Final Guidance on Smallpox Development Using the Animal Rule

In preparation for a potential smallpox outbreak or bioterrorism attack, a final FDA guidance provides recommendations for testing vaccines in animals.

Under the FDA’s Animal Rule, drugmakers can test live viruses on animals in lieu of endangering human subjects. The FDA guidance requires data from at least two lethal animal studies to show efficacy of the vaccine because “no single animal model is known to be the best predictor of human responses to smallpox.”

The guidance directs sponsors conducting such animal studies to prepare designs for one or more clinical trials in the event of a public emergency involving smallpox.

“The design of these animal studies should be based on the general principles of human clinical trial design,” says the guidance.

Google Data Partnership Under HHS Scrutiny

A data-sharing partnership between Google and a 21-state private health network is under scrutiny by the Department of Health and Human Services over whether the effort is HIPAA-compliant.

Launched last week, Google’s Project Nightingale gives the tech giant the ability to analyze the personal health information from the Ascension Catholic hospital system, using artificial intelligence (AI) tools to help Ascension devise personalized medical treatments.

The hospital system claims that Google’s G-Suite is HIPAA-compliant and builds on its robust data and security protection protocols. Google said that it would answer HHS’s questions as part of the probe but believes that it has maintained HIPAA compliance.

Mayo Clinic entered into a similar agreement with Google earlier this year to store its data in their cloud and use its AI toolset to analyze the information. Mayo Clinic, however, specified that the information held by Google would be de-identified to remove any danger of individual patients being recognized.

IMDRF Updates Guidelines for Clinical Investigations of New Devices

Three updated international guidelines on medical devices released last week define what clinical evidence is needed for marketing approval of a new device.

In an effort to keep current with industry developments, the International Medical Device Regulators Forum’s (IMDRF) guidelines for investigational devices define when a devicemaker must conduct a clinical trial and when it may rely on existing evidence gathered from published literature.

Devicemakers must conduct their own clinical trials when they cannot find evidence of safety and effectiveness in previously conducted trials, according to the guideline on clinical investigations.

The guideline on clinical evaluation specifies that “data relevant to the clinical evaluation may be held by the manufacturer or a third party, or be available in the scientific literature, for the device in question or for comparable devices.” The guideline also says that real-world evidence is acceptable.

The third guideline contains terms and definitions related to clinical evaluation and clinical investigation.

Read the clinical investigation guideline here: https://bit.ly/34WCDxi.

Read the clinical evaluation guideline here: https://bit.ly/371g1xp.

Read the definitions guideline here: https://bit.ly/2XdZTEs.

Study Points to a Decade of Underreporting Clinical Trials

Trial results entered in Clinicaltrials.gov in the past decade show a poor quality of reporting from both industry sponsors and academic medical centers, according to a new report published in the New England Journal of Medicine.

Of the 36,000 trials completed since the database launched in September 2008, only 66 percent, have complied with a federal mandate to report final data within one year of completion, researchers found.

Industry-sponsored trials showed a higher rate of compliance — 77 percent reported — than nonindustry trials, only 63 percent of which have reported their results.

“We have observed that industry sponsors tend to be well-staffed and have a centralized process for supporting the submission of results, whereas nonindustry sponsors tend to rely on individual investigators with minimal centralized support,” says the report.

Trial transparency advocate TranspariMED also found in a March 2019 study that one-third of universities failed to report their trials within the one-year time limit. Similar problems exist in European trials, TranspariMED noted in an April 2019 study. Only about half of trials had complied with the mandate to report results to the EU Clinical Trials Register (CenterWatch Weekly, May 6, 2019).

Authors of the ClinicalTrials.gov study concluded that contributing data throughout a trial using electronic data capture and other digital tools would improve the reporting rate and quality of the data.

The study was conducted with the support of the Intramural Research Program of the National Library of Medicine, which administers ClinicalTrials.gov.

Adverse Event Brings Solid Biosciences DMD Gene Therapy Trial to Halt

Another Duchenne muscular dystrophy gene therapy trial has been held up by the FDA following a serious adverse event in one patient. The agency halted Solid Biosciences’ trial of SGT-001 twice before due to reported serious adverse events.

The patient is in recovery and the five other patients treated in the study are “all doing well,” the company said.

Pfizer and Sarepta Therapeutics are also developing DMD gene therapies. Pfizer reported similar serious adverse events in a single patient in June but the other five patients in the trial showed immune responses as expected.

Sarepta has not reported any serious adverse events for its DMD gene therapy. In August, the firm contested an FDA adverse event reporting system (FAERS) submission on a patient enrolled in its microdystrophin trial for DMD (CenterWatch Weekly, Aug. 12, 2019). The company called the submission “erroneous,” and the study’s drug safety monitoring board recommended that the trial continue.

CenterWatch Reveals Sponsor and CRO Results from 2019 Global Site Survey

Well-trained CRAs are the most important aspect of a site’s relationship with a sponsor or CRO, according to CenterWatch’s 2019 Global Site Relationship Benchmark Survey.

More than 4,000 sites responded to the survey this year, rating the most desirable traits in a sponsor or CRO. Most sites, 66 percent, said they value professional and knowledgeable CRAs. Attributes also appearing in the top 10 include organization and preparation, accessible staff, timely drug availability, and open communication.

The survey shows sites’ dissatisfaction with sponsors’ handling of protocols, with only about 40 percent of sponsors receiving high marks in this area. CROs handling of budgets and payments also was scored low, with only about 37 percent receiving high marks.

Click here to purchase a copy of the report: https://bit.ly/32N1Ada.

Three Observational Studies to Use Apple Watch and iPhone

Three new observational studies will test the ability of the Apple Watch and iPhone to provide warning signals for irregular heartbeat and other physiological symptoms that may indicate the need for medical intervention.

Apple will partner with the National Institutes of Health, the American Heart Association and several academic research centers to gather data that will help develop algorithms for predicting certain irregularities.

The Apple Heart and Movement Study aims to enroll 500,000 people over the next five years. The Apple Women’s Health Study wants 1 million people to sign up over the next 10 years, and subjects will be asked to track their menstrual cycle and respond to regular surveys about it. And the Apple Hearing Study aims to enroll 150,000 people over the next two years with subjects completing hearing tests while their phones detect and measure loud noises in their environment.

Participants, who must be over age 18, will be able to control which types of information they share and can delete data within 24 hours of its collection. Apple has said it will not sell data collected from the studies.

Apple’s first foray into observational research was a heart study with the Stanford Medicine system completed last week that used the Apple Watch to monitor 419,000 participants’ heart rates and provided ECG patches to the .5 percent of participants whose watches notified them of irregularities.

Alzheimer’s Biomarker Repository Provides Data and Tools for Clinical Trials

The nonprofit UsAgainstAlzheimer’s has launched the first searchable database for Alzheimer’s biomarkers.

The database will contain information on amyloids, brain iron levels, cell signaling, genetic variations from DNA, eye movement, inflammation, neuronal damage, Tau, oxidative stress and vasculature to measure the onset of the disease.

Intended for use by researchers, policymakers, patients and caregivers, the database can be edited and updated by qualified researchers and scientists.

New Trial Will Test PTSD-Specific Therapies

A new trial funded by the U.S. Army will test what could become the first treatments developed specifically for posttraumatic stress disorder (PTSD).

The adaptive clinical trial, which will be conducted by Cohen Veterans Bioscience (CVB), will use precision gene therapy to study how the disorder affects brain physiology.

The three-and-a-half-year trial will start in the fall of 2020, with the goal of advancing to phase 3 of testing by 2022.

The trial will be overseen by a committee representing the Veterans Health Administration, the National Institute of Mental Health, the National Institute of Alcohol Abuse and Alcoholism, the FDA and the Defense Health Agency’s Psychological Health Center of Excellence.

WCG Acquires Waife & Associates

WCG Clinical has acquired clinical trial management consultancy Waife & Associates (W&A).

W&A has provided management consulting services for biopharmaceutical clinical research companies for more than 25 years, serving more than 200 clients worldwide. W&A’s founder Ronald S. Waife will become WCG’s chief management consulting officer.