September 8, 2019
Calls for the nomination of acting FDA Commissioner Ned Sharpless to fill the permanent leadership role at the agency continue to grow as the cutoff date for his acting status approaches.
President Trump reportedly met with another potential candidate last week — Stephen Hahn, chief medical executive of the MD Anderson Cancer Center in Houston. But dozens of health groups and four former FDA chiefs endorsed Sharpless last week in letters to the White House and HHS Secretary Alex Azar.
The health organizations — which include 50 cancer groups — praised Sharpless as “an extraordinarily strong and respected leader” in his short time at the agency’s helm.
Sharpless has “gained the respect of the agency staff and a broad spectrum of the public,” former Commissioners Robert Califf, Andrew von Eschenbach, Margaret Hamburg and Mark McClellan said in a separate letter.
Former FDA Commissioner, Scott Gottlieb, who recommended Sharpless for his current role, called the former NCI director “an outstanding physician and scientist.”
Also believed to be in the running are Alexa Boer Kimball, a Harvard dermatology professor and president and CEO of Harvard Medical Faculty Physicians, and Brett Giroir, assistant HHS secretary for health.
Sharpless must step down on Nov. 1 if he doesn’t receive the nomination for the permanent position.
Read the former FDA commissioners’ letter here: https://bit.ly/2lX61SH.
Read the health group letter here: https://bit.ly/2kwuQo8.
Gather Patient Input to Guide Drug Development: The FDA’s Four-Part Plan
Sponsors early next year will have a chance to weigh in on the development of a new FDA guidance on the collection, capture, storage and analysis of clinical outcome assessment (COA) data provided by patients.
The FDA also plans to issue two related draft guidances on methods to identify what is important to patients, and how to select, develop and modify fit-for-purpose clinical outcomes assessments.
The first in the series of the four draft patient-focused guidances was issued in June 2018 and focuses on collecting comprehensive and representative input from patients. Specific topics in the guidance include defining target populations, determining sampling methods for collecting patient input, qualitative and quantitative research methods, and approaches for collecting and managing data.
Patients should be considered experts on their own conditions, the agency says, noting that they are in a unique position to contribute to the understanding of benefit-risk considerations throughout the development process.
The draft guidance on identifying what is important to patients was originally scheduled for release by mid-2019 but has not yet been issued.
Read the first draft guidance here: https://bit.ly/2kwA6rQ.
Read the FDA’s four-part plan here: https://bit.ly/2m363Zt.
Test Seizure Treatments on Children as Young as Two, FDA Guidance Advises
Sponsors of drugs to treat partial onset seizures (POS) in pediatric patients should extend their trials to children as young as two, according to a final guidance the FDA released last week.
The guidance replaces a February 2018 draft guidance that placed the lower age limit at four years. A minimum of 100 pediatric patients of all ages two years and up should be exposed to the drug for at least six months of treatment, the guidance says.
Read the guidance here: https://bit.ly/2k1JvYh.
Johns Hopkins Center to Study Psychedelics as Therapies for Brain Conditions
Johns Hopkins University will be home to the first U.S. research center to focus on the effects of psychedelic and hallucinogenic drugs.
Researchers at the new Center for Psychedelic and Consciousness Research will study the drugs’ potential as treatments for conditions such as post-traumatic stress disorder, depression, addiction and Alzheimer’s disease, looking at the effects on memory, brain function, learning and memory, and mood.
In addition to studies on new therapies, the center plans to investigate creativity and well-being in healthy volunteers.
Johns Hopkins has had federal approval to study psilocybin — a chemical found in certain types of mushrooms — since the early 2000s, but most of the drugs the new center plans to study are still illegal in the U.S.
The center, which will be the largest research center for psychedelics in the world, is funded by a $17 million grant from a group of private donors. The only other psychedelics research center opened in April at Imperial College in London.
Modis Therapeutics acquired by Zogenix
Zogenix, which develops rare disease therapies, announced that it has acquired Modis Therapeutics, which focuses on developing novel therapies for rare genetic diseases with high unmet medical need.
The acquisition brings Modis’ MT1621 into the Zogenix portfolio. MT1621 is a proprietary, late-stage investigational medicine that targets Thymidine Kinase 2 deficiency (TK2d) that has received FDA Breakthrough Therapy and EMA PRIME designations.
PPD Acquires Bioclinica’s Clinical Research Site Business
PPD’s Accelerated Enrollment Solutions (AES) business unit has acquired the clinical research site business of Bioclinica.
The acquisition adds new sites and opens key markets for PPD in China, Latin America and Western Europe. The deal further bolsters the company’s presence in North America, Europe and South Africa, and provides dedicated research sites in Florida with significant neuroscience expertise.
Cancer Drugs Are 27 Percent of New U.S. Approvals: Tufts
Cancer drugs now account for 27 percent of all new drug approvals in the U.S., up from just four percent in the 1980s — in part because of improved trial designs, novel drug formats and new targets — according to a new study from the Tufts Center for the Study of Drug Development (CSDD).
Between 1980 and 2018, the FDA approved 126 cancer drugs to treat solid and hematologic cancers, the center found. The researchers found that while development time for cancer drugs during 1999-2018 was nine percent longer than for non-cancer drugs, regulatory approvals during the same period was 48 percent shorter for the cancer drugs on average.
Higher percentages of new cancer drug approvals received priority ratings from the FDA and had orphan drug status during 1999-2018, compared to new non-cancer drugs, Tufts found.
Between 1993 and 2018, solid tumor cancer drug approvals increased more than blood cancer approvals. The total time for clinical development and approvals for blood cancer drugs was 17 percent longer than drugs for solid tumors.
Read more about the CSDD report at: https://bit.ly/2su7gde.
GRN to Host “Be the Site of Choice” Conference
Sites can learn how to stand out in a crowd at the “Be the Site of Choice” conference next month in Philadelphia.
Hosted by the WCG Global Research Network (GRN), the one-day conference focuses on methods for making sites more competitive. Industry experts talk about what sponsors want from sites, what sites need from sponsors to succeed and metrics sites should collect to evaluate and improve their performance.
The conference will be held Oct. 7. To register, click here: https://bit.ly/2lBOQpG.
Australian-Based CRO Buys Prism Clinical Research
Australian CRO Nucleus Network has acquired St. Paul, Minnesota-based Prism Clinical Research for $7 million.
The acquisition will give Nucleus Network, Australia’s largest dedicated phase 1 clinical trial organization, its first entry into the U.S. market.