Clinical Trials

Clinical Trials

An SPH team works to reduce the dangers inherent in clinical trials.

To the extent that our lives depend on drugs like Lipitor and on procedures like MRIs, they also depend on clinical trials—the experiments by which scientists ultimately prove the safety and efficacy of medications and treatments. To the human beings who participate in them, however, clinical trials pose risks.

Cathie SpinoAs an assistant professor at Harvard in the early 1990s, School of Public Health biostatistician Cathie Spino was part of the team that worked on the trials for AZT—the first drug to help AIDS patients survive—and she later spent ten years as a statistician doing clinical trials for Pfizer. “When you know that you could make a difference in a patient’s life,” she says, “that’s to me where it’s at.”

But the dangers are real—especially in Phase 1 clinical trials, where an experimental drug or treatment is tested for the first time in humans to evaluate its safety and dose range. (Phase II trials assess a drug’s effectiveness and yield further safety information, and Phase III trials typically compare a drug’s results with standard treatments or placebo to confirm effectiveness and monitor side effects.)

As director of the SPH Biometrics and Outcomes Research Core (BORC), Spino helps oversee multisite clinical trials for two national disease networks as well as a number of single-site trials. To guarantee the greatest possible safety in each trial it oversees, BORC employs a small army of statisticians, data-entry specialists, project managers, study monitors, programmers, database administrators, and trained medical personnel, who collectively help design and monitor studies, gather and analyze data, and summarize findings. They also work to ensure that trial data are confidential, data connections secure, randomization is properly administered, and the requisite regulatory documents are obtained.

“When you’re doing a multicenter clinical trial, it becomes really important that you’ve got a really good protocol and really good standards,” Spino says. Among the trials BORC is monitoring are a comparison study of surgical techniques to treat incontinence and a study of the long-term outcomes for children with biliary atresia who’ve had liver transplants. Spino hopes to expand BORC’s work to include studies in women’s health, pediatrics, and mental health.

“One of the reasons I love working in clinical trials is because I think it’s a great mix of statistical theory and practical application,” she says. “And at the end of the day, you answer questions that can help people.”

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factCHECK, 2008

  • It usually takes 12–15 years for a new compound to be approved as a medicine in the United States
  • On average, it costs $800 million for a pharmaceutical company to develop a new medicine
  • Typically, a pharmaceutical company recoups its development costs for only three of ten medicines
  • Before 1938, manufacturers could market a drug without submitting any information to the FDA or any other agency; the Federal Food, Drug and Cosmetic Act (FD&C Act) of 1938 was passed when over 100 children died from taking a sulfa drug that had not been tested in people