The FDA said Friday it is illegal to perform clinical research in the U.S. on a new technology that showed promise last year in helping a woman who had multiple miscarriages because of a genetic disorder, to give birth to a baby boy in Mexico without that disorder.
The technology, known as mitochondrial replacement technology (MRT), impacts the mitochondrial DNA passed down only from mother to child. In September 2016, MRT was used to help a mother who carries genes for the fatal Leigh Syndrome and had multiple miscarriages to give birth.
According to the New Scientist, Dr. John Zhang of the New York-based New Hope Fertility Center and his team removed the nucleus from one of the mother’s eggs and inserted it into a donor egg that had had its own nucleus removed. The resulting egg included nuclear DNA from the mother and mitochondrial DNA from a donor and was then fertilized with the father’s sperm.
Prior to the boy's birth, the Institute of Medicine (IOM) of the National Academies of Sciences Engineering, and Medicine issued a report detailing how it believes FDA should regulate MRT trials. FDA, however, made clear on Friday that although the use of MRT is a possible treatment for mitochondrial disorders, the technique also "introduces a genetic modification and raises safety concerns."
Since December 2015, Congress has said in its annual federal appropriations laws that FDA is prohibited from accepting applications for clinical research using MRT.
PRA Buys Symphony Health
In the latest bid by a CRO to diversify, PRA Health Sciences has agreed to acquire a data and analytics company for $530 million in cash. The sum paid could be larger depending on Pennsylvania-based Symphony Health exceeding financial targets in 2017 and 2018, according to PRA's announcement of the deal.
In the announcement, PRA called Symphony Health "a pioneer in the physician-level targeting environment that biopharmaceutical companies use today to provide strategic market data that analyzes the industry from the perspectives of providers, payers and patients to a variety of private and public organizations." PRA stated that Symphony Health is expected to have revenues of more than $200 million this year. The company serves more than 400 customers.
“We are excited about this new relationship and look forward to helping Symphony Health continue to grow their existing business and working with them to expand geographically using PRA’s global footprint,” said PRA CEO Colin Shannon. “Symphony Health will also provide us with rich data insights that will allow us to customize our clinical studies to be as unique as the patients who they are designed around. By creatively harnessing the power of our technology and data assets, we are redefining the clinical development process for a more patient-centric future.”
The transaction is expected to close in the third quarter of this year.
monARC Builds Patient Networks for Next-Gen Trials
monARC, a startup company founded by pharma veteran Komathi Stem, has developed an interoperable, smart, health record platform that passively consolidates clinical data from medical records, images, and labs.
New treatments are greatly delayed during the clinical trial process, with a large bottleneck being at the design and enrollment stage of clinical trials. monARC helps researchers avoid costly amendments to clinical trial designs, prequalifies patients to exponentially accelerate patient enrollment, and enables connected clinical trials to broaden access to a wider and more diverse subject population.
monARC activates patient research networks powered with a transformative data collection and analytics platform that converges data from clinical care and digital devices, such as smartphones and sensors, into a smart health record. That record is a holistic, computable, longitudinal medical record that generates deep insights to inform trial designs and stratify patients. In addition, the platform leverages telemedicine and mobile technologies to facilitate trial participation from anywhere at anytime.
With the technology, patients are able to directly donate their data and participate in clinical trials. This is expected to result in faster, better, and cheaper clinical trials that bring treatments to market faster. The approach is also expected to accelerate trial recruitment, improve retention, and enroll a more diverse population of patients. The platform could save biopharma companies three to four years of development time and generate $80 million in operational cost savings per program.