Developments in 2018

Citizen Petitions and Petitions for Stay of Action Subject to § 505(q)

On December 4, 2018, the Federal Trade Commission (FTC) commented on FDA’s revised draft guidance, Citizen Petitions and Petitions for Stay of Action Subject to Section 505(q) of the Federal Food, Drug, and Cosmetic Act. In addition to reiterating FDA’s authority under § 505(q) of the Federal Food, Drug, and Cosmetic Act (FDCA) to summarily deny certain citizen petitions, the revised draft guidance describes considerations FDA will use to determine whether a petition was submitted primarily to delay approval of a competing drug. These considerations include the following:

• The petition was submitted unreasonably long after the petitioner learned or knew about the relevant information

• The petitioner submitted multiple and/or serial petitions

• The petition was submitted close in time to expiration of a known patent or exclusivity

• The petition’s scientific positions were unsupported by data or information

• The petition was the same or substantially similar to a prior petition to which FDA had already substantively responded

• The petitioner had not commented during other opportunities for input

• The petition requested a standard more onerous or rigorous than the standard applicable to the petitioner’s product

• Other relevant considerations, such as the petitioner’s history with the FDA  

In a statement approved in a 5-0 vote, FTC indicated that it “shares the FDA’s concerns about patient access to lower-cost generic drugs and biosimilars” and “has a longstanding interest in sham petitioning and other abuses of the government processes that may inhibit competition.” FTC noted its investigations regarding complaints of abuses of the citizen petition process as potential violations of federal antitrust law. FTC indicated that it “stand[s] ready to work closely with the FDA on citizen-petition abuse and other issues that may harm competition,” suggesting that citizen petitions submitted by competitors are likely to face a high level of scrutiny from both FDA and FTC.

FTC stated that it "shares the FDA's concerns about patient access to lower-cost generic drugs and biosimilars."

New Drug Development Modernization Plan

In July, FDA announced its new drug development modernization plan to provide the structural framework necessary to advance many goals of the Cures Act. As part of the plan, the Center for Drug Evaluation and Research (CDER) will add new review divisions organized more closely around disease types. A central goal of the plan is the implementation of multidisciplinary teams to enhance internal collaboration and external collaboration with scientists, expert physicians, patients, and other stakeholders.

Patient-Focused Drug Development

Section 3002 of the Cures Act requires FDA to develop one or more guidance documents over a period of five years regarding the collection of patient experience data (i.e., data collected by any person that is intended to provide information about patients’ experiences with a disease or condition, including the impact on patient lives and patient preferences with respect to treatment). FDA launched a Patient Focused Drug Development (PFDD) initiative in response to this and commitments under the Prescription Drug User Fee Act V (PDUFA V), and issued the first draft guidance under the PFDD, Patient-Focused Drug Development: Collecting Comprehensive and Representative Input: Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders, in June.

Framework for Real-World Evidence Program for Drugs and Biologics

In December, FDA issued Framework for FDA’s Real-World Evidence Program under section 3022 of the Cures Act for assessing the potential use of a real world evidence (RWE) in connection with the agency’s drug and biologic review program. Click here for an in-depth discussion of the Framework. Additionally, in furtherance of its efforts to encourage multi-stakeholder collaboration that supports the generation of more and better RWE for medical devices and regulatory decision making, FDA continues to build out the National Evaluation System for health Technology (NEST). NEST will generate evidence across the total product lifecycle of medical devices by leveraging RWE from clinical registries, electronic health records, medical billing claims, and other sources and apply advanced analytics to the data tailored to the data needs and innovation cycles of medical devices.

Biologics INTERACT Program

In June, FDA also announced its INitial Targeted Engagement for Regulatory Advice on CBER products (INTERACT) program to enable sponsors of biologics products to obtain preliminary informal consultation with FDA at an early stage of development, prior to a pre-investigational new drug application (IND) meeting. Through an INTERACT meeting, sponsors can obtain initial, nonbinding advice from FDA regarding chemistry, manufacturing and controls, pharmacology/toxicology and/or clinical aspects of the development program. This may assist sponsors in conducting early product characterization and preclinical proof-of-concept studies, initiating discussion for new delivery devices, staying informed about overall early-phase clinical trial design elements, and identifying critical issues or deficiencies to address in the development of innovative products. Sponsors should submit (1) a summary of the product and disease being treated, (2) information about the product development to date and future development plans, if appropriate and (3) questions the sponsor wishes to have addressed. The Center for Biologics Evaluation and Research (CBER) will attempt to hold INTERACT meetings within 90 calendar days of receiving requests, depending on other resource constraints.

INDICATIONS AND USAGE Section of Labeling

FDA released an Indications and Usage Section of Labeling draft guidance in July, intended to help applicants draft proposed indications for use (including limitations for use, where appropriate) for prescription drug and biological products. The INDICATIONS AND USAGE section is intended to enable health care practitioners (HCPs) to readily identify appropriate therapies for patients by communicating the drug’s approved indication(s). Therefore, the draft guidance is also intended to help ensure that the INDICATIONS AND USAGE section is consistent within and across drug and therapeutic classes, which is particularly relevant to indexing of indications in electronic drug databases and searchability in electronic medical information systems, both of which facilitate clinical decision making and HCP awareness of available therapies.

Looking Ahead to 2019

Significant discussion around drug pricing will likely continue as the new Congress is seated in January 2019. On October 18, 2018, the US Department of Health and Human Services (HHS) published a proposed rule, Medicare and Medicaid Programs; Regulation To Require Drug Pricing Transparency, 83 Fed. Reg. 52,789, to require direct-to-consumer television advertisements of prescription drugs and biologics covered by Medicare or Medicaid to include the Wholesale Acquisition Cost or “list price” for a 30-day supply of any product that costs more than $35 a month. The list price must be written in a type size legible to television viewers. The proposed rule does not require that the list price be read aloud. Proponents argue the proposed rule would prompt consumers to become more price sensitive, in turn slowing the rise of drug costs. Drugmakers that fail to comply would be penalized by being named on a list issued by HHS and through possible enforcement action.  

The same day that HHS announced the proposed rule, the Pharmaceutical Research and Manufacturers of America (PhRMA) announced a new voluntary action that would direct consumers to company websites with pricing information. The announcement appears to be an effort to propose an alternative to more aggressive federal regulation. The initiative will begin by April 15, 2019, and it would provide consumers with pricing information that includes the list price, the expected out-of-pocket costs of the drug and available patient assistance programs.

Read more on FDA 2018 Year in Review.