In Part 1 last week, Vector took a look at digital health apps, telemedicine, genomics, phenomics and new behavioral diagnostics as transformative trends in pediatrics. This week, we complete our list. These posts will also appear as an article in the fall issue of Children’s Hospitals Today magazine.
6. New pharma research and development (R&D) models
Academic medical centers have always worked with the pharmaceutical industry but never so closely as now. In the old model, industry drove therapeutic development. A company might fund an academic project or supply reagents, but the relationship generally ended with the project (and publication of a paper).
Now, with drug pipelines drying up and R&D costs rising, Big Pharma is under pressure to change. New industry-academia collaborations are forging creative partnerships, altering how both parties do business. The new models are allowing hospital researchers to do what they’ve never done before: take the lead in R&D.
Why the plethora of industry-academic collaborations? Academic medical centers have deep expertise in disease and the biology that underlies it, and a long-term commitment to finding cures—not to mention access to human tissue samples and to patients for clinical trials. Hospitals know what their patients need, and pharma is listening.
One example of this sort of collaboration: Pfizer’s Centers for Therapeutic Innovation network, which has established partnerships with Boston Children’s Hospital and Children’s Hospital of Philadelphia to support biomedical research. The company and hospitals work together to identify potentially influential, early-stage research at the hospital. The company then gives hospital researchers access to its commercial resources and drug development tools. Partnerships like this one will have huge effects, especially in research on orphan diseases where the medical need is great and where many of the ingredients for clinical trials are in place.
7. Innovation meets healthcare reform
Political and economic pressures to cut costs are forcing health care to adapt, and this is producing many innovations. It may be partially a survival move, but the emerging innovations in payment models, care coordination and care delivery are turning out to be big wins for patients.
As state insurance exchanges kick in, empowered patients and employers may be incentivized to migrate toward plans that support innovations like virtual telehealth visits, medical homes and greater utilization of non-physicians. Again, rural Alaska offers a great model: In remote areas, young junior college or high school graduates now deliver dental care. They complete 18-month training programs and are able to fill cavities and perform basic restoration procedures.
In his frequent talks around the country, Donald Berwick, MD, co-founder and president emeritus of the Institute for Healthcare Improvement and former Administrator of the U.S. Centers for Medicare & Medicaid Services, is calling upon physician “guilds” and hospitals to lead this change. “The legacy payment systems don’t support innovations,” he notes. “We need the help of the guilds. It’s got to be guided by evidence, but it is time to change. We need to say not ‘no,’ but ‘how?’”
But before embracing an innovation, we should demand evidence that it works. “Not all change is improvement, and not all innovations help,” he asserts.
8. Patient engagement
Parents today are comfortable with an instant exchange of knowledge through social media and online medical research. That’s especially true for pediatric diseases, where family support networks not only share information and tips but also influence and sometimes underwrite research. Many parents have health apps on their smartphones, and hospitals are starting to catch up by offering tools: from iPads with apps that help patients track their progress in the hospital to games with avatars that kids play at home, encouraging them to take charge of their health.
Miami Children’s Hospital systematically breaks down patient engagement into five touch points, each with its own tools: Pre-Arrival, Pre-Treatment, Point of Care, Discharge and Follow-up. At Pre-Arrival (in the waiting room) patients can check in digitally and are queried about medication adherence and gaps in care. They’re given information and reminders—about needed vaccinations, for example—and invited to participate in relevant clinical trials. At Discharge, there is similar follow-up on care gaps, a quick experience survey, and recruitment for support programs or follow-up visits.
At its best, patient engagement isn’t just about technology. It’s about an authentic two-way partnership in which clinicians can learn from families and offer them tools to help them manage their child’s care (check out the Care Map above, developed by parent Cristin Lind). Parents of children with special needs have long acted as the primary care coordinators for their children, often facing stress in navigating the health care system. A study recently published in Health Affairs surveyed 112 patient-centered medical home practices in 22 states, and found that while nearly all practices sought patient feedback, fewer than one third integrated them into governance structures or surveyed them for advice.
9. The 3D printing revolution
Commercial 3D printers build customizable objects layer by layer out of plastic, ceramics, resin or sometimes even more exotic materials. Medicine, too, is embracing 3D printing: to model a patient’s anatomy, to plan a surgery, to create tools and even to create actual tissues. Children’s National Medical Center in Washington uses patient scans to print life-sized models of their hearts, so doctors can practice procedures or surgeries ahead of time and adapt to the unique structure. They are able to switch between different plastics to make models that not only look realistic but also feel the way the actual heart will feel in surgery. At Boston Children’s Hospital, researchers are using data from patient MRI scans to print brain models to test a neurosurgical robot.
Three dimensional printing can produce implants for surgery too: Doctors at C.S. Mott Children’s Hospital (Ann Arbor, Mich.) printed an airway splint that kept a baby breathing, using a plastic that the body would slowly absorb as healthy tissue replaced it. Living cells, it turns out, can be also printed: A group from the U.K. successfully printed uniformly sized droplets of human embryonic stem cells gently enough to keep them alive and retain their ability to differentiate. A researcher at Wake Forest University (Winston-Salem, N.C.) has created cartilage tissue with a 3D printer by precisely layering live cells with synthetic materials, while Japanese researchers have printed 3D human tissue chips made up of two cell types for use in drug testing.
10. Learning from the rare to treat the common
As all children’s hospitals know, what we call “rare” diseases, when taken together, aren’t rare at all. Pharmaceutical companies have a growing interest in rare disease research, which offers favorable business models and often a faster path to clinic. As a result, discoveries are surging—and they hold lessons for doctors, researchers and patients trying to understand more common conditions.
In diseases like autism and epilepsy, rare genetic syndromes that include seizures or autistic features are shedding light on the underlying biology, suggesting treatment approaches that might also work in more common forms. Consider that the gene p53, now known to be spontaneously mutated in almost half of all cancers, was first discovered as the cause of Li-Fraumeni syndrome, an extremely rare familial cancer disease. Or take Hutchinson-Gilford progeria syndrome, which affects maybe 100 children in the world. Discovery of the gene causing this rare “aging” syndrome is shedding light on processes of normal aging.
Increasingly, too, rare diseases provide a launch pad for treatments that could benefit larger numbers of patients. Treatments like gene therapy or gentler ways of preparing patients for stem cell transplants were tested first in patients with rare bone marrow failure syndromes.
For more of what’s at the cutting edge, don’t miss our National Pediatric Innovation Summit + Awards (September 26-27).