Imagine a patient checking their blood sugar level several times a day with a glucose meter to keep their diabetes under control. Or maybe someone with a hip joint replacement or a stent inserted in their coronary artery to treat a heart blockage. Or maybe someone who participated in a clinical trial to help researchers better understand a new device to treat a condition.
It’s possible those patients had some unexpected experience with the device or clinical trial they would like to share with their physician, the manufacturer, other patients, or even FDA. Until now, there was really no easy way to do so.
Now, there is a way. For the first time, an FDA advisory committee will focus on patient-related issues. In October 2017, FDA will hold the inaugural meeting of the new Patient Engagement Advisory Committee (PEAC). The topic will be challenges of clinical trial design, conduct, and reporting identified by patients.
FDA chose this subject because patients often have concerns about participating in clinical trials or drop out once they have enrolled in a trial. Inconsistent or minimal participation in clinical trials can make it difficult to reach reliable conclusions or to determine the level of benefit for patients. It also can take longer to bring technological advances to the patients who need them.
The nine core voting members of the patient advisory committee, including the chair and the consumer representative, all have direct experience as a patient or as a care-partner for a patient. They are experts in the field of patient engagement, and their experience extends beyond their personal disease or condition to the broader patient perspective, which is a critical piece of FDA’s work. While patient representatives currently participate in many FDA advisory committee meetings, there has never been a committee that was wholly focused on patients.
FDA Issues Guidance On IRB Waiver
The FDA has issued a guidance titled "IRB Waiver or Alteration of Informed Consent for Clinical Investigations Involving No More Than Minimal Risk to Human Subjects." This document provides guidance to sponsors, investigators, and institutional review boards (IRBs) on enforcement of FDA regulations governing informed consent requirements for clinical investigations that involve no more than minimal risk to human subjects.
The 21st Century Cures Act amended the Federal Food, Drug, and Cosmetic Act to provide authority for FDA to permit an exception from informed consent requirements when the proposed clinical testing poses no more than minimal risk to the human subject and includes appropriate safeguards to protect the rights, safety, and welfare of the human subject. FDA intends to issue regulations to reflect this statutory change. Until FDA issues these regulations, this guidance informs sponsors, investigators, IRBs and other interested parties that FDA does not intend to object to an IRB waiving or altering informed consent requirements, as described in the guidance, for certain minimal risk clinical investigations.
In addition, this guidance explains that FDA does not intend to object to a sponsor initiating, or an investigator conducting, a minimal risk clinical investigation for which an IRB waives or alters the informed consent requirements as described in the guidance.
The guidance is now available on FDA’s website.
FDAnews Joins WIRB-Copernicus Group
WIRB-Copernicus Group (WCG), a provider of solutions to improve the quality and efficiency of clinical research, announced it has acquired FDAnews, a provider of global regulatory information for executives in the clinical research, biopharmaceutical and medical device industries.
FDAnews joins publisher CenterWatch to create a new resource for the life science community. FDAnews/CenterWatch provides a suite of newsletters, books, databases, market intelligence reports, conferences, and webinars, including Drug Industry Daily, CenterWatch Monthly, the Clinical Trials Data Library, the Medical Device Quality Congress and the FDA Inspections Summit.
“We are delighted to welcome the accomplished team from FDAnews to our family of companies and excited to see what the combination of FDAnews and CenterWatch will produce for our industry,” said Donald A. Deieso, PhD, chairman and CEO of WCG. “We have long prided ourselves on providing thought leadership and information solutions to the clinical trials community and look forward to extending our offerings to other segments of the biopharmaceutical and medical device ecosystem.”
Will Right-To-Try Threaten The Integrity Of Clinical Trials?
As Congress renegotiates the FDA Reauthorization Act, a must-pass piece of legislation that lets the FDA collect fees from pharmaceutical companies that submit their products for FDA review, Sen. Ron Johnson (R-Wis.) has threatened to hold up the bill unless the Senate adds a “right-to-try” amendment to the House version currently on the Senate calendar. But Kelly McBride Folkers, a research associate in the Division of Medical Ethics at the NYU School of Medicine, believes that despite its benevolent intention to help patients, right-to-try legislation threatens the integrity of clinical trials.
Money collected through the FDA Reauthorization Act funds approximately 70 percent of the Center for Drug Evaluation and Research (CDER), the FDA division that regulates prescription and over-the-counter drugs. This bill must pass in order for the agency to continue approving drugs for safety and efficacy. If the user-fee bill is not reauthorized by September 30, Folkers notes thousands of FDA employees could lose their jobs.
“A right-to-try amendment would tack onto this legislation a way for terminally ill patients to use investigational drugs outside of clinical trials and without FDA oversight,” notes Folkers. “But, existing state right-to-try laws do not require that drug companies give patients exercising their “right” experimental drugs, and the policy allows companies to charge patients for them, even if they don’t work or could be fatal. The federal policy would be no different. Ironically, adding a right-to-try amendment to a bill that funds a large part of the FDA’s operations would theoretically allow patients to use drugs that aren’t yet shown to be safe or effective — the very reason the agency exists and requires user fees.”
Type 1 Diabetes Vaccine Headed For Trials
By the year 2050, it’s anticipated that 5 million people will be diagnosed with Type 1 Diabetes (T1D) in the U.S. alone. The disease, which affects both children and adults, currently cannot be prevented or cured. Blood glucose monitoring and insulin injections are the current method for patients to treat their condition.
The belief that T1D could be related to viral infection has led some to propose the possibility of creating a vaccine for the disease. In Finland, researchers have been exploring this connection and potential vaccine for approximately 25 years. After such a laborious scientific journey, they believe they’ve found the viral group that can trigger T1D. The team has now created a prototype vaccine which will move into human clinical trials by 2018.
While it’s unlikely that the vaccine would become an immediate cure-all T1D, a successful trial could dramatically shift the future of the disease. Complications of the disease, which can result when it goes undiagnosed or is ineffectively managed, can range from heart attack to stroke, amputation, kidney failure, and even blindness.
The threat of these complications constantly hangs over the heads of those with T1D. Unfortunately, as the team notes, this vaccine would not be a cure for T1D, but if it proves successful in preventing the onset of the condition, it could change the lives of millions of people around the world.
China Strengthens National Clinical Research Centers
China will strengthen the establishment of national clinical research centers, according to the Ministry of Science and Technology. A total of 32 national clinical research centers, involving 11 disease categories such as cardiovascular, malignant tumor and respiratory system, were given licenses at a conference on advancing clinical research held by national authorities in Beijing on Saturday.
A development plan, a guideline and an assessment plan on national clinical research centers were released at the conference. According to Wang Zhigang, vice minister of science and technology, national clinical research centers should take on the main responsibility in linking clinical medicine with life sciences and biotechnology research, and applying clinical research into practice.
National clinical research centers will be located at 30 top hospitals and more than 2,100 medical institutions.