Medical and Regulatory Affairs

Clinical Research: Phase 1 - Phase 4

PM360: How COVID Has Reshaped the Clinical Trial Regulatory Landscape

The concept of patient centricity is hardly new, but in recent months it has assumed new urgency as industry and regulators have had to adapt to an evolving landscape. The evolution is spurring substantial changes in how clinical trials are developed and implemented, as regulators revise their processes and issue updated guidance documents to encourage...

Clinical Research: Phase 1 - Phase 4

Gene Therapy for Parkinson’s Disease: Strategies and Operational Requirements

Introduction Gene therapy holds great promise as a potential treatment for Parkinson’s disease, a disorder for which currently available medications do not causally treat the underlying disease mechanisms. However, given the early stage of gene therapy research in this therapeutic area, questions abound regarding its utility and practicality in treating Parkinson’s disease. Those questions have...

Consulting

New FDA Draft Guidance Provides Insights on Use of PROs in Oncology Trials

Incorporating patient-reported outcomes (PROs) into clinical trials can help sponsors better understand patients’ symptoms and how a therapy will affect their quality of life, and these insights can be particularly valuable in oncology research. However, there has been a lack of guidance to help sponsors ensure consistency and quality when incorporating PROs into cancer drug...

Medical and Regulatory Affairs

ANDA or 505(b)(2): Choosing the Right Abbreviated Approval Pathway for Your Drug

For developers seeking to obtain approval for previously approved drug products in the United States, the U.S. Food and Drug Administration (FDA) offers two abbreviated approval pathways — an abbreviated new drug application (ANDA) and a 505(b)(2) application. Both of these applications rely on the agency’s finding of safety and effectiveness for a listed drug,...

Clinical Research: Phase 1 - Phase 4

ContractPharma: Gene Therapy Trials

Since the first gene therapy trial in 1990, the regulatory landscape has shifted from broad, loosely defined guidance with little variation between different cell and gene therapies to a more robust collection of guidelines and directives focused on testing and manufacturing considerations for these products in specific disease states and therapeutic settings. This article will...

Clinical Research: Phase 1 - Phase 4

PharmaTimes: Keeping Pace

In recent years, the gene therapy regulatory landscape has evolved from a broad, general guidance to a narrower focus on testing and manufacturing considerations in specific disease states. This article explores how factors such as environmental risk assessment and biosafety can impact gene therapy clinical study start-up as well as the continuing evolution of guidances...

Medical and Regulatory Affairs

PharmaLive: Overcoming the Placebo Roadblock on the Path to Novel Analgesic Drug Development

Placebo response is one of the most significant challenges faced by drug developers who are investigating new pain medications. According to a review of published chronic neuropathic pain trials, placebo responses have increased in magnitude over time, making it even more difficult to definitively demonstrate treatment advantage.1 Research has also shown that up to 60...

Global Compliance

Frequently Asked Questions: GxP Quality Guidelines and Regulations

Overview What is GxP? GxP refers collectively to several different types of “good practice” quality guidelines and regulations, each serving a specific purpose. In pharmaceutical product development, these include but are not limited to: GCP (Good Clinical Practice)GLP (Good Laboratory Practice)GMP (Good Manufacturing Practice) GxP standards broadly cover what are commonly referred to as the...

Medical and Regulatory Affairs

Dermatology Webinar Series: Looking Ahead to the Future of Dermatology Research

New treatment options for moderate to severe dermatological diseases and areas of high unmet need are driving a revolution in clinical development of dermatology drugs. But questions remain: What indications should be pursued? How to design a study that targets the right endpoints? And how will environmental constraints affect the research? In this three-part series,...

Medical and Regulatory Affairs

Applied Clinical Trials: The Rise of Gene Therapy: Advanced Regulatory, Site, and Enrollment Considerations

Gene therapy development is gaining momentum. In 2020 alone, gene therapy attracted $12 billion in global financing through just the first three quarters with more than 370 gene therapy clinical trials underway worldwide.1 The U.S. Food and Drug Administration (FDA) itself predicted it may be approving 10 to 20 gene therapies per year by the...