Background

The authors estimate the probability of successful development and duration of clinical trials for medications to treat neuropathic and nociceptive pain. The authors also consider the effect of the perceived abuse potential of the medication on these variables.

Methods

This study uses the Citeline database to compute the probabilities of success, duration, and survivorship of pain medication development programs between January 1, 2000, and June 30, 2020, conditioned on the phase, type of pain (nociceptive vs. neuropathic), and the abuse potential of the medication.

Results

The overall probability of successful development of all pain medications from phase 1 to approval is 10.4% (standard error, 1.5%). Medications to treat nociceptive and neuropathic pain have a probability of successful development of 13.3% (standard error, 2.3%) and 7.1% (standard error, 1.9%), respectively. The probability of successful development of medications with high abuse potential and low abuse potential are 27.8% (standard error, 4.6%) and 4.7% (standard error, 1.2%), respectively. The most common period for attrition is between phase 3 and approval.

Conclusions

The authors’ data suggest that the unique attributes of pain medications, such as their abuse potential and intended pathology, can influence the probability of successful development and duration of development.

Editor’s Perspective
What We Already Know about This Topic
  • Despite the prevalence and societal costs of pain in the United States, investment in pain medication development is low, due in part to poor understanding of the probability of successful development of such medications
What This Article Tells Us That Is New
  • This study examined outcomes and parameters of 469 pain pharmaceutical development programs of 399 unique active pharmaceutical ingredients between 2000 and 2020
  • Development of new medications with high abuse potential decreased since the peak of the opioid epidemic, while development programs for low abuse potential medications increased
  • The probability of successful development programs was 27.8% for high abuse potential compounds and 4.7% for low abuse potential compounds
  • The probability of successful development of a treatment for nociceptive pain was 13.3%, and that for a treatment of neuropathic pain was 7.1%
  • Development of pain medications in large phase 3 safety and efficacy trials took an average of 30 months