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FDA and EMA Partner for Metastatic Breast Cancer Discussion Panel
The FDA’s Oncology Center of Excellence (OCE) recently collaborated with the European Medicines Agency (EMA) in hosting a historic panel discussion, “Conversations on Cancer: Living with Metastatic Breast Cancer. “
“We are very interested in exploring similarities and differences that may exist both in drug regulation, treatments, and perceptions of diseases. We also hope today’s conversation will be the first of many joint conversations on cancer by the FDA and EMA,” said Dr. Richard Pazdur, the FDA’s OCE director and medical oncologist.
The event marked the first international collaboration with the FDA and EMA highlighting the experiences of U.S. and European metastatic breast cancer patients, including four panelists currently living with the disease.
“EMA is dedicated to incorporating patient perspective. Today’s discussion represents a significant step in this direction, a prime example of our commitment to engaging with patients and the broader community,” said Caroline Voltz, Team Leader for EMA’s Oncology and Drug Therapies Office.
OCE’s Conversations on Cancer series was initiated to serve as an interface between societal issues and the science of oncology, as well as drug regulation.
Previous Conversations he included discussions of cancer clinical trial and health care access in Native American, LGBTQ+, Latinx, Asian American, Pacific Islander, and African American communities; the important role of oncology nursing; lung cancer in nonsmokers; and dialogue between the HIV and oncology communities. OCE believes that patient perspectives are crucial in determining what is working, what could be better, and how to help others survive this deadly disease.
The joint FDA-EMA event included a discussion on the differences and challenges that cancer experts face in making treatments ailable to patients. The four participating patients focused on current barriers to receiving high-quality, equitable, accessible, and affordable medical care in both the United States and Europe.
“We hen’t got a [global] system that is collecting data nor the real-world evidence of even older drugs and how they are working. It’s hugely upsetting because patients are dying, unable to access necessary drugs – what is the point of creating a drug if patients cannot access them? There are also different regulatory approval layers in [the] U.S. and Europe as barriers,” said Jo Taylor, founder of MET UP UK, the only metastatic breast cancer patient advocacy group in the U.K.
Globally, almost 30% of breast cancer patients are diagnosed with advanced-stage or metastatic disease.
The October 19 panel was hosted on YouTube and recorded with subtitles. Attendees were able to post comments and questions to OCE’s X account, formerly known as Twitter.
FDA Approves First Vaccine to Prevent Disease Caused by Chikungunya Virus
The world’s first vaccine to prevent disease caused by the potentially debilitating chikungunya virus has been approved by the FDA.
Ixchiq, manufactured by the French company Valneva SE, is approved for individuals 18 years of age and older who are at increased risk of exposure to the virus, which is primarily transmitted through the bite of an infected mosquito.
“Infection with chikungunya virus can lead to severe disease and prolonged health problems, particularly for older adults and individuals with underlying medical conditions,” said Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research “[The] approval addresses an unmet medical need and is an important advancement in the prevention of a potentially debilitating disease with limited treatment options.”
Chikungunya – which comes from a word meaning “to become contorted” in the Kimakonde language spoken in parts of east Africa – was first detected in Tanzania in 1952, triggering the first documented outbreaks in cities in Thailand and India some 15 years later.
According to the World Health Organization, chikungunya is an emerging global health threat with at least 5 million cases of chikungunya virus infection reported during the past 15 years. The highest risk of infection is in tropical and subtropical regions of Africa, Southeast Asia, and parts of the Americas where chikungunya virus-carrying mosquitos are endemic. However, the virus has spread to new geographical areas, causing a rise in global prevalence of the disease.
Image of an adult Aedes aegypti mosquito, feeding. Courtesy the Centers for Disease Control and Prevention.
In February, the Pan American Health Organization (PAHO) issued an epidemiologic alert about elevated chikungunya activity in the Americas, urging countries to prepare their health care systems for the medical management of it and other mosquito-borne diseases. PAHO reported that cases were up sharply in 2022 compared to the previous year, with 13 countries reporting chikungunya infections.
Ixchiq is a live, weakened version of the virus, administered as a single dose by injection into the muscle. The safety of the vaccine was determined through two clinical studies involving about 3,500 adults in North America. Common side effects included headache, fatigue, muscle pain, joint pain, fever, nausea, and tenderness where the needle went in the skin. Severe reactions that prevented daily activity or required medical intervention occurred in 1.6% of vaccine recipients. Two people who received the vaccine had to be hospitalized.
The vaccine was approved using the Accelerated Approval pathway. Accelerated approval allows the FDA to approve certain products for serious or life-threatening conditions based on evidence of a product’s effectiveness that is reasonably likely to predict clinical benefit. In the FDA evaluation of Ixchiq for accelerated approval, evidence of effectiveness is based on immune response data in clinical trial participants. As a condition for approval for Ixchiq, the agency is requiring that confirmatory clinical studies be conducted to verify clinical benefit.
Ixchiq was granted Fast Track and Breakthrough Therapy designations, and the application was granted Priority Review. The FDA also awarded the manufacturer of Ixchiq a tropical disease priority review voucher, under a provision included in the Food and Drug Administration Amendments Act of 2007. This provision aims to encourage the development of new drugs and biological products for the prevention and treatment of certain tropical diseases.
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Ixchiq (Chikungunya Vaccine, Live)CDRH and LAO Experts Share Insight on Medical Device Regulation at Latin America Regional Meeting
For more than a decade, regulators and industry across the Americas he been committed to strengthening the regulatory capacity of the region’s medical device national regulatory authorities, with the support of the Pan American Health Organization (PAHO).
One enue for strengthening capacity has been the region’s annual meeting on device regulation, held this year in El Salvador on October 11-13, which was co-hosted by PAHO and El Salvador’s National Directorate of Medicines and the Salvadoran Social Security Institute.
The hybrid XI Regional Meeting on Medical Devices Regulation drew participants from 22 countries across the region. FDA subject matter experts from the Center for Devices and Radiological Health (CDRH) and the agency’s Latin America Office (LAO) presented remotely. The meeting offered open sessions for promoting dialogue between regulators and stakeholders, and closed sessions for regulators only. Attendees included regulators, industry, academia, and members of the Inter-American Coalition for Regulatory Convergence for the Medical Technology Sector (a private sector group that has unified the 17 largest industry bodies for the medical technology sector in the region to provide hemispheric industry consensus recommendations for regulatory convergence priorities).
Image courtesy of PAHO.
At the open session, Ethny Obas, a lead consumer safety officer with CDRH, presented on the electronic issuance of export certificates, or e-certs, and provided clarification on the new process that will be implemented next year. Such certificates, issued by the FDA, attest to the marketing status of a medical device in the United States, as well as the compliance status of the device manufacturer with current good manufacturing practice regulations. Devices that can be legally marketed in the United States may be exported anywhere in the world without prior FDA notification or approval of exportation. However, hing an export certificate is required by many device regulatory authorities in Latin America (and elsewhere) for companies to legally sell their product in their territories. Those export certificates will be going electronic, meaning no more paper as of January 2, 2024.
Focusing on regulatory issues, Erin Cutts, a regulatory policy analyst with CDRH, presented on the use of Real-World Evidence (RWE), which provides an understanding of device performance in actual clinical settings to inform regulatory benefit/risk decision-making. The evidence derived from the analysis of data related to patient health status and/or delivery of health care may be collected from a variety of sources. Use of RWE includes the ability to collect outcomes not always feasible in traditional trials, as well as the reduction of the time and cost to answer important questions.
CDRH has taken several concrete steps to promote RWE adoption and use for regulatory purposes and to optimize the infrastructure needed to develop RWE. Cutts described how CDRH has published guidance and examples of RWE being used in regulatory decision making and underscored its international value. Real-world data from any country can be collected to answer questions about clinical practice and outcomes applicable to all countries. Regulatory convergence on how to approach real-world data collection and the uses of RWE help patients across the globe he access to safe, effective, high-quality, and innovative medical devices.
Regulatory reliance was also a hot topic. The World Health Organization defines the concept in its Annex 10 Good Reliance Practices guideline document as:
"The act whereby the national regulatory authority (NRA) in one jurisdiction may take into account and give significant weight to assessments performed by another national regulatory authority or trusted institution, or to any other authoritative information in reaching its own decision. The relying authority remains independent, responsible, and accountable regarding the decisions taken, even when it relies on the decisions and information of others."
Vesa Vuniqi, an international relations specialist with the LAO, talked about the FDA’s public databases and the marketing, compliance, and safety information that is ailable to support the implementation of reliance mechanisms in Latin America. These include CDRH’s numerous public medical device databases — on 510(k) premarket notifications, premarket approvals, and many other product-oriented data collections — as well as the public FDA Data Dashboard highlighting information on manufacturers and inspections, compliance actions, and recalls. At the regulators-only session, Vuniqi addressed the FDA’s own activities related to reliance, as well as the benefits of adopting reliance mechanisms and opportunities for the countries in the region. She also talked in detail about postmarket surveillance and the regulatory approach to recalls; this was of interest for the Latin America regulators since several of them rely on the FDA’s recall information.
For the closed session, Cutts presented updates on the activities of the International Medical Device Regulators Forum (IMDRF), a voluntary group of medical device regulators from around the world aiming to accelerate international medical device regulatory harmonization and convergence. Brazil, Canada, and the United States are currently the only IMDRF members from the Americas. Argentina is an official observer, and Chile and Cuba are affiliate members who can participate in the IMDRF by attending open meetings and using IMDRF documents in part or in whole as the basis for their own regulatory framework. During her presentation, Cutts also provided valuable input during the discussions about the Latin America regulators participation in the IMDRF working groups.