REGULATORY MEDICAL WRITING FOR RARE DISEASES: Tips and online resources.
- Tristan GICQUEL
- Dec 4, 2023
- 2 min read
Updated: Feb 28, 2024
MY JOURNEY AS A REGULATORY MEDICAL WRITER IN R&D FOR RARE DISEASES: CHALLENGES, DISCOVERIES, AND ACHIEVEMENTS
"Over the past few years, my work in rare diseases has led me to exciting projects where I've blended medical-regulatory expertise with research approaches to promote the scientific, commercial, and medical rationale behind developing therapeutic innovations.
Each challenge in deciphering complex, often under-documented cases, felt like an exhilarating quest for a 'needle in a haystack'.
In my regulatory medical writing projects for rare diseases, I've enjoyed investigating, researching, and harmonizing data to provide sufficiently robust and innovative studies for healthcare professionals, pharmaceutical industries, and regulatory decision-makers, contributing to the development of new therapies with the ultimate goal of improving patient care.
Each advancement, whether in diagnosis or the development of new therapies, represents a victory for me. That's why I'm excited to share essential tools and resources that can enlighten and facilitate our research in this fascinating and complex field."
ONLINE RESOURCES FOR REGULATORY MEDICAL WRITING IN RARE DISEASES
Here are some online resources you may find useful if you've exhausted your PubMed search. Moreover, you'll likely have a specific rare disease to describe, which can help narrow down your search to find ideal resources for your needs.
For epidemiological, medical, and sociological research :
Orphanet stands out with its comprehensive database on rare diseases, providing vital diagnostic tools and detailed information on orphan drugs, enriched with precise data on the prevalence and epidemiology of each rare disease to inform healthcare professionals and medical writers.
An integrated bioinformatics resource offering a centralized platform for accessing comprehensive data on rare diseases, thus facilitating research, analysis, and medical and regulatory writing in this field.
A unique hub of information, offering not only in-depth data on genetic conditions and rare diseases, compiling sources from Orphanet and RARe-SOURCE, but also integrating continuous scientific updates and patient support resources, making it crucial for comprehensive and cutting-edge medical writing.
A leader in genomic research, providing deep knowledge of the genetic bases of rare diseases and scientific advancements in this field. Its added value lies in its ability to illuminate the complex regulatory aspects related to genetic diseases, providing crucial information for robust, evidence-based regulatory arguments.
Essential for obtaining high-quality systematic reviews and meta-analyses on rare disease treatments, offering critical assessments and syntheses of existing literature, crucial for a thorough, evidence-based understanding of medical interventions in this specialized field.
For in-depth research on ontology and the international classification of diseases :
International Classification of Diseases 11th Revision (ICD-11): Provides a standardized international classification of diseases, crucial for medical and regulatory writers, facilitating consistent coding and communication of rare diseases worldwide.
As the most comprehensive repository of biomedical ontologies, it provides essential resources for the classification and terminology of rare diseases, enabling a deep understanding and precise classification of pathologies in the medical field.
An exhaustive database on human genes and genetic disorders, offering detailed and specific information on the genetics of rare diseases, essential for their accurate identification and classification.cises.
For research on chemical engineering and the study of active principle mechanisms of action :
A key resource for research in chemical and pharmacological engineering, offering detailed information on receptors and drug action mechanisms, crucial for understanding the molecular interactions of active principles in rare disease treatments.
Serves as an exhaustive database for compound chemistry, providing essential information on chemical structures, properties, and biological activities of active principles, indispensable for studies on action mechanisms and the development of new treatments for rare diseases.s.
For regulatory research and identification of authorized therapies in Europe, Canada, the U.S., and Japan :
EMA is a key resource for understanding European regulatory frameworks for rare disease drugs, offering detailed insights into approved therapies and requirements for market authorization in Europe.
CADTH provides essential assessments of the effectiveness and safety of rare disease drugs in Canada, facilitating the identification of authorized therapies and Canadian regulatory standards in this field.
FDA is an essential source for drug approvals in the United States, offering updated information on rare disease treatment approval processes, as well as regulatory guidelines and specific evaluation criteria in the U.S.
PMDA offers detailed information on regulatory standards and drug approval procedures for rare diseases in Japan, essential for understanding the regulatory landscape and therapeutic options available in this country.
And finally, the non-exhaustive list of specialized journals in rare diseases that are open access and peer-reviewed :
With their open-access, peer-reviewed publications, these journals are also valuable resources for medical and regulatory writers, offering in-depth perspectives and updates on the latest research, innovative treatments, and advancements in the field of rare diseases and orphan drugs.
HOPING THIS BRIEF SUMMARY HELPS YOU IN YOUR FUTURE WRITING PROJECTS IN R&D FOR RARE DISEASES!
If you need help with your medical or regulatory writing, feel free to contact me at contact@orphanquantum.com
See you soon,
Tristan.
* If I find more resources, I will add them to this list. ** Please let me know if you are aware of other rare disease resources that I may have missed and that could be relevant to include in this list.
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