FDA Regulatory News and Trends

Welcome to the latest issue of FDA Regulatory News and Trends, designed to help you identify significant legal developments and navigate the evolving business, legal and regulatory world.

FDA announces new action plan for rare neurodegenerative diseases. On June 23, the FDA announced its new Action Plan for Rare Neurodegenerative Diseases Including Amyotrophic Lateral Sclerosis (ALS). This, the agency said, is a five-year strategy for improving and extending the lives of people living with rare neurodegenerative diseases by advancing the development of safe and effective medical products and facilitating patients’ access to novel treatments. FDA Commissioner Robert Califf said, “The effects of rare neurodegenerative diseases are devastating, with very few effective therapeutic options available to patients. We recognize the urgent need for new treatments that can both improve and extend the lives of people diagnosed with these diseases.” The action plan is a blueprint for how the agency will move forward in tackling challenges in drug development for these diseases. Among other things, the agency will establish a task force for these diseases, set up a public-private partnership, develop disease-specific science strategies and leverage its ongoing efforts.

FDA issues warning letter to Mexican company concerning its manufacturing practices. On June 13, the FDA issued a warning letter to a Mexican company, Glicerinas Industriales, S.A. de C.V., noting that the company recently refused to submit to an FDA inspection of its plant. The FDA said that the company is a supplier of drug components such as glycerin to manufacturers of finished drug products that sell into the US market and that the use of glycerin contaminated with diethylene glycol has resulted in lethal poisoning incidents in human beings worldwide. The FDA also told the company that, until the FDA is permitted to inspect its facility and to confirm that the company is complying with current good manufacturing practices, it may withhold approval of any new applications or listing the company as a drug manufacturer.

FDA issues guidance on its cooperation with similar food-safety agencies of other countries. On June 21, the FDA issued a final guidance document concerning its regulatory oversight activities for food products covered by a Systems Recognition Arrangement (SRA) between the FDA and another country’s food safety authority. The agency noted that, from time to time, it may enter into an SRA with the agency of another country. Its decision to do so is based on the conclusion that food safety systems with similar elements and similar levels of oversight result in similar food safety outcomes. The FDA’s decision to enter into an SRA, or not to do so, depends on the nature of the other country’s food safety system and what safeguards it provides to ensure food safety. The agency noted that, in making these decisions, it focuses not only on the ability of food safety systems to help prevent food safety problems, but also on the ability of food safety authorities to identify, address and contain food safety issues and outbreaks when they do occur, as well as to learn from past events and strengthen the system over time.

FDA issues final guidance for drug industry on conducting food-effect studies. On June 24, the FDA announced the availability of a final guidance for industry titled, “Assessing the Effects of Food on Drugs in INDs and NDAs—Clinical Pharmacology Considerations.” This guidance provides recommendations to sponsors planning to conduct food-effect studies for orally administered drug products as part of investigational new drug applications, new drug applications and supplements to these applications. This guidance finalizes a draft guidance issued on February 26, 2019. It discusses the considerations that should be used in planning food-effect studies, which are formal studies that explore the effect of food consumption on the effectiveness of a particular drug. It points out, for example, that assessing the effect of food on the absorption of an orally administered drug contributes to the optimization of the safety and efficacy of the product and helps provide adequate instructions for drug administration in relation to food.

FDA awards priority review voucher for Rinvoq. On June 24, the FDA announced its approval of a new drug product that qualifies for a priority review voucher. This product is Rinvoq (upadacitinib), produced by AbbVie. Under the Federal Food, Drug, and Cosmetic Act, as amended by the Food and Drug Administration Safety and Innovation Act (FDASIA), the FDA can award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. The agency announced that AbbVie’s FDA supplemental application for Rinvoq meets these criteria. This means, under the FDA’s voucher program, that the company qualifies for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

Two upcoming FDA public meetings will discuss digital aspects of medical devices. The FDA has announced that on July 12 and 13, 2022, its Patient Engagement Advisory Committee will meet to discuss and make recommendations on the topic of Augmented Reality (AR) and Virtual Reality (VR) Medical Devices. AR/VR devices are increasingly being applied to healthcare settings, the FDA said. From diagnostics to clinical decision making, to surgical support, and to directly treating patients, AR/VR devices are being used in many medical specialties. These devices have novel attributes for end users that will have an impact on the FDA’s evaluation of their safety and effectiveness. The recommendations provided by the committee will address the factors that the FDA and industry should consider when evaluating the benefits, risks, and the extent of uncertainty in the benefit-risk assessment for AR/VR medical devices. The meeting will be open to the public. In addition, the FDA announced that on July 28 and 29, 2022, the General and Plastic Surgery Devices Panel of its Medical Advisory Committee will meet to discuss whether there is sufficient information to reclassify computer-aided devices for adjunctive diagnostic information of lesions suspicious for melanoma from class III to class II, and what special controls may be appropriate to provide reasonable assurance of safety and effectiveness for these devices if they are reclassified as class II devices. This meeting will also be open to the public. Class III devices are those that are considered to pose the highest risk to patients, while class II devices are deemed to have lower risk.

FDA issues warning letters to companies selling kratom or essential oils to treat opioid use. On June 30, the FDA issued warning letters, jointly with the Federal Trade Commission, to four companies selling unapproved kratom products and one company selling essential oils for the treatment or cure of opioid use disorder and withdrawal symptoms. Kratom is an herbal extract that is derived from the leaves of an evergreen tree grown in Southeast Asia. The FDA has not approved kratom or essential oils for the treatment or cure of opioid use disorder and withdrawal symptoms. In addition, the agency has received concerning reports about the safety of kratom. The FDA is advising consumers not to use kratom or essential oils for the treatment or cure of opioid use disorder and withdrawal symptoms. The companies that received warning letters are Herbsens Botanicals, Klarity Kratom, Kratom Exchange, Omni Consumer Products LLC d/b/a YoKratom, and MONQ, LLC.

FDA announces pilot program for electronic premarket notification submissions. On June 21, the FDA announced that its Center for Biologics Evaluation and Research (CBER) is announcing a pilot program for sponsors of CBER premarket notification submissions that wish to use the center’s voluntary Electronic Submission Template and Resource (eSTAR) Pilot Program. The center’s voluntary eSTAR Pilot Program is intended to improve consistency and efficiency in both the industry’s preparation and the FDA’s review of 510(k) submissions. During the voluntary eSTAR Pilot Program, participants will have the opportunity to provide input to the FDA on the program.

FDA issues guidance on patient-focused drug development. On June 30, the FDA issued a draft guidance titled “Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments.” This guidance is the third in a series of guidance documents that are intended to facilitate the advancement and use of systematic approaches to collect and use robust and meaningful patient and caregiver input that can more consistently inform medical product development and regulatory decision-making. When final, this guidance will represent the current thinking of the FDA’s Center for Drug Evaluation and Research, its Center for Biologics Evaluation and Research, and its Center for Devices and Radiological Health on this topic. The purpose of the guidance is to help sponsors identify or develop fit-for-purpose clinical outcome assessments that accurately measure patients’ health and are appropriate for use in a medical product development program. The guidance is intended, the FDA said, to help sponsors use high-quality measures of patients’ health in medical product development programs. Ensuring high-quality measurement is important for several reasons, the FDA said. These include measuring what matters to patients; being clear about what was measured; appropriately evaluating the effectiveness, tolerability, and safety of treatments; and avoiding misleading claims.

COVID-19 patients can now get Paxlovid from pharmacists. On July 6, the FDA announced that patients who test positive for COVID-19 can now get the antiviral pill Paxlovid directly from their pharmacists. This action by the FDA removes limits that had restricted prescribing authority to healthcare providers and Test-to-Treat sites. Because Paxlovid must be taken within five days of the onset of COVID-19 symptoms, the change could spur expanded access to it and more-timely treatment of eligible patients. The change was made by the FDA through an amended emergency use authorization. “The FDA recognizes the important role pharmacists have played and continue to play in combatting this pandemic,” Dr. Patrizia Cavazzoni, director for the FDA’s Center for Drug Evaluation and Research, said in a news release. Paxlovid, which is manufactured by Pfizer Inc., is meant to treat mild-to-moderate cases of COVID-19 in people who are at high risk for severe COVID-19. It is approved for adults and for children ages 12 and up who weigh at least 88 pounds.

FDA and PTO team up to try to reduce drug prices. The FDA and the US Patent and Trademark Office (PTO) have announced that they are teaming up to take a close look at pharmaceutical patenting procedures in order to possibly reduce drug prices. On July 6, the PTO said that it plans to crack down on the patenting of “incremental, obvious changes to existing drugs that do not qualify” for new protections. The initiative follows President Joe Biden’s 2021 executive order that asked the agencies to leverage their collective experience in promoting “innovation, competition, and the approval and regulation of safe and effective drugs. This effort is the result of officials at the PTO and the FDA exchanging letters outlining their concerns about pharmaceutical patents. Last September, the then acting head of the FDA wrote a letter to the PTO citing a study that found that 78 of new drug patents awarded between 2005 and 2015
were not for new drugs, but existing ones.

FDA guidance explains agency’s policy in rescinding the “breakthrough therapy” designation. On June 24, the FDA released a draft guidance document setting forth the considerations that the agency uses in withdrawing the designation of a “breakthrough therapy.” Under federal law, the FDA’s breakthrough therapy program is intended to facilitate and expedite the development of those drugs that receive the designation and involves a resource commitment from the FDA to provide early and frequent advice, conduct multidisciplinary meetings involving senior managers, and when appropriate, expedite the review of the resulting marketing applications. In the guidance document, the agency explained the considerations that it may use in withdrawing the “breakthrough” designation, such as the emergence of another therapy for the same condition or new evidence that the breakthrough therapy may not be a substantial improvement over other therapy.

FDA guidance establishes CGMP for non-penicillin antibacterials. On June 24, the FDA released a draft guidance document intended to establish a current good manufacturing practice (CGMP) to prevent, in the drug manufacturing process, any cross-contamination of non-penicillin beta-lactam antibacterial drugs with other drugs such as penicillin. The guidance recommends complete and comprehensive separation of the manufacturing operations of non-penicillin beta-lactam antibacterial drugs from the manufacturing operations of other drugs. The agency explains that this CGMP is of considerable importance because many patients are allergic or hypersensitive to penicillin in ways that can be life-threatening, and thus it is necessary that no cross-contamination of other drugs with penicillin can occur.

FDA proposes new way to approach Rx-to-OTC switch. On June 27, the FDA issued a proposed rule titled “Nonprescription Drug Product with an Additional Condition for Nonprescription Use.” The rule, according to the FDA, is intended to broaden the range of marketed nonprescription drugs available to consumers, thus empowering them to self-treat certain common conditions and improving public health. The agency said that, if finalized, the proposed rule would establish a new option for a drug company that wants to market a nonprescription drug. Under the proposed rule, when the FDA finds that labeling alone is not sufficient to ensure that the consumer can appropriately self-select and use a drug product in a nonprescription setting, an applicant may submit an application proposing an additional condition for nonprescription use that a consumer must successfully fulfill to obtain the nonprescription drug product. This could be, for example, in the form of a phone app in which a consumer would have to answer a set of questions before getting access to the drug.

FDA will convene advisory panel meeting this year to discuss pulse oximeters. On June 21, the FDA issued an update to its current safety advice about pulse oximeters. Pulse oximeters are familiar small medical devices that quickly measure the oxygen content of blood by hooking onto a finger or an ear. The agency announced that, because of ongoing concern that pulse oximeters may be less accurate in people with darker skin pigmentations, it is planning to convene a public meeting later this year of its Medical Devices Advisory Committee to discuss the available evidence about the devices’ accuracy, recommendations for patients and healthcare providers, and the amount and type of data that manufacturers should provide to assess the devices’ accuracy.