work as well as a sizable increase in discretionary spending for the agency in FY 2021 ($43 million) compared to last year’s appropriation.

The bill also incorporated a litany of health care provisions including several amendments to FDA’s primary authorizing statute, the Federal Food, Drug, and Cosmetic Act (FD&C Act), as well as the Public Health Service Act that applies to biological products and vaccines. These amendments affect diverse areas of the agency’s mandate such as the Rare Pediatric Disease Priority Review Voucher Program, generic drug labels, clinical superiority of orphan drugs, and biosimilars and biological product patent transparency, as outlined further below. Several of these individual pieces of legislation were previously discussed by our colleagues at ML Strategies when they moved out of committee mark-up and to the full House for consideration (see prior post here).

  • Extension of FDA’s Rare Disease Priority Review Voucher Program
    The Rare Pediatric Disease Priority Review Voucher Program was originally created by Congress as part of FDA’s 2012 user fee reauthorization legislation but was set to expire after September 30, 2020 unless further extended by Congress. Companies that produce a drug or biologic that is approved to treat a previously-designated rare pediatric disease may receive a voucher for priority review of a subsequent drug product application. The COVID-19 relief bill officially extended the authorization for FDA to continue operating this Voucher Program through FY 2024.  


  • New FDA Authority to Require Updating Generic Drug Labels
    Under existing law, a generic drug is required to have the same label as its so-called reference listed drug, or RLD, which is the brand or innovator product that the generic has relied upon to get to market. Typically, RLD product labels undergo several changes over the course of the brand drug’s lifecycle, changes that are not always reflected in corresponding generic drug labels. For instance, a generic drug label may not reflect new indications or new safety-related information discovered after significant numbers of people or certain populations use the branded drug. In addition, over time, the branded product may no longer be on the market for business or various other reasons, even though the generic products remain FDA-approved and marketed. Without a currently marketed RLD, those generics must rely on a label that cannot readily be updated by the RLD’s owner. In practical effect, this means that many generic drug labels are considerably out of date.
    The 2021 appropriations package included the Making Objective Drug Evidence Revisions for New (MODERN) Labeling Act, which provides FDA with express authority to require that sponsors of certain generic drugs modernize outdated labeling to ensure accurate information is relayed to health care providers, pharmacists, and consumers. Such mandatory labeling updates could be triggered by one or all of following agency findings: 
  1. There is new scientific evidence available pertaining to new or existing conditions of use for the generic drug.
  2. The approved labeling does not reflect current legal and regulatory requirements.
  3. There is relevant accepted use(s) in clinical practice that is not reflected in the drug’s approved labeling.
    FDA must report any such mandatory generic drug labeling updates to Congress on an annual basis, and the newly amended FD&C Act also establishes specific procedural steps for both the agency and the generic drug sponsor to follow when this process is initiated. A generic drug that is the subject of a mandatory labeling update but fails to have its labeling revised in a timely manner may be deemed misbranded under the Act.
  • Clarification of Orphan Drug/Biologic Marketing Exclusivity Standards
    An orphan drug designation is a special status granted by FDA (upon request of a sponsor) to a drug or biologic product intended to treat a rare disease or condition. For a drug to qualify for orphan designation both the drug and disease or condition must meet certain criteria specified in the Orphan Drug Act and in FDA’s implementing regulations; a designated orphan product that is subsequently approved for marketing is awarded seven years of marketing exclusivity during which the “same drug” cannot be approved by FDA for the same rare disease.
    As part of the last Prescription Drug User Fee Act (PDUFA) reauthorization package in 2017, Congress made an amendment that created some confusion with respect to FDA’s standards for granting orphan drug exclusivity to a drug that is the same as a drug already approved for the same rare disease or condition, which requires a showing that the second product is “clinically superior” to the first product. In particular, it was unclear whether an orphan drug designated prior to the enactment of the 2017 amendment would be held to that new language in the FD&C Act or otherwise treated as “grandfathered” (an issue that a federal district court wrestled with in 2019 as well). The COVID-19 relief bill further amends the Act to remove any uncertainty about this question – in other words, the clinical superiority requirement applies to all orphan drug marketing applications approved after the enactment of the 2017 PDUFA legislation even if the drug was designated as orphan before that date.
  • Biosimilar Pathway Amendment & Biological Product Patent Transparency
    The COVID-19 relief bill also amended the Public Health Service Act, which contains the statutory framework for an abbreviated marketing pathway for biosimilar biological products. Applications for licensure of a new biosimilar product now “may include information to show that the conditions of use prescribed, recommended, or suggested in the labeling proposed for the biological product have been previously approved for the reference product,” when previously the language made this information mandatory. This change is expected to improve the efficiency of the biosimilar application submission process and to help bring more competitive biosimilar products to market.
    Also of importance to innovator biological product developers and biosimilar companies alike are Congressional directions to improve the transparency around patents protecting potential reference products. In a prior post, we noted the updates to the Purple Book Database FDA greenlit, providing transparency and more user-friendly search functionality for the entire industry. Despite the recent changes implemented by the agency to improve this resource, however, biologics manufacturers have not been required to list any patent information in the Purple Book, which made it less useful to this highly-competitive industry. Newly enacted language requires biological product sponsors to submit patent information for their products to FDA so the data can be published in the Purple Book (when patents have been disclosed by the biological product sponsor as part of a “patent dance” with a biosimilar developer). In addition, within 180 days of the law’s enactment, FDA must publish in a searchable, electronic format Purple Book:
  1. a list of each biological product with a patent;
  2. the date of licensure of the product and application number; and
  3. the licensure status and marketing status of each product.

FDA must also ensure the Purple Book includes the products that were “transitioned” from drug-to-biological product by operation of law in March 2020 and publish updates to the electronic resource at least every 30 days.

(And, although not an amendment to the FD&C Act, it is worth adding that a bill called The Preventing Online Sales of E-Cigarettes to Children Act also caught a ride on the 2021 appropriations package and FDA’s Center for Tobacco Products could play a role in the enforcement of those new requirements against retailers.)

Second, on January 5, 2021, former President Trump also signed into law a bill called the Orange Book Transparency Act, which had been pending in Congress for some time. In particular, the amendments to the FD&C Act implemented via this legislation specify the patent information that must be submitted and listed in the Orange Book; clarify that canceled or invalid patents must be timely removed; add authority to the statute for FDA to list marketing exclusivities in the Orange Book (something FDA already does); direct FDA to solicit public comments on information listed in the Orange Book (which FDA has already done) and issue a report on the subject to Congress; and instruct the Government Accountability Office to study whether certain patents should be listed in the Orange Book at all. In addition, companies will now have to notify FDA of a listed patent’s invalidation within 14 days, so that the Orange Book listing can be updated accordingly. Although this bill formally codifies FDA’s existing patent listing policies and procedures and does not create major new requirements for either industry or the agency, its enactment is viewed as a further signal that Congress continues to be interested in re-examining the “careful balance” it created in 1984 with the Hatch-Waxman Amendments that ushered in the now-well-known generic drug approval pathway. And the two reports mandated to be prepared and submitted to Congress are likely to fuel additional reform efforts that could end up as part of the upcoming 2022 PDUFA reauthorization cycle.

Last but certainly not least, also on January 5, a bipartisan bill called the Safeguarding Therapeutics Act became law. It amends the FD&C Act to give the executive branch specific authority to seize counterfeit medical devices, a product category that has been subject to increased interest from fraudsters since the COVID-19 pandemic began. It also adds a new definition of “counterfeit device” to the FD&C Act, paralleling the existing definition of “counterfeit drug” in Section 201.

As a new and more progressive 117th Congress begins the people’s work of legislating and investigating, we will continue to keep you up-to-date on any important developments that may impact FDA-regulated industries and products.

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Joanne counsels global clients on the regulatory and distribution-related implications when bringing a new FDA-regulated product to market and how to ensure continued compliance after a product is commercialized.