Melissa Wojcik, M.S., is an associate scientist III with the Translational Sciences Department at Biogen Idec in Cambridge, Mass. Her work supports preclinical and clinical assay development for the company’s neurologically based disease therapeutics.
The other day I found myself in an awkward conversation after stepping onto the elevator. After little exchanges about the weather and this year’s flu rates, my elevator companion asked me a simple question: “So what do you do?” I could have simply answered that I am a bioanalytical scientist at Biogen Idec who does data support preclinical toxicology studies for biotherapeutics, but that’s sterile. I went with a different angle. I gave him my elevator pitch: “You know those ridiculously long and garbled scientific documents packed inside the prescriptions you buy?” He chuckled to himself as he realized that he practically threw out every insert he ever pulled out of his prescriptions over the years. I continued, “That’s what I do. My data defines the safety and efficacy drug profile in the prescription inserts. Most people just throw them out, but they’re extremely important documents. They tell you the drug is safe and effective.”
Besides describing a drug’s life story or safety profile, the inserts also outline the disease indication, dosage and administration, dosage forms and strengths, contraindications, warnings and precautions, adverse reactions, drug interactions, use in specific populations, the overdosing consequences, chemical structural description, clinical pharmacology, nonclinical toxicology, clinical studies, how it is supplied, stored, and handled, and patient counseling information (e.g., Tamiflu package insert). The drug companies would prefer not to include them because they may cause their publically traded stocks to crash by warning consumers of potential issues: If people knew the full risk safety benefit, then they might choose a different therapy. But ultimately the insert is an aid in deciphering the Food and Drug Administration’s (FDA) “buyer beware” program.
Consumer safety is FDA’s number one priority. It accomplishes this by ensuring that a company’s drug safety programs are compliant with federal regulations. A drug company cannot claim its product can diagnose, treat, cure, mitigate, or prevent a disease without FDA approval. Companies pay steep consequences for violating FDA regulations. In 2004 the big British pharmaceutical company GlaxoSmithKline (GSK) pled guilty for its inappropriate marketing of its antidepressant medication Paxil. GSK crossed legal lines with not one but two executive agencies: FDA and its sister agency, the Federal Trade Commission. GSK paid a hefty $3 billion fine for the fraudulent marketing and off-label use of Paxil.
FDA begins defining safety with preclinical toxicology studies. FDA requires that drug company scientists begin safety tests in small rodents. If they find that the drug-dosing scheme chosen is safe, then they move to larger animal models like canines and nonhuman primates. These size-escalating animal studies are called single-dose, or multi-dose, escalating studies, in which the number of doses and drug concentrations are evaluated for safety. If safety is established in animal models, then FDA scientists begin testing the drug in humans in three stages of clinical trials. The toxicology questions include: What drug concentration is lethal? For small molecules, what organs are affected by the drug? What metabolites are present after drug metabolism? For large molecules, does the animal elicit an immune response against the drug? What is the antidrug antibody response doing to the drug? Is it allowing the drug to reach its target or is it neutralizing it? Because of FDA’s strict regulations, all of those questions, and more, are answered in the forms of statistical tables and various data in the appropriate sections of each insert, and they are imperative to your safety.
So the next time the pharmacist fills your prescription, you should read the insert and be sure to visit the National Library of Medicine’s mobile resources for more information on medications. It hosts valuable safety and efficacy data for your drug. And don’t forget there’s a whole behind-the-scenes group of scientists working hard to answer FDA’s drug safety and efficacy questions.
As the elevator doors opened, my elevator friend thanked me and sneezed as he left for the pharmacy.