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By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developingtherapies for ultra-rare diseases and conditions that affect exceedingly small populations.
Bauer, Senior Regulatory Drug Expert & James E. Valentine — Incorporating patient and caregiver experiences into every phase of drugdevelopment has become increasingly prioritized during both development and review ( see, e.g. , previous coverage here ). There have been challenges in gene therapydevelopment.
These are exactly the types of things that were addressed at ELF Scientific Workshop in May 2024 which addressed challenges in developingtherapies for ultra rare diseases (see our blog about this here , which includes a link to the recording).
Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and controls (CMC).
Workshop addresses oncology dose optimization across full span of development In a series of broad-ranging, frank discussions, attendees at a joint FDA-American Association of Cancer Research (AACR) workshop worked through opportunities and challenges for dose optimization across the span of cancer drugdevelopment activities.
The unique testing platform combines machine learning, automation and high-throughput screening directly on patient samples to predict responses to potential therapies and ultimately determine which therapies will be most effective for specific cancers. Learn more at notablelabs.com and follow us @notablelabs.
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molecular assessment within two weeks after receiving plasmids, to advance R&D process for antibody drugdevelopment. With its sophisticated quality and service systems, JS Bio will accelerate the application of Etta Biotech’s products in antibody drugdevelopment in the industry. About JS Bio.
Title Type Comments Close Key Information and Facilitating Understanding in Informed Consent Guidance for Sponsors, Investigators, and Institutional Review Boards Draft Guidance April 30 Early Alzheimer’s Disease: DevelopingDrugs for Treatment Draft Guidance May 13 Select Updates for the Premarket Cybersecurity Guidance: Section 524B of the (..)
Title Type Comments Close Risk Evaluation and Mitigation Strategy Logic Model: A Framework to Link Program Design With Assessment; Draft Guidance Draft Guidance August 5 Standardized Format for Electronic Submission for Marketing Applications Content for the Planning of Bioresearch Monitoring Inspections for Center for Biologics Evaluation and Research (..)
You will meet individuals, advocacy leaders, industry, and research experts who have been there and done it, over two days of in-person only panel discussions, hands-on workshops, and expert office hours.” LET’S HELP THEM FLY! RDDS 2023 focuses on collaboration – the foundation of success in rare disease research.
By offering a range of services, such as vaccination clinics, wellness programs, and educational workshops, these facilities support the health and well-being of entire communities. This includes providing accessible therapy rooms, communication aids, and flexible service options.
2 Days of Kids Summer Camp” appeared to be “2 Days of Spa and rejuvenation therapy for Mothers” I squished my eyes to take a second look and it said, “2 Days of Kids Summer Camp”. While the parents were all busy with work, the kids had a puppet-making workshop. The moment I received the mail, I enrolled my Kids. “2
By Amanda Conti | Aug 13, 2024 10:00 PM CDT Regulatory context: Psychedelic regulation and drugdevelopment A growing body of evidence suggests that psychedelics may provide clinical benefit for certain purposes, especially mental health conditions. Importantly, midomafetamine was proposed for use in conjunction with psychotherapy.
Title Type Comments Close Notification of a Permanent Discontinuance or Interruption in Manufacturing Under Section 506C of the Federal Food, Drug, and Cosmetic Act Guidance June 5 Patient Preference Information—Submission, Review in Premarket Approval Applications, Humanitarian Device Exemption Applications, and De Novo Requests, and Inclusion in (..)
In a first, FDA tackles treatments for stimulant use disorder A new draft guidance from the FDA gives developers a roadmap to advance the development of novel therapies to address stimulant use disorders. Regulatory context FDA held a workshop in December 2019 to discuss development of substance use disorder therapies.
Title Type Comments Close Classification Categories for Certain Supplements Under BsUFA III Guidance October 10 Formal Meetings Between the Food and Drug Administration and Sponsors or Applicants of Biosimilar User Fee Act Products Guidance October 10 QTc Information in Human Prescription Drug and Biological Product Labeling Guidance October 10 Postmarketing (..)
Today, the FDA unveiled a contract notice that explains the process for how it will develop that report, obtain public input, and deliver the report’s final recommendations. a disease or condition is defined as rare by the Orphan Drug Act (ODA) if it affects fewer than 200,000 people. In the U.S.,
CDER is also on track to complete the 58 first-year requirements under the latest reauthorization of the Prescription Drug User Fee Act (PDUFA), which included updated programs or processes, web updates, guidance documents, public meetings or workshops, and more. With 15 gene therapies now approved in the U.S.,
Title Type Comments Close Fixed-Combinations and Single-Entity Versions of Previously Approved Antiretrovirals for the Treatment or Prevention of Human Immunodeficiency Virus-One Under the President’s Emergency Plan for Acquired Immunodeficiency Syndrome Relief Guidance November 1 Post-Warning Letter Meetings Under GDUFA Guidance November 6 In-Home (..)
As the FDA’s Center for Drug Evaluation and Research (CDER) characterized it in a 2023 call for comments : “Evolving understanding of disease biology and molecular pharmacology, advancements in drug discovery, and growth in novel therapeutic platforms (i.e., One key update compared to previous work: Moving from pilots to programs.
Additionally, the plan aims to improve the quality of life for the over 12 million cancer survivors who benefited from earlier detection, effective therapies and supportive care. The EMA started providing guidance on the clinical development of anticancer therapeutics in 1996. Even under the shadow of the Covid-19 pandemic, 1.2
The side effects of some anticancer treatments can result in dose interruptions or de-escalations, or even outright discontinuation of an efficacious therapy. In 2015, the FDA, along with the American Association for Cancer Research (AACR), held a workshop on dose optimization for small molecules.
EMA’s psychedelic workshop leaves stakeholders with as many questions as answers Earlier this year, the EMA hosted a multi-stakeholder workshop to discuss regulatory issues surrounding drugdevelopment of psychedelic products. EMA has now published a report on the workshop; read on for AgencyIQ’s analysis.
Title Type Comments Close Digital Health Technologies for Detecting Prediabetes and Undiagnosed Type 2 Diabetes January 31 Advancing the Development of Therapeutics Through Rare Disease Patient Community Engagement Meeting February 12 Advisory Committee; Genetic Metabolic Diseases Advisory Committee; Establishment Notice February 12 Advanced Manufacturing (..)
Title Type Date Cleared by OIRA Legal Deadline None Notable FDA Comment Periods Closing in December and January FDA comment periods are typically open for 30-60 days, unless they are extended.
Title Type Comments Close Digital Health Technologies for Detecting Prediabetes and Undiagnosed Type 2 Diabetes Request for Information February 29 (Extended) Requests for Reconsideration at the Division Level Under the Generic Drug User Fee Amendments Draft Guidance March 11 Advanced Manufacturing Technologies Designation Program Draft Guidance March (..)
Identifying a safe and effective dose for registrational trials is one of the most critical and complex aspects of early-stage drugdevelopment for rare diseases. Traditionally, early drugdevelopment focuses on ensuring the safety of clinical doses, with regulatory authorities prioritizing patient protection.
Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. CBERs approach here was to take FAQs from across sponsor interactions, public workshops, email requests, etc.
Established in 2017 under the 21st Century Cures Act, the OCE brings together multidisciplinary scientific expertise to accelerate the review and approval of drugs, biologics, and medical devices for cancer care. These reviews have provided life-saving cancer therapies a median of 3.1
As Executive Director of CMC Regulatory Affairs at Biohaven , she leads efforts to develop innovative therapies from the clinic to market approval. Her expertise in regulatory strategy and process development has been instrumental in advancing important treatments.
September 2023 Legislative deadlines in Q3 Legislation Program Tag Requirement Due Date FDORA, Section 3202 Orphan Drug Program FDA shall publish final guidance related to the draft guidance titled, “Rare Diseases: Common Issues in DrugDevelopment.” The workshop will solicit and include industry and public feedback.
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