It seems like a great idea. We all have our genomes sequenced at birth, and any findings that suggest a future medical problem are addressed with early interventions, optimizing our health and extending our lives. But are parents of newborns ready to embrace the vision? Yes and no, according to interim results of a first-of-its-kind randomized trial of newborn sequencing. Findings from what’s known as the BabySeq Project were presented last week at the American Society of Human Genetics (ASHG) 2016 Annual Meeting. …
Boston’s digital health world is humming with tech talent, idealistic health care professionals and business-savvy accelerator organizations. The passion was palpable last week as 300-plus people gathered at MassChallenge’s latest Pulse@Check digital health meetup, hoping to turn their health care ideas into reality.
The event, hosted by Boston Children’s Hospital’s Innovation and Digital Health Accelerator (IDHA) and Cerner, a lead developer of health care IT systems, presented numerous opportunities and tips for digital health startups. …
Recent laws like the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act are encouraging clinical trials in children. Yet, as with adult trials, these trials commonly stall out or, if completed, remain unpublished several years later, finds a study published online today in Pediatrics.
“Our findings may speak to how commonplace discontinuation and non-publication are in medical research in general,” says Natalie Pica, MD, PhD, a senior resident at Boston Children’s Hospital and the study’s coauthor. “We need to make sure that when children participate in clinical trials, their efforts are contributing to broader scientific knowledge.” …
There’s a natural tension between wanting the FDA to ensure safety and efficacy before a drug enters the market and wanting to speed up what many view as a glacially slow approval process. The rare disease community tends to fall in the second camp, and has become increasingly vocal in calling for more clinical trials, more flexibility in their design and redefinition of what constitutes a benefit.
ALS advocates, for example, have called for a parallel track, “in which FDA provides an early approval based on limited data, and then continues the learning process in a confirmatory clinical trial and if needed, patient registries to collect additional data from patients receiving the drug outside the clinical trial…”
Recent legislation is encouraging patient engagement in drug development, especially for conditions with profound unmet medical needs. In its 2012 iteration, the Prescription Drug User Fees Act (PDUFA) introduced public meetings to get input from the patient community, captured in a series of informative white papers. …
“Wouldn’t it be great if we could come up with a noninvasive diagnostic assay to detect pancreatic cancer at an earlier, more treatable stage?” asked Lori Aro of Myriad Genetics. Her company has been trying to do so for years. So why hasn’t it happened?
Aro, senior director for new product planning at Myriad, outlined the business obstacles at a recent panel hosted by Boston Children’s Hospital’s Technology and Innovation Office (TIDO).
First, who are the target patients for a pancreatic cancer test? Skinny diabetics, patients with chronic pancreatitis, patients with hereditary cancer risk — or all three? “Those three patient types all sit in different doctor’s offices,” said Aro. Simultaneously reaching endocrinologists, gastroenterologists and high-risk patients would be an insurmountable challenge, Myriad concluded.
Second, the assay would likely need to be validated in all three patient populations, with confirmatory imaging. Could the test populations be large enough to make the results statistically significant?
Third, a new test wouldn’t change care, as there is no treatment for pancreatic cancer. In fact, no current data show that earlier diagnosis improves survival. So who would pay for it?
Aro’s story exemplifies just some of the challenges in developing a new diagnostic test. …
Seeing the surprising success of “poop pills” in gastrointestinal C. difficile infection, pharma companies and startups are embracing the microbiome as a new therapeutic target for an astonishing range of maladies. To learn what pioneering companies in the space are thinking about the hope and the hype, Vector recently attended a panel on microbiome therapeutics at the MassBio Annual Meeting. …
At least 15 million children reside in Health Professional Shortage Areas (HPSAs) that average fewer than one health professional for every 3,500 people. In these health care deserts, time and transportation barriers prevent even children with health insurance have trouble getting timely care, particularly specialty care. Children in poor, rural areas are most at risk.
So health problems fester and get worse — and more expensive when finally addressed.
Telehealth can solve many of these problems. Through remote video/voice/data connections, dermatologists can view images of rashes and moles sent by primary care providers; cardiologists can patch into local emergency rooms and listen to heart sounds and read EKG tracings; critical care physicians and neonatologists can see and hear newborns in distress, listen to lung sounds, read their vital signs and view images. They can advise local clinicians and guide them through next steps.
However, pediatric telehealth hasn’t been adopted as widely as it could be. A white paper presented by the Children’s Health Fund at a Congressional briefing last week enumerated the obstacles: …
Through smart home hubs and the growing Internet of Things, people can now control lights, thermostats and other appliances and get information and entertainment with their always-connected digital devices. Consumers have widely adopted home automation products like Nest from Google and ecosystems like Apple’s HomeKit and Amazon’s Alexa.
But home hubs also have the potential to achieve the promise of connected health — access to health care services anywhere and anytime.
Home hubs can deliver enormous value as a means of health care delivery — not just helping casual consumers become familiar with their health and take preventive measures, but also helping manage complex care for patients with chronic illness and supporting timely decision making by clinical teams. Everybody involved with a person’s care can be plugged in, enabling coordination across providers and caregivers in a way that’s increasingly intuitive and meaningful. …
An occasional roundup of news items Vector finds noteworthy.
Zika’s surface in stunning detail; mosquito tactics
We haven’t curbed the Zika epidemic yet. But cryo-electron microscopy — a newer, faster alternative to X-ray crystallography — at least reveals the structure of the virus, which has been linked to microcephaly (though not yet definitively). The anatomy of the virus’s projections gives clues to how the virus is able to attach to and infect cells, and could provide toeholds for developing antiviral treatments and vaccines. Read coverage in the Washington Post and see the full paper in Science.
How do you get companies or investors to support your project in a startup world where “many are called, few are chosen?”
Vector attended a panel last week on the subject, moderated by Ryan Dietz, Senior Licensing Manager at Boston Children’s Hospital’s Technology and Innovation Development Office (TIDO). The panelists were:
- Tom Luby, PhD, Senior Director, New Ventures, Johnson & Johnson Innovation, Boston
- Carl Berke, PhD, Co-Founder and Managing Director, Mass Medical Angels; Partner, Partners Innovation Fund (part of Partners HealthCare); Diagnostic Technologies Representative, Boston Biomedical Innovation Center (B-BIC)
- Kevin Bitterman, PhD, Partner, Polaris Partners
Below is their distilled advice for physicians and scientists seeking to commercialize a drug discovery, device or health app. …