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.
Registration is now open for RAPS Euro Convergence 2021! Attend to join peers from EU and around the world to gain insights and exchange ideas on the regions most pressing issues.
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 03 February 2016 | By Zachary Brennan
While the US Food and Drug Administration (FDA) sits on the fence over whether to approve preclinical or clinical trials using mitochondrial replacement techniques (MRT) to help prevent the transmission of certain diseases passed from mother to child, the Institute of Medicine (IOM) of the National Academies of Sciences, Engineering, and Medicine came out with a new report on Wednesday detailing how it believes FDA should allow such trials and regulate them.
The report, commissioned by FDA, is the result of the work of a committee of academic experts from Harvard, Johns Hopkins University, California Institute of Technology, the National Institutes of Health and other top research institutes.
The committee was tasked with examining the issues of MRT and recommending whether and how FDA should move forward with initial clinical investigations that could potentially disrupt the passage of some types of inherited mitochondrial DNA (mtDNA) diseases.
Examples of mitochondrial diseases, sometimes caused by mutations in the mtDNA (and sometimes caused by mutations in genes of the nuclear DNA (nDNA)), include Leigh syndrome and Leber’s hereditary optic neuropathy.
So why did FDA commission a report on this sensitive subject? Because MRT preclinical research or clinical investigations would involve the creation, manipulation and possible destruction of embryos, which has long been controversial in the US. As the committee notes, even an FDA request that data from such research be submitted in support of an IND (investigational new drug) to start first-in-human clinical investigations could be controversial.
The recently passed omnibus spending plan even included language preventing FDA from reviewing applications related to genome-editing tools to modify the DNA of human embryos “until serious and unquantifiable safety and ethical issues can be resolved.”
FDA's Center for Biologics Evaluation and Research spokesman Paul Richards told Focus:"FDA appreciates the thoughtful work of the IOM in issuing its report on mitochondrial replacement techniques. We will be carefully reviewing the report and recommendations."
Richards also noted how the omnibus "prevents the FDA from using funds to review applications in which a human embryo is intentionally created or modified to include a heritable genetic modification. As such, human subject research utilizing genetic modification of embryos for the prevention of transmission of mitochondrial disease cannot be performed in the United States in FY 2016."
So far, biologists at Oregon Health and Science University are some of the few who have sought FDA approval for a trial using a type of mitochondrial replacement therapy. As the authors note in that paper from 2013, it is “crucial” that FDA “initiates careful review of these new developments.”
Shoukhrat Mitalipov, a biologist at the Oregon University, told Nature on the committee report: “It’s good news, but the future seems very hazy compared to a few months ago,” noting the restrictions from the spending bill.
In the UK, legislation regulating the clinical application of MRT was approved by the House of Commons and the House of Lords last year.
According to the committee, MRT, if effective, could help women have a genetically related child without incurring the risk of passing on an mtDNA disease, though FDA must first address a “unique combination of characteristics that raises a novel collection of ethical, social, and policy issues.”
Such issues involve creating embryos via the transference of healthy human eggs, that if transferred would result in offspring with genetic material from two women of different maternal lineage, which has never been approved by FDA or other US federal regulatory authorities; allowing for modifications in the mitochondrial genome that could be heritable if MRT were carried out to conceive female offspring, due to the matrilineal inheritance of mtDNA, and the effects of those modifications (whether beneficial or deleterious); some of the genetic modifications of which any resulting effects would not, at this time, be reversible; and some genetic modifications would affect every cell type of the resulting individual, meaning the changes wouldn’t be confined to a specific organ system.
“The committee concludes that the most germane ethical, social, and policy considerations associated with MRT could be avoided through limitations on the use of MRT or are blunted by meaningful differences between the heritable genetic modification of nDNA and that introduced by MRT,” the committee said.
Glenn Cohen, faculty director of the Petrie-Flom Center for Health Law Policy, Biotechnology and Bioethics at Harvard Law School, wrote on the center’s blog Wednesday: “The big headline is they have recommended FDA largely move toward allowing [MRT] to go forward under a regulatory pathway with restrictions, the most important of which is the transfer only of male embryos (to avoid germ-line issues)."
The committee report outlines six recommendations for FDA, including:
And as the report notes, the prospect of FDA approving the use of MRT could have implications for off-label uses too, which the committee says: “FDA would need to use all of the tools at its disposal to control off-label uses of MRT beyond those indications and settings for which it had been tested and approved.”
IOM Report: Mitochondrial Replacement Techniques: Ethical, Social, and Policy Considerations
Tags: MRT, mitochondrial DNA, inherited diseases, FDA regulation of MRT, CRISPR
Regulatory Focus newsletters
All the biggest regulatory news and happenings.