FDA Advises on Phase III Trials for NASH with Symptom-Free Cirrhosis

June 10, 2019

The FDA last week issued a draft guidance on Phase III trials of treatments for nonalcoholic steatohepatitis (NASH) with symptom-free cirrhosis, including recommendations on enrollment criteria, trial design, efficacy endpoints and safety considerations.

The draft guidance says sponsors should enroll only patients whose cirrhosis is secondary to NASH, using histological criteria. The agency also will accept non-histological criteria if they are scientifically supported.

Sponsors should consider using endpoints such as complication of ascites, variceal hemorrhage, hepatic encephalopathy, worsening of condition to the point of requiring liver transplant or death from any cause.

In addition, the trial protocol should specify criteria for excluding patients with decompensated cirrhosis — where the cirrhosis has progressed and the liver is no longer fully functioning. Sponsors should not enroll patients listed for liver transplantation, the agency says.

Read the draft guidance here:

NIH Lays Out $42M for Genomic Medicine Trials
The NIH will pay up to $42 million over the next five years for clinical trials on whether genomic medicine can help people with chronic diseases manage their care better.

The trials, scheduled to begin next year, represent the second phase of the agency’s Implementing Genomics in Practice (IGNITE) Network. The first trial will examine whether early access to patients’ genomic data will help them manage high blood pressure, hypertension and kidney disease.

The second trial will focus on pain and depression. It “seeks to test whether patients with acute post-surgical pain, chronic pain and depression have better clinical outcomes if pharmacogenomics guide opioid and antidepressant prescriptions.”

The trials will be held at sites at the Universities of Florida and Indiana, Duke, Vanderbilt and the Icahn School of Medicine at Mount Sinai.

New Data Tool Available to Medical Device Sponsors
Medical device sponsors now can access a data-driven benchmarking program to help accelerate study start-up.

WCG’s KMR Group has opened its Site Contracts Program to medical device companies for the first time, the company announced. Already in use by biopharma companies, the program has helped reduce trial start-up time by up to 20 percent and decrease costs by 15 percent.

“This program will give device companies a rock-solid foundation of proprietary, independently verified data on which to build their study plans and implement performance improvements, as well as unique evidence-based insights to guide their decision-making,” says KMR president and founder Linda Martin.

England Claims Record Numbers in Clinical Research
Participation in clinical trials in England continues to grow at a record pace, according to a new report from the country’s National Institute for Health Research (NIHR).

More than 870,000 patients were enrolled in English clinical research studies in the past 12 months, an increase of 140,000 over the previous 12-month period. Nearly 2,400 subjects per day were involved in a clinical trial, NIHR says.

And participation rates continue to grow year by year. The number of participants recruited for NIHR-supported studies annually has hit record levels for the past three years, with the 2018-2019 growth rate coming in at the highest ever, 20 percent.

Most patients this year were enrolled in children’s studies (81,892), followed by studies in primary care settings (78,533), reproductive health and childbirth (74,128), cancer (67,652) and mental health (65,645), the NIHR says.

Emerging Biotechs Lead the Way in 2018, Report Says
The number of active clinical-stage programs reached a record 6,984 in 2018, the Biotechnology Innovation Organization (BIO) says in a new report, with emerging biotech companies accounting for 73% of these programs.

BIO found that 45 percent of emerging companies partnered with other companies to pursue their pipelines. Half of oncology studies are being conducted by such partnerships, and 36 percent of cardiovascular and infectious disease studies are partnered.

The U.S. leads the world in the number of emerging companies entering the pipeline, with 44 percent, compared to 17 percent in Europe and 8 percent in Asia.

Read the report here:

Study Finds Lack of Faith in Randomized Testing
Evidence-based research faces a challenge, according to one study that indicates a negative public view of comparison testing.

A study conducted by researchers at the Geisinger Health System’s Center for Translational Bioethics and Healthcare Policy found evidence that A/B tests comparing the effectiveness of two treatments, practices or policies often are criticized as inappropriate despite people finding the untested implementation of A or B to be appropriate.

Researchers examined 16 studies from sectors ranging from healthcare to public policy that involved 5,873 participants from three populations. Even when each of the options being compared are themselves unobjectionable, participants expressed disapproval of testing to prove superiority of one over the other.

Although participants’ reactions may be largely due to unfamiliarity with scientific methods, the study says, negative public perception may tend to lead researchers and policymakers away from randomized testing and may make it more difficult to recruit subjects.

Thorough evaluation of treatments through randomized trials could cause greater opposition than simply implementing them without proper testing, the researchers conclude.

Read the report at:

Evidera to Acquire RWE Company Medimix
Evidera, a business unit of PPD, will acquire real-world evidence data company Medimix International, the company announced last month.

The Medimix database, which includes 2.2 million clinicians in more than 60 countries, will support Evidera’s mission to provide evidence-based solutions that demonstrate the effectiveness, safety and value of healthcare products.