Newsletter | May 21, 2020

05.21.20 -- Effective Strategies For Biotech Studies

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. This podcast explores challenges sponsors are seeing as a result of this crisis, the impact those challenges are having on trial procedures, and how modifications to the statistical analysis plan can point a path forward.

Data Driven: How Electronic Patient-Reported Outcomes (ePROs) Provide A Vital Opportunity To Capture Key Data Beyond The COVID-19 Crisis

As the FDA and research community continue to evolve in terms of how and what data gets captured during the COVID-19 pandemic, one of the areas coming to the forefront is data collection using electronic patient-reported outcomes (ePRO) tools.

Delivering The Global Expertise Required To Coordinate A Pediatric Gene Therapy Trial

A European biotech company was conducting a pediatric trial involving a rare, incurable genetic disorder. In addition to new regulatory considerations, this trial also called for an extremely challenging intracerebral administration. To succeed, they needed a partner that could develop a clear, compliant strategy for obtaining informed consent and implementing a data management plan to track patients between surgical and clinical sites.

INTERACT Meetings: Streamlining Early Clinical Development

This document details recommendations for the Center for Biologics Evaluation and Research (CBER)’s engagement with sponsors in an early development discussion of a new biologics product. Read how the INTERACT (INitial Targeted Engagement for Regulatory Advice on CBER ProducTs) meeting can be an invaluable engagement with the agency to inform the successful planning and execution of a novel product development program.

iPSP Considerations For Molecularly Targeted Cancer Drugs

An initial pediatric study plan (iPSP) for a molecularly targeted oncology drug should address the drug’s molecular target and its relevance to one or more cancers that occur in the pediatric population. This article outlines the specific elements an iPSP is expected to include.