Genetics/Genomics

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 The FDA is proposing this action to clarify and update the regulations to reduce regulatory requirements that are outdated and duplicate other means to better protect the public health against harmful exposure to radiation emitting electronic products and medical devices.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for radiation emitting electronic products and medical devices because FDA has identified the regulations as being outdated and duplicative of other means for reducing radiation exposure to the public. The FDA is proposing to update the regulations to reduce regulations that are outdated and otherwise clarify requirements for protecting the public health against radiation exposure from specific electronic products and medical devices.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: 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

HRSA Meeting: Advisory Committee on Organ Transplantation

The Health Resources and Services Administration (HRSA) within the Department of Health and Human Services is announcing the forthcoming public meeting of the Secretary's Advisory Committee on Organ Transplantation (ACOT). ACOT advises the Secretary of HHS, through the HRSA Administrator, on all aspects of organ donation, procurement, allocation, and transplantation, and on such other matters that the Secretary determines; advises the Secretary on federal efforts to maximize the number of deceased donor organs made available for transplantation and to support the safety of living organ donation; at the request of the Secretary, reviews significant proposed Organ Procurement and Transplantation Network (OPTN) policies submitted for the Secretary's approval to recommend whether they should be made enforceable; and provides expert input on the latest advances in the science of transplantation.

During the May 20, 2019, meeting, ACOT will receive updates on recent activity of OPTN and discuss efforts to increase organ donation and transplantation. Agenda items are subject to change as priorities dictate. Refer to the ACOT website for any updated information concerning the meeting.

This meeting will be held by webinar; the webinar link and call-in number can be found at the ACOT website. More information regarding the meeting can be found at this Federal Register notice.

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

AHRQ Nominations: US Preventive Services Task Force

The Agency for Healthcare Research and Quality (AHRQ) within the Department of Health and Human Services is announcing a forthcoming nomination deadline for members of the US Preventive Services Task Force (USPSTF). The USPSTF, an independent body of experts in prevention and evidence- based medicine, works to improve the health of all Americans by making evidence-based recommendations about the effectiveness of clinical preventive services and health promotion. The recommendations made by the USPSTF address clinical preventive services for adults and children, and include screening tests, counseling services, and preventive medications.

The USPSTF was first established in 1984 under the auspices of the US Public Health Service. Currently, the USPSTF is convened by the Director of AHRQ, and AHRQ provides ongoing scientific, administrative, and dissemination support for the USPSTF's operation. USPSTF members serve four year terms. New members are selected each year to replace those members who are completing their appointments.

To qualify for the USPSTF and support its mission, an applicant or nominee should, at a minimum, demonstrate knowledge, expertise and national leadership in the following areas:

  1. The critical evaluation of research published in peer-reviewed literature and in the methods of evidence review;
  2. Clinical prevention, health promotion and primary health care; and
  3. Implementation of evidence-based recommendations in clinical practice including at the clinician-patient level, practice level, and health-system level.

Additionally, the Task Force benefits from members with expertise in the following areas:

  • Public health
  • Health equity and the reduction of health disparities
  • Application of science to health policy
  • Behavioral medicine
  • Communication of scientific findings to multiple audiences including health care professionals, policy makers and the general public
  • Candidates with experience and skills in any of these areas should highlight them in their nomination materials

Nominations should be submitted in writing by mail, or electronically by email. More information about nomination criteria and the nomination process can be found at this Federal Register notice.

CDC Meeting: Board of Scientific Counselors, Office of Infectious Diseases

The Centers for Disease Control and Prevention (CDC) will hold a meeting of its Board of Scientific Counselors, Office of Infectious Diseases (BSC, OID). The agenda will include updates from the infectious disease national centers and the Center for Global Health along with focused discussions on recent outbreaks and affected populations and the CDC Surveillance Strategy and Public Health Data Strategy. Reports back from three workgroups will also be given: (1) The Board's Acute Flaccid Myelitis (AFM) Task Force; (2) the Board's Food Safety Modernization Act Surveillance Working Group; and (3) the Vector-borne Diseases Workgroup of the BSC, OID, and the Board of Scientific Counselors, National Center for Environmental Health/Agency for Toxic Substances and Disease Registry. Agenda items are subject to change as priorities dictate.

The BSC, OID, provides advice and guidance to the Secretary, Department of Health and Human Services; the Director and the Deputy Director for Infectious Diseases (DDID), CDC; and the Directors of the National Center for Emerging and Zoonotic Infectious Diseases, the National Center for HIV/AIDS, Viral Hepatitis, STD, and TB Prevention, and the National Center for Immunization and Respiratory Diseases, CDC, in the following areas: Strategies, goals, and priorities for programs; research within the national centers; and overall strategic direction and focus of DDID and the national centers.

Members of the public may attend in person or via teleconference. More information is available at this Federal Register notice.

HRSA Teleconference: Advisory Council on Blood Stem Cell Transplantation

The Health Resources and Services Administration (HRSA) announces a forthcoming teleconference meeting of the Advisory Council on Blood Stem Cell Transplantation (ACBSCT). The ACBSCT provides advice and recommendations to the Secretary of HHS and the HRSA Administrator on the activities of the C.W. Bill Young Cell Transplantation Program (CWBYCTP) and the National Cord Blood Inventory (NCBI) Program. The principal purpose of these programs is to make blood stem cells from adult donors and cord blood units available for patients who need a transplant to treat life-threatening conditions such as leukemia, and who lack a suitably matched relative who can be the donor.

During the May 7, 2019, meeting, members of ACBSCT will discuss issues related utilization of cord blood for transplant and utilization of blood stem cells in cellular therapies.

This meeting will be held by webinar. Members of the public will have the opportunity to provide comments. Public participants may submit written statements in advance of the scheduled meeting. Oral comments will be honored in the order they are requested and may be limited as time allows. Requests to submit a written statement or make oral comments to ACBSCT should be sent to Robert Walsh, DFO. 

For more information regarding the webinar link, public comments, and a forthcoming agenda, please see this Federal Register notice.

NIH Meeting: Advisory Board, Fogarty International Center

The National Institutes of Health (NIH) will hold a meeting of the Advisory Board to the Fogarty International Center.

Fogarty's advisory board is comprised of researchers, policymakers and others involved in global health. Members meet three times each year to provide second-level review of Fogarty grant applications. They also provide strategic advice to the Center on its policies, programs and other matters.

Members of the public may attend in person. More information is available at this NIH website.

NIH Meeting: From Data to Discoveries — Creating a Research Program for All of Us

The National Institutes of Health (NIH) will hold a meeting titled "From Data to Discoveries: Creating a Research Program for All of Us." One year following the launch of the All of Us Research Program, what have we learned about how to build an engaged and diverse participant community and its potential for scientific impact? The event will feature NIH Director Dr. Francis Collins, All of Us Research Program Director Eric Dishman, National Institute on Drug Abuse Director Dr. Nora Volkow, and talks from representatives of Verily, Ciitizen, the Hunter-Bellevue School of Nursing, the University of Illinois at Chicago, and PASOs as they address these important questions.

Members of the public may attend in person or via webcast. More information is available at this NIH website.

HHS Meeting: Office of Global Affairs — Stakeholder Listening Session in Preparation for the 72nd World Health Assembly

The US Department of Health and Human Services (HHS)—charged with leading the US delegation to the 72nd World Health Assembly—will hold an informal Stakeholder Listening Session. The session is intended to help the HHS Office of Global Affairs prepare the US delegation for the World Health Assembly by taking full advantage of the knowledge, ideas, feedback, and suggestions from all individuals interested in and affected by agenda items to be discussed at the 72nd World Health Assembly.

The draft agenda for the 72nd World Health Assembly is now available. Members of the public may attend this listening session meeting by sending an RSVP by email in advance of the meeting. More information about the listening sessions 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.