Rate of US drug approvals lower than previously reported


Overall success rate is only 1 in 10 rather than the 1 in 5 cited in earlier reports, says new study

The overall success rate for drugs moving through clinical trials to FDA approval from late 2003 to the end of 2010 is around one in 10, according to a study carried out by Biotechnology Industry Organisation (BIO) Industry Analysis and research service provider BioMedTracker (BMT). Previous reports, taken from earlier years, showed the rate of drug approvals is one in five to one in six.

The study builds on the findings from previous studies and uses a broader, deeper and larger sample than previous reviews of clinical trials and approvals data using the BioMedTracker proprietary database of 4,500 drugs and more than 8,000 unique development paths. Using clinical trial data from the past seven years, the analysis examines the most recent probability of success by treatment type, phase of development and therapeutic area.

‘This groundbreaking study highlights the depth and breadth of risk inherent in the drug development process more comprehensively than any other previous study,’ said Alan Eisenberg, executive vice president of emerging companies and business development at BIO. ‘Knowing more about the magnitude of risk can lead to smarter drug development as well as smarter investing.’

Key findings from the study include:

  • Overall success rates from Phase I to FDA approval is nearly 9%. This number is comprised of lead and secondary indications. When separated, lead indications have close to a one in seven rate of approval and secondary indications have a rate of one in 30.
  • Clinical trials that address secondary indications for drugs tend to be far less successful on average. This was seen in all phases of clinical development as well as in all disease areas.
  • Large molecule drugs are twice as successful in gaining approval than small molecule drugs.

The BMT/BIO study examines the clinical phase status of these development paths as of year-end 2003 through year-end 2010, which accounts for more than 59,000 data points. The study determines the percentage of drugs that advance to the next phase of development versus those that are suspended and therefore the likelihood of a drug ultimately being approved by the FDA. The data spans all companies conducting development on therapeutics for approval in the US.

The BMT/BIO analysis includes breakdowns by indication, disease group, size of company, molecule type, route of administration, nme versus biologic versus non-nme. The data encompasses the most recent data available during a time in which FDA requirements for approval have been in flux. Included in the analysis is an in-depth look at FDA decisions and approval rates by FDA review number.

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‘Having an up-to-date and detailed picture of clinical success rates, and ultimately how likely a drug is to be approved, is vital to our clients when making investment and business decisions. We believe that this study provides the market with an accurate and comprehensive picture of the relative difficulty of achieving product approval in the US,’ said Michael Hay, senior biotechnology analyst at BMT. ‘Strikingly, oncology drugs have the toughest time making their way through the clinic, despite cancer being the most closely studied area in drug development.’