Stats #53: Signal Detection Strategies for Paediatric Treatments
Content: Signal detection and pharmacovigilance are already highly regulated and challenging fields, but once you factor in children as your subject group these challenges become even greater. There are physiologic, ethical, and statistical questions that you must consider for some (but not all) efforts in post marketing surveillance.
Discuss openly with your peers the issues that complicate drug safety studies in children and recognize when these issues apply and when they don't apply. Look at and debate the merits of alternative data sources, research designs, and statistical analyses to balance the sometimes conflicting needs of regulators, drug companies, and ethics review boards.
Teaching strategies: Didactic lectures and small group exercises.
Objectives: In this class you will learn how to
- understand the medical differences in children that make pediatric research unique.
- be able to describe the controversies over informed consent and monetary compensation in pediatric studies.
- be able to identify the unique statistical challenges associated with pediatric research.
Teaching strategies: Didactic lectures and small group exercises.
An important qualifier: There is an internet acronym used a lot in email and instant messaging: IANAL.
It stands for "I Am Not A Lawyer" and it is used when someone is commenting on a legal issue but wants to remind the readers that this informal advice is not intended as a substitute for formal legal advice by a qualified lawyer. In the same vein, I want to emphasize
IANAD.
I do work closely with doctors, nurses, and other health care professionals but as you listen to my advice, keep in mind that "I Am Not A Doctor". I certainly want to comment on medical controversies to make you aware of their importance, but I am not the final arbiter of any medical controversy. My role is more to raise questions than to answer them.
Outline of this talk.
Introduction
- Where can you find this handout?
- Why don't I use PowerPoint?
- Information about my book.
- Therapeutic orphans.
- What, exactly, is a child?
How are children different?
- Medical concerns
- Ethical concerns
- Statistical concerns
- Searching for pediatric studies
- Case studies
Conclusion
Conclusion
There are indeed important issues that you need to consider when conducting research in children. I'm hoping that this presentation will encourage you to tackle these problems though rather than view this as a series of insurmountable obstacles. We desperately need more work in this area.
Every practicing physician, especially pediatricians and pediatric surgeons, departments of pediatrics, and departments of pharmacology should closely examine their own capacities and performance in this area of greatly needed activity. If we are to have drugs of better efficacy and safety for children, those responsible for child care will have to assume this responsibility for developing active programs of clinical pharmacology and drug testing in infants and children. The alternative is to accept the status of "Therapeutic Orphans" for their patients. (Editorial Comment: Therapeutic Orphans. Shirkey H. Pediatrics 104(3): 583-584. [Full text] [PDF])