While many drug developers continue to improve clinical trial operational efficiency to increase the pace of new product introductions, their medium- and long-term success increasingly will depend on their ability to embrace more efficient R&D models, according to the Tufts Center for the Study of Drug Development.

“Pharmaceutical and biotech companies—large and small, established and early stage—are forging strategic alliances, collaborative partnerships and multi-company consortia,” said Kenneth I. Kaitin, Tufts CSDD director. “Early results indicate that sharing knowledge and leveraging resources is helping sponsors find new drugs to treat many of today’s most challenging and complex indications.”

Noting that the drug development model has not fundamentally changed in more than 50 years, when the Kefauver-Harris Amendments of 1962 established the current standard for the clinical testing of investigational drugs, Kaitin said, “Perhaps the greatest gain from clinical design improvements and new partnership models will be the development of industry best practices, which will enable companies to maximize their formidable R&D investment and help ensure future commercial success.”

Kaitin made his comments in connection with the release of the Tufts CSDD Outlook 2014 report on pharmaceutical and biopharmaceutical trends. Report highlights include:

• Growing concern over expensive, late-stage clinical development failures will lead firms to reassess their use of meta-analyses and subgroup analysis and make more realistic assessments about the likelihood of candidate success.
• Adoption of adaptive clinical trial designs will accelerate, particularly in earlier clinical phases, as cross-functional teams within sponsor companies look to increase program success rates while lowering costs and disruptions from protocol amendments.
• The FDA will foster greater use of patient-reported outcomes to support labeling claims in drug applications and make greater use of social media and the Internet to communicate with patients, caregivers and patient advocates.
• Sustaining the recent pace of investments in new biotech companies will be a major challenge in 2014, spurring further development of alternative financing approaches, e.g., industry based venture capital groups and patient support foundations.
• More approvals of co-developed companion diagnostics and therapeutics are likely, but diagnostics without evidence of positive impact on health outcomes will continue to face reimbursement challenges.