January 2018

· 4 min read

Regulatory Radar: FDA What’s Ahead for 2018 – Proposed Rulemaking

A review of the semiannual Unified Agenda of Federal Regulatory and Deregulatory Actions, published in the fall of 2017, gives insight into regulations under development by the U.S. government including those affecting the Food and Drug Administration (FDA) in the upcoming year.

The following is a selected listing of proposed new rulemaking in the current Unified Agenda for 2018 related to the development and marketing of biologics and drug products.

Pediatric Study Plan Requirements for New Drug and Biologics License Applications

FDA is proposing requirements for the content of initial pediatric study plans required to be submitted under the Pediatric Research Equity Act (PREA) for new drug applications (NDAs) / biologics license application (BLAs). Sponsors of certain applications will be required to submit a plan for studying their drug/biologic in pediatric patients, a primary driver for the increased availability of labeling and other drug/biologics information for pediatric patients. Although the plans are required to be submitted based on the statutory provision, FDA indicated that a new regulation is required to provide clarity, specificity and direction to sponsors. It is also intended to assist FDA in ensuring compliance with PREA, and ultimately to help better advance the study of drugs in pediatric populations in support of advancing pediatric indications.

Medication Guides; Patient Medication Information

Patients may currently receive one or more types of written patient information regarding prescription drug products. Research has shown that frequently the information received is duplicative, incomplete, conflicting, or difficult to read and understand and, as such, this information is not sufficient to meet the needs of patients. Patient Medication Information is a new type of one-page Medication Guide that FDA is proposing to require for certain prescription drug products. To accomplish this, FDA is proposing revisions to the regulations regarding format and content requirements for certain prescription drug labeling that are authorized by the Federal Food, Drug, and Cosmetic Act and by the Public Health Service Act.

The new Patient Medication Information would be required for human prescription drug products used, dispensed or administered on an outpatient basis. The proposed rule would require clear and concise written prescription drug product information presented in a consistent and easily understood format to help patients use their prescription drug products safely and effectively. It would include requirements for Patient Medication Information development, consumer testing and distribution.

Definition of the Term "Biological Product"

FDA proposes to amend its regulation that defines “biological product” to conform to the statutory definition (42 U.S.C. 262) adopted in the Biologics Price Competition and Innovation (BPCI) Act of 2009, as the current definition includes a ‘‘protein (except any chemically synthesized polypeptide)”.

This will allow FDA to prepare for March 23, 2020 when several biologic products, previously approved under an NDA, will be deemed to be approved under a BLA. These are typically legacy protein products which, due to historical precedents, were approved under an NDA. Expect FDA to come out later with either a “Question-and-Answer” document or a draft guidance to explain the impact of this switch-over to both the (future) BLA holder and to the agency.

For example, under an NDA, a company would file a Field Alert Report (FAR) for a possible product safety quality issue whereas under a BLA, a company would file a Biological Product Deviation Report (BPDR). The expectations and timelines are different for the two reports, so companies would need to adjust their internal policies and procedures to accommodate the change.

Biologics License Applications and Master Files

Similar to the switch from an NDA to a BLA for some approved proteins on March 23, 2020, some of the affected NDAs were approved using drug master files (DMFs). A DMF, or other master file, allows an applicant to incorporate proprietary information from another manufacturer into its application by reference to the master file. Where BLAs generally may not rely on drug substance, drug substance intermediate or drug product information contained in master files, companies would be forced to update their BLAs with the drug substance manufacturing process. To avoid this, FDA is proposing a rule that would now permit the use of DMFs for BLAs subject to the BPCI Act transition.

Post-Approval Changes to Approved Applications

This proposed rule would update the existing regulations governing supplements and other changes to approved NDAs, abbreviated new drug applications and (ANDAs) and BLAs, as well as regulations regarding certain post-approval reports. No other details were provided.

National Standards for the Licensure of Wholesale Drug Distributors and Third-Party Logistics Providers

This rulemaking will establish standards for state licensing of prescription drug wholesale distributors and third-party logistics providers. The rulemaking will also establish a federal system for wholesale drug distributor and third-party logistics provider licensing for use in the absence of a state licensure program.

Current Good Manufacturing Practice for Outsourcing Facilities

This rule would set forth the minimum current Good Manufacturing Practice (cGMP) requirements for human drug products compounded by an outsourcing facility.

For details on all proposed rule-making, see the full listing of the Unified Agenda.

In addition, as noted in the December 2017 FDA Voice blog, the Agency seeks to continue its efforts in updating and modernizing its regulations in 2018 by:

  • Updating its requirements for accepting foreign clinical data used to bring new medical devices to market
  • Proposing a new framework that will allow FDA and product developers to take greater advantage of the efficiency of electronic, rather than paper, submissions for devices and veterinary drugs
  • Proposing to remove an outdated inspection provision for biologics and outdated drug sterilization requirements to remove barriers (new drug status) with the use of certain sterilization techniques (irradiation)

To keep up to date on FDA rulemaking, refer to the Unified Agenda-TRACK FDA website which provides periodic updates.

Do you have any questions? Please reach out to NSF’s experts Marinka Tellier or Andy Papas. Visit our website to learn more about NSF's pharma biotech services.

If you are interested in past issues of Regulatory Radar, visit our library.