Even at a place like Boston Children’s Hospital, where doctors regularly see children with rare diseases from all over the world, there are big challenges when it comes to drug discovery and treatment.
“Roughly 70 percent of drugs to treat children are used off-label,” says David Williams, Boston Children’s chief scientific officer. “That’s because these drugs were initially developed for adults and have not been tested formally in children.”
In order to cure rare diseases in children and adults, scientists must bridge the gap between research and industry. On May 25, Boston Children’s Technology and Innovation Development Office (TIDO) and MassBio held a candid panel discussion about what it will take to advance the development of rare disease therapies. Here are three of the biggest takeaways …
“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?
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. …
During the last decade or so, health care has been rapidly transforming from a reactive, paper-based system to a responsive digital model.
Massachusetts, under Gov. Charlie Baker’s leadership, has launched a comprehensive public-private partnership to accelerate the state’s digital health care sector. The partnership has identified multiple ways to drive investment and growth in the state.
Technology transfer from universities to private companies is just one example. In the past, each transfer required completely new agreements. Three new standardized templates for licensing, technology transfer and sponsored research will help facilitate these processes. In 2016, the partnership will expand its Mentorship Speakers Series with a stronger focus on digital health care. Finally, the Digital Healthcare Innovation Hub and Accelerator will provide a space to support and grow new digital health companies in Boston.
Vector visited with John Brownstein, PhD, Boston Children’s Hospital’s Chief Innovation Officer, to better understand the background and potential impact of this new initiative. …
When we think about innovation, especially in health care, our thoughts often turn to the highly complex: new surgical procedures, new drugs, new devices or machines, etc.
But innovation in medicine and patient care doesn’t have to be complex. Sometimes it can be very simple. Like a hat.
Karen Sakakeeny has been a clinical nurse for more than 30 years, spending much of that time in the operating room. While doing a stint in cardiac surgery, she found herself thinking about ways to improve the rewarming process for infants undergoing open heart surgery. …