The Most Transformative Drugs of the Past 25 Years

A Survey of Physicians

What constitutes an innovative drug? These authors conducted a study on transformative drug development, involving 184 expert U.S. physicians across 15 medical specialties to help answer this question.

Despite emphasis on the development of new drugs, there isn’t consensus on the definition of an innovative drug. To help clarify what constitutes an innovative drug, the authors conducted a study on transformative drug development. The effectiveness of the drug was cited as a justification for identifying a transformative drug.

The survey involved 184 expert physicians across 15 medical specialties based at 30 leading U.S. academic medical centers. Using Delphi process, the participants worked toward consensus over several rounds on what drugs have been most transformative over the past 25 years.

The list of drugs used for the survey was compiled from a list of new molecular entities and new biologic drugs approved by the FDA between January 1985 and December 2009. After refinement, a total of 434 drugs were included in the survey.

Key Findings:

  • The most common justification used by study participants to indicate a drug as transformative was “improved efficacy,” with 55 percent of participants agreeing.
  • “Novel mechanism of action” was cited by 37 percent of participants, followed by “impact on practice in field” (24%), and “scientific merit” (15%).
  • A disproportionate number of transformative products were designated as “orphan drugs” for patients with rare diseases, showing the drug’s impact beyond its specific population.

Finally, the article lists the top transformative drug or drug classes identified for each clinical field. Limitations of the study included the inability of survey participants to interact interpersonally and a design that was limited to U.S. physicians.

"Our results suggest that when considering the value of newly approved drugs, physicians from many fields appear to hold the greatest regard for highly efficacious new drug classes that address previously unmet clinical needs."