Stories about: Market trends

More clinical trials in kids? Nearly half are unfinished or unpublished

pediatric trials clinical trials
Of 559 interventional trials in children, 19 percent were stopped early and 30 percent of completed trials remained unpublished several years later, finds a new study. (Vmenkov/Wikimedia Commons)

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 Yet, as with adult trials, these trials commonly stall out or, if completed, remain unpublished several years later, finds a study published online today in Pediatrics.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.”

Read Full Story | Leave a Comment

Citizen science: Giving patients a voice in drug development

citizen science patient voice drug development

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.

Read Full Story | Leave a Comment

Getting academic diagnostic discoveries to market: 6 tips from industry

diagnosticsdiagnostics (008) Loading of QIAsymphony platform“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.

Read Full Story | Leave a Comment

Microbiome therapeutics: 6 takeaways from a MassBio panel

microbiome therapeuticsSeeing 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.

Read Full Story | Leave a Comment

The face of telehealth: Serving children in health care “deserts”

pediatric telehealthAt 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:

Read Full Story | Leave a Comment

Reimagining connected health through home hubs

home health hubs

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.

Read Full Story | Leave a Comment

News Notes: Headlines in science and innovation

An occasional roundup of news items Vector finds noteworthy.

Zika’s surface in stunning detail; mosquito tactics

Zika virus
(Purdue University image/courtesy of Kuhn and Rossmann research groups)

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.

Meanwhile, as The New York Times reports, scientists are coming together in an effort to control Zika by genetically manipulating the mosquito that spreads it, Aedes aegypti.

Read Full Story | Leave a Comment

Accessing startup resources: 10 tips for physicians and scientists

accessing startup resources

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:

Below is their distilled advice for physicians and scientists seeking to commercialize a drug discovery, device or health app.

Read Full Story | Leave a Comment

Ten child health innovations headed to SXSWi

Impact Pediatric Health child health innovation
(U.S. Army/Lori Yerden via Flickr)

Innovation in pediatrics is alive and well. On March 14, at the South by Southwest Interactive (SXSWi) festival in Austin, Tex., Impact Pediatric Health will run its second annual pitch competition for digital health and medical device startups. Based on the ten child health innovations to be pitched, it promises to be as inspiring as last year’s event.

Judges include representatives from the four founding hospitals — Boston Children’s Hospital, Cincinnati Children’s Hospital, Texas Children’s Hospital and Children’s Hospital of Philadelphia — and from Sesame Workshop, whose recently announced Sesame Ventures plans to support companies that “help kids grow smarter, stronger and kinder.”

John Brownstein, PhD, chief innovation officer at Boston Children’s and one of the judges on the panel, agrees with that mission. “When it comes to innovation, pediatrics is often a second thought or gets left out altogether,” he says. “I’m extremely impressed with the landscape this year and the breadth of startup ideas.”

Read Full Story | Leave a Comment

Rare diseases: tools, lessons, discovery

rare diseases toolsWhen rare diseases are taken together, they’re not all that rare. Their underlying genes provide biological insights that drive therapeutic advances and often shed light on more common disorders. Thanks to advances in genomics and bioinformatics, growing interest from pharma and a burgeoning citizen science movement, rare disease is poised to rock biomedicine. This Storify recaps a Twitter chat hosted by the NIH (#NIHchat) ahead of Rare Disease Day on February 29. People shared statistics, great examples of rare disease science, directories of diseases/disease organizations and tools for patients, clinicians and researchers.

Read Full Story | Leave a Comment