House Republicans Pass Right-to-Try Bill

The House passed its version of federal right-to-try legislation, sending it for reconsideration by the Senate, which passed a similar bill last August. The bill, H.R. 5247, had failed to garner enough votes on the floor last week to fast-track the bill under suspension of the House rules. Republicans that favor the legislation said they believe terminally ill patients, that have exhausted all other treatment options, should be able to try unproven therapies as a last resort. “For those patients caught between the traditional drug approval delays, a clinical trial process for which they do not qualify, and limited time, this right-to-try establishes the freedom for patients to try therapies in situations where the benefits far outweigh the risks,” said bill sponsor Rep. Brian Fitzpatrick (R-Pa.). Most Democrats opposed the bill, arguing that it would hinder the FDA’s oversight of investigational treatments — and contending that patients already have a program through which they can request access to experimental treatments. In addition, four former FDA commissioners — Robert Califf, Margaret Hamburg, Mark McClellan and Andrew von Eschenbach — as well as over 80 patient advocacy organizations opposed the bill because it would remove agency protections from the process.

FDA, EMA & the National Kidney Foundation Hold Workshop on CKD Meta-analysis

The National Kidney Foundation, the FDA and the EMA held a joint scientific workshop to review a multi-year meta-analysis examining an enormous compilation of data on chronic kidney disease — including data on nearly two million participants — to support the use of earlier markers of kidney disease progression as clinical trial endpoints for early stages of CKD. The analysis explored whether surrogate biomarkers measuring kidney damage and function — such as changes in albuminuria or changes in glomerular filtration rate — can be used as predictors of a treatment’s effect on progression to kidney failure. “This extensive meta-analysis of endpoints for chronic kidney disease builds upon previous research which recommended a 30 or 40 percent decline in GFR as the endpoint for clinical trials in some CKD populations,” said Kerry Willis, chief scientific officer for the National Kidney Foundation. “However, we’ve found that these recommended endpoints are less applicable to the clinical development of drugs targeted at earlier stages of kidney disease and for many drugs with possible hemodynamic effects...How to overcome these obstacles was the driving force behind today’s scientific workshop and this multi-year research project.” The research was conducted at Tufts Medical Center, Johns Hopkins University, the University of Utah and Groningen University in the Netherlands. The groups plan to publish the meta-analysis and workshop recommendations in a series of articles later this year.

Oncology Accelerated Approvals Showed Benefit

Most oncology treatments that received the FDA’s accelerated approval pathway in the past 25 years later demonstrated benefits in clinical trials, according to a review by agency officials published in JAMA Oncology. Between the program’s creation in 1992 and May 2017, the FDA granted 64 accelerated approvals for cancer treatments, including for 53 new molecular entities and in 93 new indications. Seventy-two percent of these indications were studied in single-arm clinical trials, 87 percent of which used response rate as their endpoint. For drugs with completed postmarket requirements, the most common study endpoint was progression-free survival and time to progression. Of the 93 indications, 51 fulfilled their postmarket requirements and verified clinical benefit in a median of 3.4 years after the initial designation, while another 37 have not yet completed confirmatory trials or verified benefit. The remaining five have been withdrawn from the market.The article is available here: www.jamanetwork.com/journals/jamaoncology/article-abstract/2673837.

Oncology Will Again Be The Top Clinical Trials Sector in 2018

Oncology will continue to be the top sector for clinical trials planned to be launched this year, according to the analytics firm GlobalData. Novartis and GlaxoSmithKline were listed as the top two industry sponsors, while MD Anderson Cancer Center led among non-industry sponsors. Immuno-oncology drugs were the most frequent. The largest number of clinical trials were planned for Merck’s Keytruda (pembrolizumab), with Bristol-Myers Squibb’s Opdivo (nivolumab) taking second. Both target PD-1. Across all diseases and phases, phase II trials account for 44 percent, according to the report. Phase I and III trials made up 26 percent and 21 percent, respectively. Postmarket phase IV trials meanwhile accounted for just 8 percent. Among sites, North America was ranked as the top location, followed by Europe and Asia-Pacific, with all three regions accounting for more than 90 percent of trial locations. “The dominance of industry sponsorship is most prominent in phase I, II and III trials, whereas non-industry sponsorship is most prominent in phase IV trials,” said Revati Tatake, global director of databases and analytics at GlobalData, adding that about two-thirds of trials are sponsored by pharmaceutical companies.