In implementing another provision of the 21st Century Cures Act, the FDA is proposing a rule to exempt minimal-risk clinical investigations from informed consent requirements. Under the proposed rule, institutional review boards could waive or alter certain informed consent elements or waive informed consent altogether when a clinical investigation poses no more than minimal risk to participants and includes appropriate safeguards to protect their rights, safety and welfare, according to a notice to be published in Thursday's Federal Register. Currently, the FDA allows such exceptions only in life-threatening situations when certain conditions are met or for certain emergency research. The revisions will align the FDA's policy with that of the Common Rule, which has allowed informed consent waivers for minimal-risk research supported by other federal agencies since 1991. Comments on the rule are due by Jan. 14.
The FDA slapped Stemgenex Biologic Laboratories LLC, of San Diego, with a warning letter about marketing an unapproved stem cell product and for deviations from current good manufacturing practice (GMP) requirements that could lead to microbial contamination. During a January inspection, an FDA investigator found that Stemgenex was processing body fat into stromal vascular fraction (SVF) to administer intravenously, by inhalation and directly into the spinal canal. Although the company had never submitted an investigational drug or a biologic license application for the SVF, it marketed the product as a treatment for Alzheimer's disease, Crohn's disease, diabetes, fibromyalgia, spinal cord injury, chronic obstructive pulmonary disease, multiple sclerosis, muscular dystrophy, Parkinson's disease, peripheral neuropathy and rheumatoid arthritis. The warning letter cited the company for 11 GMP violations, including failure to create a quality control unit, addition of a nonsterile substance to batches of its SVF product intended for higher risk routes of administration, failure to test each batch for objectionable microorganisms and lack of an environmental monitoring system in an aseptic processing area. Commenting on the warning letter, FDA Commissioner Scott Gottlieb noted the potential health benefits of regenerative medicine that have spurred progress in stem cell biology. "But we continue to see bad actors exploit the scientific promise of this field to mislead vulnerable patients into believing they're being given safe, effective treatments, when instead these stem cell producers are leveraging the field's hype to push unapproved, unproven, illegal and potentially unsafe products," he said. Besides putting patients' health at risk, such actions threaten "the long-term viability of the industry and of effective products," Gottlieb added.
The FDA is issuing a draft guidance on the use of metastasis-free survival (MFS) as an endpoint in clinical trials evaluating drugs to treat nonmetastatic, castration-resistant prostate cancer (nmCRPC). Due to the prolonged assessment period with low event rates common to nmCRPC, overall survival can be impractical as a primary endpoint for clinical trials. At a meeting in 2011, the FDA's Oncologic Drugs Advisory Committee noted that the transition from nmCRPC to radiographically detectable metastatic disease is a clinically relevant event that can be associated with morbidity and the need for additional medical interventions, whereas local progression events may never progress to distant disease or lead to systemic morbidity. "Thus, a large magnitude of treatment effect on MFS with an acceptable safety profile could be used to demonstrate clinical benefit and support product approval," the FDA said. Comments on the draft guidance are due by Jan. 13.
Citing the multitude of endpoints being assessed in clinical trials for cancer drugs, the FDA is proposing a study to determine if a disclosure on promotional materials targeting doctors would provide a truer perspective on trial data included in the materials. "In addition to the endpoints upon which FDA approval may be based, pharmaceutical companies typically assess many other endpoints to further explore the effects of their products," according to a notice slated for publication in Wednesday's Federal Register. Promotional materials for doctors sometimes "contain carefully extracted data from clinical studies that, taken out of context, can exaggerate the benefits of a drug or contribute to physicians prescribing the drug for an inappropriate patient population," the FDA said. The study the agency proposes would look at whether adding a statement such as "This presentation includes exploratory information of uncertain clinical utility and should be interpreted cautiously when used to make treatment decisions" could be used to convey the limitations of the data given in the promotional material. Comments on the proposed study should be submitted by Dec. 14.
The EMA is replacing its 2007 guideline on conducting pharmacovigilance for drugs used by children with a new good pharmacovigilance practice (GVP) chapter on specific considerations for the pediatric population. The chapter covers drugs with an approved pediatric indication or that are in pediatric development, as well as those approved for adults that are used off-label to treat children. In addition to discussing off-label use and medication errors, the GVP chapter contains pediatric-specific guidance on all major pharmacovigilance tools and processes, including risk management plans, periodic safety update reports, post-authorization safety studies, signal management and safety communication, according to the EMA. It also discusses the need to include information such as the child's age, weight and height in adverse drug reaction reports, along with the indication and the strength, dose and pharmaceutical form of the drug.