Genetics/Genomics

FDA Comment Deadline: Limited Population Pathway for Antibacterial and Antifungal Drugs

The Food and Drug Administration (FDA) is seeking public comments on the draft guidance for industry, “Limited Population Pathway for Antibacterial and Antifungal Drugs,” that was published in the Federal Register on June 13, 2018 (83 FR 27616). FDA is reopening the comment period on this draft guidance for comments to be submitted for consideration before they finish work on the final version of the guidance.

This draft guidance is intended to assist sponsors in the development of certain new antibacterial and antifungal drugs for approval under the limited population pathway for antibacterial and antifungal drugs. This draft guidance also is intended to assist sponsors in developing labeling, including prescribing information, patient labeling, and carton/container labeling, that incorporates certain statements required by section of the Federal Food, Drug, and Cosmetic Act. 

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

FDA Meeting: Limited Population Pathway for Antibacterial and Antifungal Drugs

The Food and Drug Administration (FDA) is announcing the following public meeting entitled “Limited Population Pathway for Antibacterial and Antifungal Drugs.” The purpose of the meeting is to provide a public forum for FDA to listen to comments on the draft guidance for industry, “Limited Population Pathway for Antibacterial and Antifungal Drugs,” that was published in the Federal Register on June 13, 2018 (83 FR 27616).

This draft guidance provides information on the implementation of section 506(h) of the Federal Food, Drug, and Cosmetic Act (FD&C Act), added by section 3042 of the 21st Century Cures Act, which established the limited population pathway for antibacterial and antifungal drugs (LPAD pathway). This draft guidance is intended to assist sponsors in the development of certain new antibacterial and antifungal drugs for approval under the LPAD pathway. This draft guidance also is intended to assist sponsors in developing labeling, including prescribing information, patient labeling, and carton/container labeling, that incorporates certain statements required by section 506(h) of the FD&C Act. The LPAD pathway is intended to encourage the development of certain antibacterial and antifungal drugs for limited and specific populations of patients to help address the critical public health and patient care concern that has resulted from the current decline in antibacterial and antifungal drug research and development as serious antibacterial and antifungal drug-resistant infections increase.

 

FDA is holding a public meeting to receive information and comments concerning the draft guidance on the LPAD pathway from a broad group of stakeholders, including patients, researchers, healthcare providers, manufacturers, interested industry, professional organizations, and the public. The Agency has determined that a public meeting is the most appropriate way to ensure public engagement on the draft guidance. FDA welcomes any relevant information that stakeholders wish to share.

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

HRSA Teleconference: Advisory Council on Blood Stem Cell Transplantation

Update: on May 8, 2019 (84 FR 20153), the Health Resources and Services Administration rescheduled this event from May 7, 2019, to July 2, 2019.

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.

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: Bispecific Antibody Development Programs

The Food and Drug Administration (FDA) is announcing the availability of a draft guidance for industry entitled “Bispecific Antibody Development Programs.” This draft guidance provides recommendations to assist industry and other parties involved in the bispecific antibody drug development process. The draft guidance focuses on general regulatory and scientific considerations for bispecific antibodies, but not on development of a particular bispecific antibody. Industry and other stakeholders are encouraged to engage FDA to discuss their individual bispecific antibody under development.

This draft guidance discusses general considerations and recommendations, as well as regulatory, quality, nonclinical, and clinical considerations in the context of bispecific antibody development programs. This draft guidance does not discuss development considerations for other multitarget therapies that are combinations of monoclonal antibodies, antibody cocktails, or polyclonal antibodies.

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.

CMS Comment Deadline: Next Generation Sequencing for Medicare Beneficiaries with Advanced Cancer

The Centers for Medicare and Medicaid Services (CMS) is seeking public comment on whether to provide coverage for the use of next generation sequencing (NGS) technology focusing on germline (inherited) mutations for cancer diagnosis and treatment. CMS is particularly interested in comments that include scientific evidence and address the scope of this reconsideration.

NGS is a technology to read the order of nucleotide molecules on DNA and more effectively provides detailed information on multiple types of genetic alternations simultaneously. NGS oncology panel tests also provide patients and their providers a more comprehensive genetic profile of cancer and information relevant to potential cancer treatments.

To provide a complete picture of the cancer patient, a clinician may order both tests of somatic (cancer) and germline (inherited) mutations. It is important that tests detect these mutations accurately and produce valid results that are useful in guiding therapies to improve outcomes for patients with cancer. Therefore, CMS is internally opening this decision. Specifically, CMS is only reconsidering the evidence available for tests of germline mutations to identify those with hereditary cancer who may benefit from targeted treatments based on results of the test; all other tests are beyond the scope of this reconsideration.

Members of the public may submit comments online. More information is available at this CMS website.

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

NIH Meeting: National Human Genome Research Institute, National Advisory Council for Human Genome Research

The National Institutes of Health (NIH), National Human Genome Research Institute (NHGRI), will hold a meeting of its National Advisory Council for Human Genome Research.

The National Advisory Council advises the Department of Health and Human Services, the NIH, and the NHGRI on genetics, genomic research, training, and programs related to the human genome initiative.

The Advisory Council performs second-level peer review for grant applications, and determines the program priorities for NHGRI and the goals for the government's efforts in the International Human Genome Project.

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

NIH Meeting: National Cancer Institute, Council of Research Advocates

The National Institutes of Health (NIH), National Cancer Institute (NCI), will hold a meeting of their Council of Research Advocates. Agenda items for the meeting include updates on relevant budget and legislative items, an update from the NCI Director, and updates from the NCI's Office of Data Sharing, Rural Health Programs, and Cancer Information Service.

The Council is charged with providing advice to the NCI Director with respect to promoting research outcomes that are in the best interest of cancer patients. To this end, the Council will conduct these activities with the intent to identify new approaches, promote innovation, recognize unforeseen risks or barriers, and identify unintended consequences that could result from NCI decisions or actions. Additionally, the Council will provide insight into enhancing input, optimizing outreach, and promoting strong collaborations, all with respect to non-scientist stakeholders.

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