FDA Streamlines Application for Oncology Trials with IVDs

A new final guidance from the FDA allows some sponsors of oncology trials that use an investigational in vitro diagnostic (IVD) devices to file a single investigational application rather than filing for the drug and device components separately.

Under the new guidance, sponsors of trials combining a new oncology drug with a new in vitro diagnostic (IVD) device will submit an investigational new drug application to the Center for Drug Evaluation and Research (CBER) or the Center for Biologics Evaluation and Research (CBER), including information about the investigational IVD in question.

CDER or CBER then will consult with the Center for Devices and Radiologic Health (CDRH) to determine whether the IVD presents a “significant risk” and requires the sponsor to submit to CDRH a separate application for investigational device exemption.

The guidance applies only to new combination product IND applications and does not address IND-exempt trials.

Read the final guidance here: https://bit.ly/324y7LT.

EU Device Rule Sets New Trial Requirements

Devicemakers planning to market products in the European Union need to understand new EU rules that spell out when clinical research is required and impose requirements for conducting trials.

The clinical research provisions of the EU Medical Device Regulation (EUMDR) will take effect in May 2020 and will require devices that are Class III (high risk) or implanted in a patient’s body to be proven safe and effective in a clinical trial.

EUMDR is more complex than the Medical Device Directives (MDD) previously in effect in the EU, says device quality systems expert Dan O’Leary of Ombu Enterprises LLC.

The process for applying to conduct a trial also will be more involved, O’Leary says.

The first step is to determine whether the device falls under one of several exceptions to the clinical investigation requirement, including devices that are a modification of a device already marketed by the same manufacturer that has been demonstrated to be equivalent to the original.

Clinical equivalence also can exempt a device from the trial obligation if it is used for the same clinical condition or purpose — including severity and stage of disease — at the same site in the body and in a similar population based on age, anatomy or physiology.

An additional element of the informed consent process under EUMDR requires sponsors to explain to the patient possible treatment alternatives, including follow-up measures if participation is discontinued.

Although devicemakers that currently are approved to conduct a trial under MDD will be grandfathered in under EUMDR, O’Leary warns sponsors to stay on top of the changes.

Even grandfathered trials should make sure all their documentation lines up with the new requirements, he says. “So that even though you started under the MDD, … you have the MDR requirements for the design dossier content covered, because you don’t want to get surprised at the end.”

FDA Grants Funding for Rare Disease Trials

The FDA has selected 12 organizations to share a $15 million pool of funding for rare disease trials.

The grants, which will be dispersed over a four-year period, will fund research ranging from trials of rare disease drugs for children and cancer patients to immunodeficiency treatment studies.

Of the 12 grantees, four are pharma companies, including Cumberland Pharmaceuticals, which will study therapies for Duchenne muscular dystrophy.

Cincinnati Children’s Hospital Medical Center received grants for two separate oncology trials, and Massachusetts General Hospital will study treatments for HPV.

Academic medical centers receiving grants include the University of Texas MD Anderson Cancer Center to study leukemia, the University of California, San Diego for gastrointestinal tumors, the University of Alabama at Birmingham for pediatric brain tumors, New York Medical College for T-cell immunodeficiency, and Columbia University Health Sciences for vitamin D as a treatment for sickle cell disease.

EU Clinical Trial Transparency Under Fire

The European Medicines Association (EMA) is concerned clinical trial transparency in the European Union may suffer if a recent court ruling is allowed to stand.

In two separate rulings in February 2018, the EU Court of Justice found in favor of the agency in the cases Pari Pharma v. EMA and PTC Therapeutics International v. EMA. The pharma companies claimed that their trial results should be considered trade secrets and not subject to EMA’s transparency rules. EMA argued that sharing clinical trial data is in the public interest and does not violate sponsors’ commercial interests.

The Court ruled against the drugmakers, reinforcing the EMA clinical trial transparency policy. On appeal, however, a judge sided with the pharma companies and ruled they did not have to share their trial results.

Although the judge’s rulings are not binding without approval from the full Court of Justice, the EMA warned in a statement earlier this month that allowing the appeals ruling to stand would set a precedent against data-sharing and collaboration and require the agency to rewrite its transparency policies.

Datacubed Uses Patients’ Smartphones to Inform Trials

Datacubed Health launched a tool for detecting adverse events and key outcomes in clinical trials using patients’ smartphones.

The app is preloaded with a database of geographic locations of healthcare facilities, such as hospitals and doctors’ offices.

Once the patient is in one of these locations, chat bots and surveys can be sent to their phone to gather information about the outcome of the visit. Patients’ private information remains protected in the app.

Florence Healthcare’s eHub Will Connect Sites to Sponsors

Florence Healthcare has raised more than $7 million to develop software that will connect its thousands of research sites with sponsors.

The Florence eHub will tap 5,000 trial sites into a centralized network built to manage studies, connect sites to CROs and provide sponsors with an easier way to exchange study documents and data in trials.

The eHub will be a central platform for sites to access when looking for sponsors and CROs and planning the early stages of a trial, from building standard operating procedures, to training staff and workflows.

National Campaign Seeks to Find the Next Five Million Trial Participants

Circuit Clinical will use its online trial database, TrialScout, to share trial participants’ stories and encourage more patients to consider clinical trials.

TrialScout is modeled on the restaurant review service Yelp and allows patients to rate their trial experiences. Circuit Clinical’s “Find the Five” campaign will gather the stories of five million Americans who have participated in clinical trials in the hope of spurring five million more to volunteer.

Patients can post reviews of their trials for free, but sponsors and sites must pay to see their reviews.