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 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.
Resources, news and special offers to support you and your professional development during this difficult time.
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.
Download your copy of the new events calendar and see all the online workshops, conferences, RAC exams and European online workshops RAPS has planned for 2021 at a glance.
An invaluable resource for any professional engaged in designing, composing, compiling, or commenting on regulatory documentation
From self-assessments to help you identify your strengths and areas to focus on to reference books and online courses that will help you fill in the gaps in your regulatory knowledge, RAPS has the resources to help you prepare for the RAC exam.
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.
Posted 20 June 2017 | By Zachary Brennan
In addition to protecting the US blood supply and ensuring vaccines are safe and effective, the US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) is also now regulating complex gene and cell therapies, as well as a new category of treatments: regenerative medicines.
Peter Marks, director of CBER, offered attendees of DIA’s annual conference on Tuesday a few examples of the changing landscape for the center, noting that with the advent of CRISPR/Cas9 genome editing technology, the potential for new therapies is rapidly growing.
Thanks to the 21st Century Cures Act, FDA now has a new designation for regenerative medicines, known as the regenerative medicine advanced therapy (RMAT) designation. As of last week, Marks said there have been 19 requests for RMAT designations, 18 of which CBER has acted on, and four of which have been granted, including the first in May.
He also offered the example of hemophilia A treatments, which have progressed from blood transfusions in the 1930s to a possibly curative gene therapy, which he said he hopes could win approval before his retirement.
But one question he brought up momentarily: How much would such a gene therapy, especially if it was a cure, cost?
Right now, Marks said, a year’s worth of hemophilia A treatment can cost between $100,000 and $150,000, so one can imagine how expensive a gene therapy might cost.
“Thankfully at FDA we don’t worry about the price,” Marks said, noting that if a company “can cure it once and for all, it will be an interesting value proposal.”
As media reportshave shown, the prospects for a possibly curative hemophilia gene therapy are rising.
Marks also touched on the rapidly growing field of chimeric antigen receptor-T cells among biotech companies, noting the potential advantages and challenges.
And as far as FDA’s databases of CAR-T information, which have been evaluating the safety of these new therapies for more than a year, Marks said there are currently more than 100 INDs being tracked, many of which target the CD19 antigen. He said the tracking of the safety of such treatments is a good example of FDA being able to evaluate a rapidly developing field.
Tags: CBER, CAR-T, hemophilia, Peter Marks
Regulatory Focus newsletters
All the biggest regulatory news and happenings.