The biopharma industry is failing to adopt innovative approaches to clinical trials, relying on outdated approaches that could result in promising therapies being abandoned on cost grounds.
That is according to outgoing FDA commissioner Scott Gottlieb, who says in a statement that there is “a continued reluctance to adopt innovative approaches among sponsors and clinical research organisations.”
The industry seems wedded to “legacy business models that discourage collaboration and data sharing,” he asserts, warning that unless changes are made to streamline the clinical trials process “ important therapeutic opportunities may be delayed or discarded because we can’t afford to run trials needed to validate them.”
Gottlieb – who is leaving the FDA next month and will be replaced on an interim basis by National Cancer Institute (NCI) director Ned Sharpless – says that the advent in precision medicine, with efficacy signals being tested in small groups of patients based on biomarkers, could mean earlier access to important new medicines. However, that will need more efficient approaches to testing and developing diagnostics and therapeutics.
The comments comes as the FDA has published a series of guidance documents aimed at modernising clinical trials.
The first covers enrichment strategies for clinical trials that could be used to make sure that enrolled subjects are best suited to giving an accurate readout on efficacy and safety, for example based on clinical laboratory, genomic, and proteomic factors, which could reduce the scale and duration of trials.
“The information gleaned from these approaches can also guide clinical practice, to help doctors get the right treatment to the right patient at the right time,” according to Gottlieb.
It covers reducing variability in subjects and identifying of high-risk and more responsive patients, and includes study design models including an example for adaptive enrichment from the I-SPY 2 study of neoadjuvant and personalised drugs for breast cancer.
The second guidance is a question and answer document on risk-based monitoring (RBM) of clinical investigations that complements 2013 RBM guidance, adding in recommendations for planning a monitoring approach, developing the content of monitoring plans, and addressing and communicating monitoring results.
Earlier this week the FDA also released guidance on how they can incorporate patients with more challenging health conditions into oncology clinical trials, to make the studies more representative of real world cancer care. That includes patients with brain metastases or previous malignancies, those with organ dysfunctions, and adolescent and paediatric patients.
“We look forward to working with our many stakeholders to support the development of these modern approaches and make the promise of precision medicine a reality for more patients,” concludes Gottlieb.