April 2020 Regulatory Updates

Join us at weekly Journal Club meetings and connect with students, faculty and staff of Regulatory Science Program. Read the latest Regulatory Updates April 2020. For general information on Education Initiatives, click hereHighlight posted on May 8th, 2020


NICE Backs Bayer’s Vitrakvi for NHS Use

The U.K. National Institute for Health and Care Excellence (NICE) reported that the results from trials suggest that neurotrophic tyrosine receptor kinase (NTRK) gene fusions shrink in response to Vitrakvi (larotrectinib) but that it is not clear how well the drug works compared with other treatments. For this reason, the institute recommended against the drug, finding that it was not cost-effective at the price.

Coronavirus Disease 2019 (COVID-19) 
The FDA Seeks to Expand Use of Remote Digital Pathology Devices amid Pandemic

The U.S. Food and Drug Administration (FDA) issued a temporary enforcement policy easing regulatory requirements for some Class II digital pathology devices to expand the availability of remote reviewing and reporting of pathology slides amid the coronavirus disease (COVID-19) pandemic. Under this policy, companies do not need to comply with good manufacturing practice (GMP) or unique device identification (UDI) requirements, or special controls typically applied to such devices, but they need to adhere to labeling and performance requirements in order to modify, or market, devices in line with the policy.

Hydroxychloroquine Falls Flat as COVID-19 Treatment

An expert panel of the U.S. National Institutes of Health (NIH) has recommended against the use of hydroxychloroquine with antibiotic azithromycin outside of clinical trials for COVID-19 treatments because of potential toxicity and increased risk of heart problems. Additionally, the panel did not make any recommendation on the use of hydroxychloroquine or chloroquine alone, citing insufficient evidence.

FDA Reviews Likely to Slow as COVID-19 Workload Increases

The U.S. Food and Drug Administration (FDA) is meeting its user fee-related review goals for now during the COVID-19 outbreak, but the agency may not be able to sustain its current workload. The agency is also working at full capacity to ensure that drug programs continue to see minimal interruptions, but it will need to prioritize if there is an increase in drug shortages or supply disruptions. 

FDA and China NMPA Expedite COVID-19 Emergency Approvals

The U.S. Food and Drug Administration (FDA) and China National Medical Products Administration (NMPA) have moved to fast-track the review of products that might be used to prevent or treat COVID-19. NMPA has issued more than 70 emergency approvals, including several diagnostic tests. The FDA has issued multiple Emergency Use Authorizations (EUAs) for critically important products, including ventilators, respirators and personal protective equipment (PPE) for healthcare providers.


FDA Approves First New Drug under Project Orbis​

Project Orbis is a collaborative review effort for oncology products that includes the U.S. Food and Drug Administration (FDA), Australia Therapeutic Goods Administration (TGA), Health Canada, Singapore Health Sciences Authority (HSA) and Swissmedic.​ Seattle Genetics’ Tukysa (tucatinib) is the first new drug approved by the FDA under Project Orbis. It marks the third action taken by the agency as part of the collaboration and the first time HSA and Swissmedic participated in a review.​ 

Coronavirus Disease 2019 (COVID-19)
COVID-19: FDA, EMA and 16 Drugmakers Take Part in Development Effort

The goal of the Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) partnership is to develop a coordinated research strategy to prioritize drug and vaccine candidates and coordinate clinical trials and regulatory processes.​ Focus areas include standardizing and sharing preclinical evaluation methods; prioritizing and accelerating clinical evaluation; maximizing clinical trial capacity and effectiveness, and advancing vaccine development.​

Reagan-Udall Foundation Launches COVID-19 Treatment Hub

The Reagan-Udall Foundation has expanded its Expanded Access Navigator, which facilitates pre-approval access to drugs to launch a COVID-19 “treatment hub” aimed at helping patients and healthcare providers find treatment resources.​ The hub includes a searchable directory of companies developing treatments and a listing of clinical trials and expanded access programs from ClinicalTrials.gov.​

User Fee-Related Reviews on Schedule amid COVID-19

The U.S. Food and Drug Administration (FDA) user fee funded reviews of drugs, biologics and medical devices are progressing according to schedule despite the disruption caused by the coronavirus disease (COVID-19) pandemic. The agency has extended response due dates by 90 days for 510(k)s, premarket approval applications (PMAs), humanitarian device exemptions (HDEs) and De Novo classification requests, which could impact the agency’s ability to meet its goals for total time to decision under its Medical Device User Fee Amendments commitments.​


Coronavirus Disease 2019 (COVID-19)
Regulators Urge RCTs with Control Arms for COVID-19 Drug Trials

The International Coalition of Medicines Regulatory Authorities (ICMRA) meeting summary report stressed the need for COVID-19 drug developers to include an appropriate control arm (i.e. not including antivirals or immune modulators). Regulators expressed concern due to the multitude of ongoing trials and access programs, which may lead to shortages of investigational products, and recommended this to be carefully monitored.

FDA, EC Offer Guidance on COVID-19 Convalescent Plasma

The U.S. Food and Drug Administration (FDA) and European Commission (EC) recently released guidance on convalescent plasma collected from individuals who have recovered from COVID-19 and which may potentially be used as a treatment for COVID-19. The guidance provides recommendations and information to health care providers and investigators on the administration and study of the plasma.

EMA Q&A Explains Regulatory Expectations for Drugs during the Pandemic

The European Medicines Agency (EMA) and European Commission (EC) released a question and answer (Q&A) document on regulatory expectations for marketing authorization holders (MAHs) and medicines during the COVID-19 pandemic. As far as marketing a medicine in the absence of an authorization, the Q&A indicates that member states can resort to compassionate use, or authorization of the distribution of an unauthorized medicinal product. The Q&A also states that MAHs can request an exemption due to exceptional circumstances and on public health grounds to renew marketing authorizations.

WHO Favors COVID-19 Intellectual Property Pool

The World Health Organization (WHO) aims to enable governments, industry, universities and nonprofits to work together to help develop COVID-19 drugs under the banner of the WHO. The idea was introduced by Costa Rican officials who feared that some COVID-19 products, once developed, would not be made available to poorer nations. 

FDA Grants First EUA for Blood Purification Device for COVID-19 Patients

The U.S. Food and Drug Administration (FDA) granted its first emergency use authorization (EUA) for a blood purification system, the Spectra Optia Apheresis System and Depuro D2000 Adsorption Cartridge devices, to treat patients at risk for respiratory failure due to coronavirus disease (COVID-19). Specifically, the EUA specifies the devices should only be used for patients with early acute lung injury, early acute respiratory distress syndrome or other specific severe or life-threatening symptoms or conditions.


Coronavirus Disease 2019 (COVID-19)
FDA Issues First EUA for Coronavirus Serology Test

The U.S. Food and Drug Administration (FDA) provided the first Emergency Use Authorization (EUA) for a serology test to North Carolina-based Cellex Inc. The test can detect SARS-CoV-2 antibodies, immunoglobulin M (IgM) and immunoglobulin G (IgG) generated as part of the human immune response to the virus. Although the FDA recommends that results from serology testing should not be used as the sole basis to diagnose or exclude a coronavirus infection, the test can help detect how many people have had SARS-CoV-2, even if they did not show symptoms.

FDA Creates Coronavirus Treatment Acceleration Program

The U.S. Food and Drug Administration (FDA) launched the Coronavirus Treatment Acceleration Program (CTAP), an emergency program for potential COVID-19 therapies. Currently, the agency is monitoring 10 therapies in active trials and 15 others in planning stages.

Clinical Trials during COVID-19: Updates from FDA, MHRA and TGA

The U.S. Food and Drug Administration (FDA) updated guidance features for a new appendix offering instructions of clinical trials management, protocol deviations and amendments handling, and submission of formal amendments or supplements for trials conducted under investigational new drug (IND) application or investigational device exemption (IDE) amid the pandemic. The guidance also provides recommendations for handling home delivery of investigational products; considerations for switching from facility to home infusion; delays in on-site monitoring; and obtaining signed informed consent for patients who are in isolation due to COVID-19.

The U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) has written to sponsors conducting Phase I clinical trials to confirm they have conducted a risk assessment for the potential impact of COVID-19 on their studies. MHRA expects that some trials “may be temporarily halted or terminated,” while others may continue if “clearly justified.” The Australia Therapeutic Goods Administration (TGA) posted a clarification about when clinical studies may proceed in relation to its clinical trial notification (CTN) scheme and explained situations where sponsors do not need to notify the agency of variations to their studies due to COVID-19.

FDA Reports Shortage of Sedation Drug Used for Putting COVID-19 Patients on Ventilators

The U.S. Food and Drug Administration (FDA) updated its list of drugs in shortage to include the sedation drug midazolam, which along with other sedatives is being used to treat COVID-19 patients requiring mechanical ventilation. In addition to the sedatives, the unproven but potential COVID-19 treatment hydroxychloroquine sulfate is also listed as currently in shortage.

EMA Advises on Compassionate Use Programs for Remdesivir

The European Medicines Agency (EMA) released a set of recommendations for providing Gilead’s investigational drug remdesivir under compassionate use programs to patients with coronavirus disease (COVID-19) who are ineligible for clinical trials. EMA explained how to use remdesivir and give preliminary information on its safety in addition to describing which patients may benefit from the medicine.

European Commission Proposes to Delay MDR by a Year Due to COVID-19

With an eye toward avoiding the disruption of the device market at such a critical time, the European Commission (EC) year-long delay will not alter the substance of the Medical Device Regulation (MDR). The delay also will “not impose new obligations on the concerned parties. It primarily aims at providing, for exceptional reasons in the context of the current COVID-19 outbreak, a one-year deferral as regards the date of application of certain provisions of that Regulation,” the EC said.

FDA Relaxes Rules for Infusion Pumps, Clinical Thermometers

The U.S. Food and Drug Administration (FDA) released new guidance for infusion pumps and clinical thermometers in an effort to increase availability of the devices amid the coronavirus disease (COVID-19) pandemic. The enforcement policy for infusion pumps applies to devices under four different product codes and more than 20 different types of accessories that are used with infusion pumps. To increase the availability of clinical electronic thermometers, the FDA will not object to the distribution or use of certain non-510(k) cleared devices.

India Lifts Ban of Hydroxychloroquine Exports

The Indian government said that it will lift a newly imposed export restriction on the anti-malaria drug hydroxychloroquine, a potential COVID-19 treatment. Physicians are now allowed to prescribe the drug to COVID-19 patients under an emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA), but at least 20 states have imposed restrictions and there are serious concerns about possible adverse reactions.


Coronavirus Disease 2019 (COVID-19)
New FDA Program to Accelerate Coronavirus Treatments

The U.S. Food and Drug Administration (FDA) detailed a new “special emergency program”, coronavirus treatment acceleration program (CTAP), for accelerating research and development of potential treatments for coronavirus disease (COVID-19). The FDA intends to respond to developers within a day and will try to conduct trial protocol reviews in some cases within 24 hours. The FDA will also work closely with applicants and other regulatory agencies to expedite quality assessments for products to treat COVID-19 and to transfer manufacturing to alternative or new sites to avoid supply disruption.

DOJ Targets Fake COVID-19 Vaccine Kit Website

The Department of Justice (DOJ) has taken its first enforcement action for fraud related to COVID-19 vaccine kits. The DOJ sued NameCheap, the operator  of the website coronavirusmedicalkit.com in a Texas federal court for offering access to “free” World Health Organization vaccine kits in exchange for a shipping charge. U.S. District Judge Robert Pitman issued a restraining order blocking public access to the website.

Notifying FDA of Drug Shortages during the Pandemic: FDA Offers Guidance

The U.S. Food and Drug Administration (FDA) published new guidance on how manufacturers should notify the FDA of permanent halts or interruptions to manufacturing certain products that are likely to lead to a meaningful disruption in supply. Companies should submit notifications no later than five business days after the discontinuance or interruption occurs and update every two weeks, including information on the expected timeline for recovery. FDA staff also have been proactively reaching out to manufacturers to identify potential disruptions or shortages of drugs.

FDA Authorizes Use of New Two-Minute Test Kit for Coronavirus

The U.S. Food and Drug Administration (FDA) has authorized the emergency use of Bodysphere Inc.’s test that can detect the coronavirus in nearly two minutes. The test is administered like a glucose test but is designed for use strictly by medical professionals.

FDA Warns Irish Company for Marketing Unapproved COVID-19 Treatments

As there are still no approved treatments for COVID-19, the U.S. Food and Drug Administration (FDA) warned Ireland-based Carahealth for offering unapproved herbal products and other products referred to as “Immune Tonic” on its website to mitigate, prevent, treat, diagnose or cure COVID-19. The FDA points to dubious claims on the company’s website, such as, “Constituents in herbs exhibit a diverse array of anti-viral, virostatic, immune enhancing and anti-influenza activities that may prevent and treat pandemic influenza.”

Ford, GM, GE to produce 50,000 ventilators in 100 days

Ford said the simplified ventilator design, which is licensed by GE Healthcare from Florida-based Airon Corp and has been cleared by the U.S. Food and Drug Administration (FDA), can meet the needs of most COVID-19 patients and relies on air pressure without the need for electricity. GM said it plans to produce up to 10,000 ventilators a month by this summer at a plant in Kokomo, Indiana.

FDA Warns Pfizer Manufacturing Site in India

The U.S. Food and Drug Administration (FDA) sent a warning letter to Pfizer’s injectable product manufacturing site in Visakhapatnam, India after an inspection. The letter explains how the site did not adequately investigate root causes and implement corrective and preventive action (CAPA) to address deficiencies with its sterility testing. In a follow-up to the warning, the FDA asked Pfizer to provide an assessment and remediation plan for its CAPA program, a complete assessment of documentation systems used throughout the site’s manufacturing plant and laboratory.

FDA Issues and Expands EUA for Respirator Decontamination System

The U.S. Food and Drug Administration (FDA) gave emergency approval to the first system for decontaminating respirators to extend their use amid the coronavirus disease (COVID-19) pandemic, before expanding the terms of the authorization. The emergency use authorization (EUA) initially limited the number of respirators that could be decontaminated using the system to 10,000 per day. After receiving requests from industry, FDA reissued the EUA without a cap on how many respirators the system can process per day. The EUA retains a limit of 20 decontamination cycles per respirator.