Clinical News Roundup: FDA Rejects Evidence For Muscular Dystrophy Drug

By Ed Miseta, Chief Editor, Clinical Leader

FDA Panel: Lack Of Sufficient Evidence For Muscular Dystrophy Treatment

In 2014, the FDA noted an unmet medical need in Duchenne muscular dystrophy (DMD), and the need to make new treatments available. In that same statement, the agency also noted its commitment to working with all companies to expedite the development and approval of safe and effective drugs to treat the disease.

In the years leading up to that statement, the FDA worked extensively with Sarepta Therapeutics on eteplirsen, a treatment for DMD. The FDA provided guidance with respect to the data that would be necessary to determine if the treatment was effective and to support the filing of an NDA.  In April 2014, Sarepta announced that with additional data to support efficacy and safety, an NDA “should be fileable.”

In its advice to Sarepta, the FDA has consistently maintained that it would be necessary to include data in its NDA demonstrating that eteplirsen increases production of the muscle protein dystrophin. Eteplirsen’s proposed mechanism of action is through increasing the production of this muscle protein.

To the dismay of patients and their family members, an FDA panel this week voted against recommending approval of the drug. The advisory committee, voting 7 to 3 against with 3 abstentions, noted the small study of just 12 patients did not provide sufficient evidence of the drug’s effectiveness.

A Wall Street Journal article quotes panel chairman G. Caleb Alexander, a professor at Johns Hopkins School of Public Health, as saying he didn’t feel the study was well-controlled. Other FDA reviewers had previously concluded the study fell short of producing sufficient evidence. Panel members who abstained from the vote were undecided based on the evidence and testimony of parents who believed their children were helped by the drug.

 Lilly, Merck, Pfizer Share Recruitment Case Studies

A new white paper released by eyeforpharma, titled “Advancing Patient Recruitment & Engagement in Clinical Trials,” provides readers with the opportunity to learn patient recruitment strategies from Lilly, Merck, and Pfizer.

Oftentimes the success or failure of a clinical trial rests on the ability of the sponsor to recruit enough patients for the trial. The National Institute of Health (NIH) estimates 80 percent of trials fail to meet recruitment targets on time. Thirty-seven percent of sites in a trial fail to meet enrollment targets, and 10 percent fail to enroll a single patient.

“Patient recruitment can be the single most difficult element of a clinical trial,” notes the paper. “Trials frequently face lengthy and costly delays because patient recruitment yielded fewer patients than needed, and this has been demonstrated to be the case across the board, regardless of disease area or company particulars.”

The paper provides case studies from Lilly, Pfizer, Merck, and the AKU Society, detailing successful recruitment strategies. Paulo Moreira, VP of global clinical operations for EMD Serono, a Merck subsidiary, notes the company has completely overhauled the traditional approach to patient recruitment. The company’s new system will build an infrastructure that allows for cooperation with patients throughout the clinical trial process. Patients generally view trials as a last resort. The new program seeks to present trials as an extra option to be considered with current treatment options.

At Lilly, a new TrialGuide website contains the details of current Lilly trials and seeks to communicate trials to patients in an easy to understand format. The site contains videos of patients explaining their motivations for participating in trials. Much of the information on the site is presented in a graphical fashion. Educational content on the site aims to be dynamic, interactive, and diverse – not just words on a page.

At Pfizer, Craig Lipset, head of clinical innovation, worldwide research & development, notes that to create a better patient experience, companies must start by listening. TrialFinder is a new resource on Pfizer’s website that provides information about the company’s actively enrolling clinical trials.

Lipset believes many companies using social media as a tool for recruitment are using it in an antisocial way. “Advertising on Facebook or other networking sites does not qualify as true engagement. Failing to engage and share content makes companies essentially ‘invisible’.”

Lipset notes using these tools can deliver benefits to both sponsors and patients, but companies should be cautious of “engaging without first listening.”

The full paper can be requested here.

Can Machines Make Clinical Trials More Successful?

An article on Medical Xpress notes scientists are working to teach machines to make clinical trials more effective. The goal is to have computers figure out why patients decide to accept or decline invitations to participate in clinical trials.

The researchers at Cincinnati Children’s Hospital Medical Center are using “machine learning” technologies to predict whether or not patients will participate. The goal is to impact patient recruitment strategies and ultimately increase participation in trials. Researchers hope their efforts will also help more studies come to completion with more meaningful data.

According to the article, 6o percent of patients approached with traditional recruitment practices ultimately agreed to participate. Researchers predict that their new automated algorithm could push acceptance rates up to about 72 percent. As the algorithm is further developed and refined, the hope is to push acceptance rates even higher.

Researchers See Promise In Using Cannabis To Treat Severe Epilepsy

Fox 13 in Salt Lake City reports researchers at the University of Utah and Primary Children’s Hospital have spent two years evaluating whether pharmaceutical-grade cannabis can help treat young women with severe epilepsy.

Most of the patients participating in the study have previously been on trials of 10 different seizure medications that have proven to be unsuccessful. Researchers say the current results, in which young female patients were treated with Epidiolex, a liquid form of Cannabidiol, are promising.

“There have been mild side effects, most of them have been tolerable. For example, children have had diarrhea because of the oil the substance is dissolved in, some have been more tired, some changes in appetite,” says Dr. Francis Filloux, a researcher overseeing the study.

Researchers will wrap up testing within the next six months. If approved by the FDA, the medication could be on the market within two years.

Is Metformin The Fountain Of Youth?

Would-be research participants from around the world are suddenly scrambling to take part in a project to see if the Type 2 diabetes pill metformin can delay or prevent diseases brought on by advanced age, including heart ailments, cognitive decline, and cancer.

To test the pill, gerontologists at 14 aging centers around the U.S. will follow 3,000 seniors for 6 years. In the clinical trial, half of the seniors would receive metformin with the rest receiving a placebo.

According to Fox News, news of the research being conducted by Nir Barzilai, director of the Institute for Aging Research at the Albert Einstein College of Medicine, is leaking out and leading to seniors around the world wanting to take part in the study.

According to the article, would-be participants—from Cherry Hill, N.J., the Four Corners area of New Mexico, the Netherlands and beyond—have inundated Dr. Barzilai with calls and letters. Other researchers in the project have been swamped as well. For now, they will have to wait. Formal patient recruitment is still a year away.

New Networking Technologies Connect Patients With Researchers

Connecting patients with researchers has always been a struggle. With over 80 percent of U.S. patients treated in community healthcare settings, those institutions clearly hold the key to facilitating that connection. Genetic Engineering & Biotechnology News (GEN) is reporting new technology tools will reduce the time and effort involved with collecting clinical data and enable better patient engagement.

“As more healthcare data becomes digitized, it creates a foundation to support the development of new tools and platforms to facilitate research,” says Kate Torchilin, PhD and GEN contributor. “These tools reduce the time and effort involved in collecting clinical data for research purposes and they empower broader patient engagement, making it more plausible for community healthcare organizations to adopt these approaches.”

One example of these new tools is the Novaseek Clinical Data Network for Research (CDNR), a research platform that accepts data from multiple hospitals’ electronic medical records (EMRs), allowing researchers to query relevant data such as diagnostic tests, medications, and diagnoses. “Genomic profiles also are increasingly included in these records,” says Torchilin. “And with the growth of wearable devices, patients may begin adding that data to their own medical records for an even more comprehensive look at their health and lifestyle.”

 With this data, researchers can more easily stratify patient populations, collect biospecimens for precision medicine discoveries, and conduct noninterventional studies, such as determining the effects of lifestyle factors on disease.

Benchmark Research CEO Wins Innovation Award

Mark Lacy, CEO of Benchmark Research, has received the Innovation in Clinical Research award from the Association of Clinical Research Professionals (ACRP). The award was presented during the 2016 ACRP meeting in Atlanta. The meeting was jointly held with the 2016 U.S. Clinical Researcher of the Year awards, where Lacy served as chair and master of ceremonies. Daphne Dureau, sub-investigator and associate site director at Benchmark Research New Orleans was awarded “People’s Choice” for her submission to the Academy of Clinical Research Professionals Academy Certification Essay Contest.

“I’m delighted that our colleagues and peers believe in what Benchmark is doing, combining research and urgent care, and excited to open the doors of our first three urgent and family care clinics,” says Lacy. “I’m thankful to have been given the opportunity to serve as chair and master of ceremonies for clinical researcher of the year and so incredibly proud of Daphne for her brilliant essay.”