Tag: FDA

COVID-19

PREMIER VOICES #11: Statistical Considerations in the Wake of COVID-19

COVID-19 has dramatically changed the way we conduct clinical trials and left many sponsors without answers when it comes to the future of their studies. Recognizing the issues is one thing, but figuring out where we go from here demands a new way of looking at some common challenges. In this episode, Premier Research’s Shari...

COVID-19

Statistical Considerations for FDA COVID-19 Guidance

In response to the newly released U.S. Food and Drug Administration (FDA) guidance on COVID-19, the Statistics Department at Premier Research has committed to highlighting specific and necessary actionable considerations that directly address key FDA recommendations in the guidance. These considerations call attention to some general actions required to ensure the operational integrity and scientific...

Consulting

Snapshot: A New Guidance Document Program From the FDA

As a regulatory affairs professional advising various stakeholders on the interpretation and implementation of guidance documents from the FDA, I can safely say that the task is challenging in the best of cases. FDA guidance documents have a tendency to be vague and difficult to decode. As experts, we are always debating about what the...

Consulting

INTERACT Meetings: Streamlining Early Clinical Development

In October 2018, the Food and Drug Administration (FDA) issued Standard Operating Policy and Procedure (SOPP) – SOPP 8214 Version 1.0: INTERACT Meetings with Sponsors for Drugs and Biological Products. This document details recommendations for the Center for Biologics Evaluation and Research (CBER)’s engagement with sponsors (such as industry and investigators) in an early development...

Medical and Regulatory Affairs

The FDA’s New Guidance on Natural History Studies in Rare Diseases: What You Need to Know

In March 2019, the FDA released draft guidance on the design and implementation of natural history studies to support the development of safe and effective treatments for rare diseases. The document, Rare Diseases: Natural History Studies for Drug Development,[1] addresses one of the major challenges sponsors encounter when developing therapies for rare disease: the lack...

Medical and Regulatory Affairs

Draft Guidance: Hematologic Malignancies: Regulatory Considerations for Use of Minimal Residual Disease in Development of Drug and Biological Products for Treatment

The FDA has released a new draft guidance for sponsors planning to use minimal residual disease (MRD) as a biomarker in clinical trials conducted under an investigational new drug application (IND) or to support marketing approval of drugs and biological products for the treatment of specific hematologic malignancies. Read the full guidance here or click...

Medical and Regulatory Affairs

Draft Guidance: Early Drug Development and the Role of Pre-IND Meetings

The FDA has released a new draft guidance for sponsors of drug and biological products for the treatment of rare diseases in early development and in the planning of and participation in formal pre-investigational new drug application (pre-IND) meetings. Read the full guidance here or click the image to download.

Medical and Regulatory Affairs

Are You Ready for the FDA’s eCTD Filing Deadline? That’s May 5, 2018. Yes. Tomorrow.

It’s already been three years since the FDA issued its “Guidance on Providing Regulatory Submissions in Electronic Format—Certain Human Pharmaceutical Product Applications and Related Submissions Using the eCTD Specifications.” This guidance established a mandatory requirement for electronic submission of certain applications, amendments, supplements, and reports to the FDA’s Center for Biologics Evaluation and Research (CBER)...

Medical and Regulatory Affairs

The Regulatory Landscape for Immuno-gene Therapeutics in Hematological Malignances

Over the past few years, there has been a dramatic increase in the use of genetically modified cells for cancer immunotherapy, including chimeric antigen receptor (CAR)-T cells, recombinant T-cell receptor T cells and genetically modified CD34+ cells for the treatment of hematological malignancies. Clinical trials of immuno-gene therapeutics are becoming increasingly common, and regulatory guidelines...

Medical and Regulatory Affairs

Learning to Listen: Voice of the Patient Grows Louder in Product Development

Days before Christmas 2017, a nationwide call went out requesting nominations of patients and caregivers to join a new public/private-sponsored Patient Engagement Collaborative. Among the promised duties for this yet-to-be-filled body of 16 patient community representatives: Propose new collaboration models in which patients and patient advocates are partners in healthcare product development and U.S. Food...