Health Canada Issues Guidance on Off-Label Uses of Drugs in Trials

Health Canada released new guidance that the agency hopes will make it easier to study the off-label benefits of drugs in clinical trials.

Canadian regulations currently require any treatments purchased for clinical trials for their off-label properties to be designated “investigational drugs.” But Canadian officials worry that’s adding unnecessary hurdles that slow life-saving or life-changing research.

Under the new guidelines, Canadian officials will ask a series of questions about a given treatment before deciding whether it has to be designated an investigational drug, including:

• Does the drug have “an established safety profile when used off-label in the study population;”
• Is it being used as part of a comparator arm in a clinical trial, as supportive or rescue therapy, or whether it’s part of a standard therapy “that another investigation drug is being used to supplement in the investigational arm of the trial;”
• Is there any uncertainty about the risks and benefits of the off-label use;
• Is the drug already authorized in Canada and will trial doses will be bought in Canada;
• Does the off-label use increase the risks for patients beyond the normal risks of taking the medicine; and
• Is it “otherwise clear that the drug to be used off-label is not being tested in the clinical trial.”

Sponsors will have to provide the proposed off-label drug’s Notice of Compliance or Drug Identification Number and explain what the original indication was for. They also will have to explain the proposed off-label use in the trial, how the proposed use is “consistent with current or recognized medical practice,” why the off-label use shouldn’t be considered as a separate clinical trial, and the risks of off-label use, the agency says.

Read the guidance here: https://bit.ly/2XIl2Wy.

French Titan Dassault Acquires Health Software Firm for $5.8 Billion

Tech giant Dassault Systèmes is buying health software company Medidata Solutions in a whopping $5.8 billion arrangement.

Medidata brings to the table cloud-based technology and clinical expertise that helps fuel the development of therapies for a host of customers, including CROs, drugmakers, biotech firms, and medical centers and sites. The company says it recently has branched out into real-world evidence and analytics.

Janssen Offers Patient-Centered Communication Training

Janssen has developed a training platform aimed at helping clinical trial professionals and patients communicate effectively.

The “HealthCaring Conversations” program currently is in the pilot stage, being used only by a selection of Janssen-sponsored trials, but the company plans to begin rolling it out to all of its trials worldwide in October.

Based on behavior science and a large literature review, the program offers digital learning courses, on-site training and webinars to help trials increase enrollment and retention by teaching them how to communicate with potential trial subjects.

Janssen plans to offer the program to non-Janssen trials eventually and invites site managers to attend its workshop on patient communication at the Society for Clinical Research Sites conference in October.

Dutch Hospitals Test Performance-Based Payment for Off-Label Cancer Treatments

A cancer patient who responds well to an off-label treatment in a clinical trial should not have to pay out of pocket for the drug just because it is not considered as standard of care, a Dutch research institute believes.

The Netherlands Cancer Institute-Antoni van Leeuwenhoek Hospital is launching a pilot study in which drugmakers and health insurers will share the cost of a drug that has proven effective for an individual even though it isn’t indicated for that person’s type of cancer.

The pilot study will use Bristol-Myers Squibb’s cancer drug Opdivo (nivolumab) for off-label treatment of various rare tumors. The drugmaker will guarantee the medication costs for a patient’s 16-week evaluation phase, and the health insurers will reimburse patients for whom the off-label medicine appears safe and effective after the evaluation period.

“It is becoming increasingly clear that every tumor is biologically unique and that treatment must therefore increasingly be customized to the individual patient,” the institute says. “

Large-scale comparative drug studies usually needed for the approval of new indications cannot be conducted for rare tumors, the institute says.

Performance-based drug payments are gaining traction elsewhere, including the US. In November 2018, the Centers for Medicare and Medicaid Services approved Michigan’s plan to allow its Medicaid program to negotiate payment arrangements with drugmakers based on patient outcomes.