Food and Drug Administration (FDA)

FDA Comment Deadline: Amendments to Regulations on Records, Reports, and Performance Standards for Radiation Emitting Electronic Products

The Food and Drug Administration (FDA) is seeking comments on a proposed rule to amend and repeal parts of the radiological health regulations covering recommendations for radiation protection during medical procedures, certain records and reporting for electronic products, and performance standards for diagnostic x-ray systems and their major components, laser products, and ultrasonic therapy products. The FDA is proposing this action to clarify and update the regulations to reduce regulatory requirements that are outdated and duplicate other means to better protect the public health against harmful exposure to radiation emitting electronic products and medical devices. The FDA is comprehensively reviewing existing regulations to identify opportunities for repealing and amending regulations that will result in meaningful burden reduction while allowing the Agency to achieve our public health mission and fulfill statutory obligations.

This proposed rule would amend and repeal certain regulations for radiation emitting electronic products and medical devices because FDA has identified the regulations as being outdated and duplicative of other means for reducing radiation exposure to the public. The FDA is proposing to update the regulations to reduce regulations that are outdated and otherwise clarify requirements for protecting the public health against radiation exposure from specific electronic products and medical devices. The regulations being proposed for amending or repealing are the radiation protection recommendations for specific uses, records and reporting requirements for electronic products, applications for variances, and performance standards for diagnostic x-ray systems and their major components, laser products, and ultrasonic therapy products.

Members of the public may submit comments via mail, fax, or at Regulations.gov. More information is available at this Federal Register notice

FDA Comment Deadline: Patient Perspectives on the Impact of Rare Diseases — Bridging the Commonalities

The Food and Drug Administration (FDA) is seeking public comment to accompany the meeting titled  “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities.”

This public meeting is intended to obtain patients' and caregivers' perspectives on impacts of rare diseases on daily life and to assess commonalities that may help the Agency and medical product developers further understand and advance the development of treatments for rare diseases. Developing a treatment for a rare disease can present unique challenges, such as the small number of individuals affected and heterogenous etiologies and manifestations. While the differences between rare diseases are critically important, it is also important to assess commonalities to synergize product development in rare diseases. The goal of this meeting is to identify common issues and symptoms in rare diseases to help advance medical product development, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.

The development of drugs, biologics, and devices for rare diseases involves unique challenges. The goal of this meeting is to identify common issues across rare diseases to help address some of these challenges. Rare diseases, often referred to as orphan diseases, are defined based on rarity of occurrence. Although these diseases are individually rare, collectively they are not. According to the National Institutes of Health, there are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States. Many of these rare diseases are serious or life-threatening and many affect children.

The combination of government incentives and scientific advances has fueled extraordinary development in orphan drugs. Since the Orphan Drug Act was passed in 1983, drugs and biologics for over 750 rare disease indications have been developed and approved for marketing. In addition to drugs and biologics, there has been progress in the development of devices for rare diseases. Since 1990, the FDA has approved 74 medical devices for orphan indications under the Agency's Humanitarian Device Exemption program. Despite these successes, we recognize that thousands of rare diseases still have no approved treatments.

Developing a treatment for a rare disease can present unique challenges. Potential challenges include the small number of individuals affected, lack of understanding of the natural history of the disease, phenotypic heterogeneity, and lack of validated endpoints for use in clinical trials. Overcoming these challenges requires collaboration between many stakeholders, including scientists, product developers, regulators, policy makers, and patients. FDA is committed to working with stakeholders to advance treatment options for patients with rare diseases.

This public meeting will focus on the perspective of those affected by rare diseases. Patients, family members, and caregivers will provide important input on the impact of rare diseases on daily life. While the differences between rare diseases are critically important, this meeting will assess commonalities. The specific goal of this meeting is to identify common issues and symptoms in rare diseases to help advance medical product development, potentially through the generation of novel endpoints or trial designs that focus on commonalities across a variety of rare disease.

This public meeting will consist of panels of patients/caregivers and facilitated discussions. The aim of the meeting is to hear directly from patients with rare diseases and their caregivers and family members. The meeting will include patients with any rare disease and their caregivers and family members. It is not restricted to a specific rare disease or group of rare diseases.

Members of the public may submit comments online at Regulations.gov or via mail. More information about the meeting and the comment deadline is available at this Federal Register notice.

FDA Comment Deadline: Sunscreen Drug Products for Over-the-Counter Human Use

The Food and Drug Administration (FDA) is issuing this proposed rule and seeking public comment to put into effect a final monograph for nonprescription, over-the-counter (OTC) sunscreen drug products. This proposed rule describes the conditions under which FDA proposes that OTC sunscreen monograph products are generally recognized as safe and effective (GRASE) and not misbranded. It is being published as part of the ongoing review of OTC drug products conducted by FDA. 

Members of the public can comment by mail, fax, or at Regulations.gov. More information is available at this Federal Register notice

FDA Comment Deadline: International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use

The Food and Drug Administration (FDA) is seeking public comments to accompany an upcoming, regional, public meeting entitled “US Food and Drug Administration and Health Canada Joint Regional Consultation on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).” The purpose of the public meeting is to provide information and solicit public input on the current activities of the ICH, as well as the upcoming ICH Assembly Meeting and the Expert Working Group Meetings in Amsterdam, Netherlands, scheduled for June 2 through 6, 2019. The topics to be addressed at the public meeting are the current ICH guideline topics under development that will be discussed at the forthcoming ICH Assembly Meeting in Amsterdam.

The ICH, formerly known as the International Conference on Harmonisation, was established in 1990 as a joint regulatory/industry project to improve, through harmonization, the efficiency of the process for developing and registering new medicinal products in Europe, Japan, and the United States without compromising the regulatory requirements for safety and effectiveness. One of the goals of harmonization is to identify and then reduce regional differences in technical regulatory requirements for pharmaceutical products while preserving a consistently high standard for drug efficacy, safety, and quality.

In 2015, the ICH was reformed to establish it as a true global initiative and to expand beyond the previous ICH members. More involvement from regulators around the world is expected, as they join counterparts from Europe, Japan, the United States, Canada, and Switzerland as ICH observers and regulatory members. Expanded involvement is also anticipated from global regulated pharmaceutical industry parties, joining as ICH observers and industry members. The reforms built on a 25-year track record and have allowed ICH to continue its successful delivery of harmonized guidelines for global pharmaceutical development and their regulation.

Members of the public may submit comments via mail, fax, or at Regulations.gov. More information is available at this Federal Register notice or at the FDA website

FDA Comment Deadline: Development of Antibacterial Drugs for the Treatment of Nontuberculous Mycobacterial Disease

The Food and Drug Administration (FDA) is seeking public comments to accompany the public workshop entitled “Development of Antibacterial Drugs for the Treatment of Nontuberculous Mycobacterial Disease.” The purpose of the public workshop is to discuss the clinical trial design considerations, including endpoints, related to the development of antibacterial drug products for treatment of nontuberculous mycobacterial (NTM) disease.

Members of the public may submit comments by mail, fax, or at Regulations.gov. More information is available at this Federal Register notice

FDA Workshop: Generic Drug Regulatory Science Initiatives

The Food and Drug Administration (FDA) will hold a public workshop that will provide an overview of the current status of the regulatory science initiatives for generic drugs and will provide an opportunity for public input on research priorities in these topic areas. FDA is seeking input from a variety of stakeholders––industry, academia, patient advocates, professional societies, and other interested parties––as it fulfills its commitment under the reauthorization of the Generic Drug User Fee Amendments to develop an annual list of regulatory science initiatives specific to generic drugs. FDA will take the information it obtains from the public workshop into account in developing the fiscal year 2020 Regulatory Science Plan. 

Members of the public may attend in person or via webcast with advance email registration. More information, including the agenda, is available at this FDA website.

FDA Comment Deadline: Modifications to Compliance Policy for Certain Deemed Tobacco Products

The Food and Drug Administration (FDA) is announcing the availability of and seeking public comment on a draft guidance for industry entitled “Modifications to Compliance Policy for Certain Deemed Tobacco Products.” The draft guidance discusses changes to the compliance policies for pre-market review requirements for certain deemed tobacco products and describes how FDA intends to prioritize its enforcement resources with regard to the marketing of certain deemed tobacco products that do not have pre-market authorization.

Members of the public may submit comments by mail, fax, or at Regulations.gov. More information is available at this Federal Register notice

FDA Comment Deadline: Enforcement Policy for Certain Marketed Tobacco Products

The Food and Drug Administration (FDA) is announcing the availability of and seeking public comment on a draft guidance for industry entitled “Enforcement Policy for Certain Marketed Tobacco Products.” FDA is issuing this draft guidance to provide information regarding FDA's enforcement policy for certain marketed tobacco products that become the subject of a not substantially equivalent (NSE) order. This policy extends to new tobacco products created by modifying the quantity of a provisional tobacco product in a pending substantial equivalence report that become subject to NSE orders.

The draft guidance also provides information on FDA's enforcement policy for when FDA receives from an applicant a request for supervisory review under 21 CFR 10.75 within 30 calendar days of the issue date of the NSE order. The draft guidance provides that for these new tobacco products, FDA intends to offer copies of those final scientific reviews that supported the basis of the Agency's decision to the applicant concurrent with the NSE order for provisional tobacco products. 

FDA is issuing this draft guidance consistent with FDA's good guidance practices regulation (21 CFR 10.115). The draft guidance, when finalized, will represent the current thinking of FDA on “Enforcement Policy for Certain Marketed Tobacco Products".

Comments on this draft guidance may be submitted electronically through this portal at Regulations.gov, or via mail. For more information regarding the draft guidance and the comment submission process, please see this Federal Register notice

FDA Meeting: Patient Perspectives on the Impact of Rare Diseases — Bridging the Commonalities

The Food and Drug Administration (FDA) is announcing an upcoming public meeting titled  “Patient Perspectives on the Impact of Rare Diseases: Bridging the Commonalities.”

This public meeting is intended to obtain patients' and caregivers' perspectives on impacts of rare diseases on daily life and to assess commonalities that may help the Agency and medical product developers further understand and advance the development of treatments for rare diseases. Developing a treatment for a rare disease can present unique challenges, such as the small number of individuals affected and heterogenous etiologies and manifestations. While the differences between rare diseases are critically important, it is also important to assess commonalities to synergize product development in rare diseases. The goal of this meeting is to identify common issues and symptoms in rare diseases to help advance medical product development, potentially through the creation of novel endpoints or trial designs that focus on commonalities across a variety of rare diseases.

The development of drugs, biologics, and devices for rare diseases involves unique challenges. The goal of this meeting is to identify common issues across rare diseases to help address some of these challenges. Rare diseases, often referred to as orphan diseases, are defined based on rarity of occurrence. Although these diseases are individually rare, collectively they are not. According to the National Institutes of Health, there are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States. Many of these rare diseases are serious or life-threatening and many affect children.

The combination of government incentives and scientific advances has fueled extraordinary development in orphan drugs. Since the Orphan Drug Act was passed in 1983, drugs and biologics for over 750 rare disease indications have been developed and approved for marketing. In addition to drugs and biologics, there has been progress in the development of devices for rare diseases. Since 1990, the FDA has approved 74 medical devices for orphan indications under the Agency's Humanitarian Device Exemption program. Despite these successes, we recognize that thousands of rare diseases still have no approved treatments.

Developing a treatment for a rare disease can present unique challenges. Potential challenges include the small number of individuals affected, lack of understanding of the natural history of the disease, phenotypic heterogeneity, and lack of validated endpoints for use in clinical trials. Overcoming these challenges requires collaboration between many stakeholders, including scientists, product developers, regulators, policy makers, and patients. FDA is committed to working with stakeholders to advance treatment options for patients with rare diseases.

This public meeting will focus on the perspective of those affected by rare diseases. Patients, family members, and caregivers will provide important input on the impact of rare diseases on daily life. While the differences between rare diseases are critically important, this meeting will assess commonalities. The specific goal of this meeting is to identify common issues and symptoms in rare diseases to help advance medical product development, potentially through the generation of novel endpoints or trial designs that focus on commonalities across a variety of rare disease.

This public meeting will consist of panels of patients/caregivers and facilitated discussions. The aim of the meeting is to hear directly from patients with rare diseases and their caregivers and family members. The meeting will include patients with any rare disease and their caregivers and family members. It is not restricted to a specific rare disease or group of rare diseases.

Members of the public may attend in person. Registration for the meeting will be done online, with option to register for the comment period on the day of the meeting. For more information regarding the registration process, along with a link to a forthcoming agenda, please see this Federal Register notice.

FDA Meeting: International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use

The Food and Drug Administration (FDA) is announcing an upcoming, regional, public meeting entitled “US Food and Drug Administration and Health Canada Joint Regional Consultation on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH).” The purpose of the public meeting is to provide information and solicit public input on the current activities of the ICH, as well as the upcoming ICH Assembly Meeting and the Expert Working Group Meetings in Amsterdam, Netherlands, scheduled for June 2 through 6, 2019. The topics to be addressed at the public meeting are the current ICH guideline topics under development that will be discussed at the forthcoming ICH Assembly Meeting in Amsterdam.

The ICH, formerly known as the International Conference on Harmonisation, was established in 1990 as a joint regulatory/industry project to improve, through harmonization, the efficiency of the process for developing and registering new medicinal products in Europe, Japan, and the United States without compromising the regulatory requirements for safety and effectiveness. One of the goals of harmonization is to identify and then reduce regional differences in technical regulatory requirements for pharmaceutical products while preserving a consistently high standard for drug efficacy, safety, and quality.

In 2015, the ICH was reformed to establish it as a true global initiative and to expand beyond the previous ICH members. More involvement from regulators around the world is expected, as they join counterparts from Europe, Japan, the United States, Canada, and Switzerland as ICH observers and regulatory members. Expanded involvement is also anticipated from global regulated pharmaceutical industry parties, joining as ICH observers and industry members. The reforms built on a 25-year track record and have allowed ICH to continue its successful delivery of harmonized guidelines for global pharmaceutical development and their regulation.

Members of the public may attend the meeting in person with advance registration, or by webcast with advance registration. More information is available at this Federal Register notice or at the FDA website

FDA Comment Deadline: Implanted Brain-Computer Interface Devices for Patients With Paralysis or Amputation — Nonclinical Testing and Clinical Considerations

The Food and Drug Administration (FDA) is announcing a forthcoming comment deadline on a draft guidance titled "Implanted Brain-Computer Interface Devices for Patients With Paralysis or Amputation-Nonclinical Testing and Clinical Considerations."

Implanted brain-computer interface (BCI) devices are neuroprostheses that interface with the central or peripheral nervous system to restore lost motor and/or sensory capabilities in patients with paralysis or amputation. This draft guidance provides recommendations for nonclinical testing and study design considerations for investigational device exemptions feasibility and pivotal clinical studies. This draft guidance is not final nor is it in effect at this time.

The field of implanted BCI devices is progressing rapidly from fundamental neuroscience discoveries to translational applications and market access. Implanted BCI devices have the potential to bring benefit to people with severe disabilities by increasing their ability to interact with their environment, and consequently, providing new independence in daily life. On November 21, 2014, the Center for Devices and Radiological Health (CDRH) held an open public workshop with the aim of fostering an open discussion on the scientific and clinical considerations associated with the development of implanted BCI devices. FDA considered the input provided during this workshop to develop this guidance document. This guidance document provides clinical study design and nonclinical testing recommendations associated with BCI devices.

This is a leapfrog guidance: A type of guidance that serves as a mechanism by which the Agency can share initial thoughts regarding emerging technologies that are likely to be of public health importance early in product development. This leapfrog guidance represents the Agency's initial thinking and our recommendations may change as more information becomes available.

Comments on this draft guidance may be submitted through this online portal at Regulations.gov, or via mail in written form. For more information on the draft guidance or the comment submission process, please see this Federal Register notice.

FDA Workshop: Development of Tissue-Agnostic, Biomarker-Based Indications

The Food and Drug Administration (FDA), in cooperation with the American Society of Clinical Oncology and Friends of Cancer Research, is hosting a workshop titled "Development of Tissue-Agnostic, Biomarker-Based Indications." The workshop aims to identify clinical trial designs and strategies to accelerate drug development based on biomarkers that are agnostic to tissue types. The workshop is intended to explore concepts in research and development that would assist the research community and regulators in understanding how to optimize product development and patient access to safe and effective biomarker-driven therapies that have clinical use across a variety of cancer types. 

Workshop objectives include:

  • Seek feedback from industry, academia, patients and other stakeholders on trial designs and analysis approaches that will aid in understanding the effect of a biomarker and its related diagnostic assay across multiple tissue types;
  • Ensure that cancer patient advocates and the leading experts in clinical research, pharmacology, drug manufacturing, and clinical care have an opportunity to contribute to the discussion about how to demonstrate safety and efficacy of a drug across multiple cancer types and how to communicate that information to the clinical community; and
  • Obtain input from multiple stakeholders on approaches the FDA uses to review applications for drug products that are intended to treat cancers based on the presence of a biomarker, rather than specification of the tissue type.

Members of the public may attend in person or via webinar with advance registration. More information, including the agenda, is available at this FDA website.