Welcome to our new website! If this is the first time you are logging in on the new site, you will need to reset your password. Please contact us at raps@raps.org if you need assistance.
The site navigation utilizes arrow, enter, escape, and space bar key commands. Left and right arrows move across top level links and expand / close menus in sub levels. Up and Down arrows will open main level menus and toggle through sub tier links. Enter and space open menus and escape closes them as well. Tab will move on to the next part of the site rather than go through menu items.
The regulatory function is vital in making safe and effective healthcare products available worldwide. Individuals who ensure regulatory compliance and prepare submissions, as well as those whose main job function is clinical affairs or quality assurance are all considered regulatory professionals.
Share your knowledge and expertise with your regulatory peers by submitting an in-depth, evidence-based article focusing on key areas and emerging issues in the global regulatory landscape.
One of our most valuable contributions to the profession is the Regulatory Code of Ethics. The Code of Ethics provides regulatory professionals with core values that hold them to the highest standards of professional conduct.
Your membership opens the door to free learning resources on demand. Check out the Member Knowledge Center for free webcasts, publications and online courses.
Like all professions, regulatory is based on a shared set of competencies. The Regulatory Competency Framework describes the essential elements of what is required of regulatory professionals at four major career and professional levels.
RAPS Euro Convergence brings regulatory peers from the EU and worldwide together in one forum to gain insights and exchange ideas on the region's most pressing issues. Register today to attend 10-12 May 2021.
Registration is now open for RAPS Convergence 2021! Gather with the regulatory community 12-15 September for four days of learning, engagement, and excitement.
With contributions from more than 30 authors from seven countries, the new edition incorporates a global overview of the field and is designed to help you get the most out of your regulatory intelligence endeavors.
Regipedia is an interactive resource created to benefit RAPS members with 24/7 access to more than 2,300 regulatory terms.
Hear from leaders around the globe as they share insights about their experiences and lessons learned throughout their certification journey.
Posted 06 December 2017 | By Zachary Brennan
The US Food and Drug Administration (FDA) on Wednesday released draft guidance that could help speed the development of treatments for rare pediatric disease drugs.
Janet Woodcock, director of FDA’s Center for Drug Evaluation and Research, said the draft guidance "could eliminate the need for certain clinical studies and, when pediatric clinical studies are needed, could reduce the total number of patients who would receive a placebo instead of a potentially helpful drug."
The 14-page draft uses Gaucher disease as an example for building an approach with a controlled, multi-arm, multi-company clinical trial that minimizes the number of patients necessary to be treated with placebo.
According to Wednesday’s Federal Register notice, the draft guidance was originally developed as a strategic collaboration between FDA and the European Medicines Agency (EMA) to enhance the efficiency of Gaucher disease drug development, which was released in 2014 for public comment. "The draft guidance is an updated version of the document and has no fundamental changes to the original intent and content," the notice said.
Though the general principles in the draft "should be viewed as a proposal only," FDA says, "The principles underlying the proposal may be extended to other areas of drug development in rare diseases."
For instance, it may be necessary to develop, validate and employ age-specific endpoints, the draft notes. And because "the quality of available clinical outcome assessments (COAs) can vary, qualification and standardization is strongly recommended. Developers are encouraged to discuss the selected COA for the outcomes of interest with the FDA; involvement of relevant stakeholders, including patients is encouraged."
The guidance also addresses long-term follow-up, extrapolation from adult studies to demonstrate efficacy, modeling and simulation to optimize the design of studies and inform dosing rationales, and a strategy for designing a multi-arm, multi-company drug development program.
"It includes a description of the main inclusion criteria, relevant age groups, suggested efficacy endpoints, and study duration. Although such a program can be very challenging, the aim of the strategic plan is not only to facilitate agreement on individual applications, but also to address the feasibility of developing multiple drug products for a rare disease in a time-efficient manner," the draft says.
Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model Guidance for Industry
Tags: rare pediatric disease, Gaucher, FDA draft guidance
Regulatory Focus newsletters
All the biggest regulatory news and happenings.