"Pharmaceutical and biotech companies, large and small, established and early stage, are forging strategic alliances, collaborative partnerships, and multi-company consortia," said Tufts CSDD director Kenneth I. Kaitin. "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."
Mr. Kaitin noted that the drug development model has not fundamentally changed in the past 50 years, when the Kefauver-Harris Amendments of 1962 established the current standard for the clinical testing of investigational drugs. He commented, "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."
According to the report, increasingly 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 candidate success. Also, adoption of adaptive clinical trial designs will accelerate as cross-functional teams within sponsor companies look to increase program success rates while lowering costs and disruptions from protocol amendments. Additionally, the report states that the FDA will make greater use of patient-reported outcomes to support labeling claims in drug applications and increased use of social media to communicate with patients and caregivers.
The report also mentions that sustaining the recent pace of investments in new biotech companies will be a major challenge in 2014, fostering more alternative financing approaches via industry based venture capital groups and patient support foundations. More approvals of co-developed companion diagnostics and therapeutics are likely, the report noted, but diagnostics without evidence of positive impact on health outcomes will continue to face reimbursement challenges.