2014 continued to see massive evolution in health care—from digital health innovations to the maturation of technologies in genomics, genome editing and regenerative medicine to the configuration of the health care system itself. We asked leaders from the clinical, research and business corners of Boston Children’s Hospital to weigh in with their forecasts for 2015. Click “Full story” for them all, or jump to:
The consumer movement in health care
Evolving care models
Genomics in medicine
Stem cell therapeutics
1. Industry will angle to engage with patients
Three converging trends—savvy consumers demanding more engagement from care providers, hospitals facing pressures to reduce costs, and a move toward new payment models—are pushing industry players to take a closer look at how to reach patients beyond the acute care setting.
Look for more consumer-centered innovations, like DisCo (supported by the Boston Children’s Hospital’s Innovation Acceleration Program) which closes the post-discharge communication loop by sending automated check-in messages to the patient’s cell phone or e-mail. If the patient responds with a problem or a concern, a nurse phones to address the issue.
To keep up with ever-changing consumer demand, companies will continue to invest in innovation, and especially in digital health. The endgame will be to develop solutions that meet consumers’ health needs and seamlessly integrate into their lifestyle. Look to examples in CVS’s newly announced Health Digital Innovation Lab and Walgreens’ telemedicine app offering 24/7 physician access, as these two companies battle over who can better reach, engage and retain retail health care consumers. —Gena Koufos, RN, MS, MBA, Program Manager, Innovation Acceleration Program, Boston Children’s Hospital
2. Consumers will begin to vote with their wallets
People are starting to feel the crunch in their wallets from new health care costs. Insurance plans have changed to push people to make lower cost choices and absorb more out-of-pocket costs through co-insurance and high deductible plans. Consumers are waking up to the financial impact of health care, and their confusion is prompting them to ask more questions about the value of the services they are getting. This is the year of the consumer movement in health care.
Percolating around the edges of this change has been the growth of consumer health-tracking technology such as sensors and wearables, and Apple HealthKit, a platform that can bring together data from multiple health and ﬁtness apps and share it with participating electronic health record (EHR) systems. Until now, these gadgets have mostly been at the fringes. The tipping point for widespread adoption will come as more financial incentives align for consumers to reduce their health care costs through prevention of chronic diseases. —Alexandra Pelletier, Program Manager, FastTrack Innovation in Technology (FIT), Boston Children’s Hospital
3. The year of care integration
2015 will be the year when the activities that support care integration (care coordination, care plans, and shared and collaborative care models) will cross the threshold from “nice to have” to “must have” for health care organizations committed to delivering high-value patient- and family-centered care. In 2014, the Integrated Care Program at Boston Children’s saw a dramatic rise in the demand for care integration services. We expect this uptake to gain momentum in 2015 as organizations that invested early begin to realize gains in patient experience, quality and avoided unnecessary costs.
Further, 2015 will also be a defining year for the long-term sustainability of care integration. Leading indicators will include payer decisions to reimburse for care coordination services; the integration of tools into the electronic health record that support and measure care coordination services; and further dissemination of formal care coordination training.
Subspecialists will actively engage in the evolution of integrated care models, enabling more effective care delivery in partnership with their primary care colleagues who continue to implement the family- and patient-centered medical home. —Richard Antonelli, MD, Medical Director, Integrated Care and Strategic Partnerships, Boston Children’s Hospital
4. Tackling medical complexity
The results of a wonderful set of studies will shed light on the ways that we can optimize health care for chronically ill, medically complex children—like this randomized clinical trial just published by JAMA, which compared a comprehensive outpatient management program with usual community care. A current bipartisan bill encourages states to innovate and improve care models for children with medical complexity, and our own study, published last month in Health Affairs, made a business case, suggesting that cost savings from reduced hospital and emergency department use could underwrite investments in outpatient and community care.
Hospital discharge care—critical in children with medical complexity—will become increasingly more patient-centered, methodical and thoughtful. There is so much to learn, and there are some very meaningful projects wrapping up across the U.S. that will help to push the field forward. —Jay Berry, MD, MPH, Complex Care Service, Boston Children’s Hospital
5. Global medicine will expand
As countries like Qatar and China continue to invest in their health care capabilities, more international partnerships will develop to support new academic medical centers abroad. In November, I had the opportunity to visit Doha, Qatar as part of an MIT Hacking Medicine team. Qatar is pouring enormous amounts of capital into expanding its technological capabilities, including its $7.9 billion Sidra Medical and Research Center, which will be equipped with smart beds that keep patients moving to avoid bed sores and palm-scanning technology to store patient records. Thousands of expatriates from the U.S. and around the world are being recruited to staff Sidra. In October, Massachusetts General Hospital signed a framework agreement with a Chinese hospital to build a facility in Guangzhou Province, about 30 miles from Hong Kong.
Such partnerships could help bridge the international gap in medical services and training, whether it’s through the recruitment of Western-trained doctors or through the use of innovative delivery methods like telemedicine services. These partnerships would create new referral channels for American medical institutions, resulting in new international revenue streams and first-mover advantages, strengthening the institutions’ reputation abroad. —Judy Wang, MS, Program Manager, Telehealth Program, Boston Children’s Hospital
6. Genomic sequencing at birth advances
As genomic discoveries continue, costs fall and evidence of diagnostic utility accumulates, 2015 will see increased interest in genomic sequencing and requests from parents of children born with complex medical conditions. To date, some 7,500 single-gene, heritable disorders have been cataloged by medical genetics experts, and the underlying molecular cause of disease described in nearly half of these disorders.
Informed by the MedSeq program, Boston Children’s Hospital and Brigham and Women’s Hospital will be intimately involved in researching newborn sequencing this year. BabySeq will begin enrolling families with newborns, both healthy and ill, into a trial that will use whole-exome sequencing for potential medical diagnoses and evaluate its impact on care. Expect to hear much more about this study over the coming year.
Will parents participate? Yes! Researchers at Brigham and Women’s and Boston Children’s recently published results of a study in which they surveyed more than 500 parents with newborns. The majority of parents said they were at least somewhat interested in the option of newborn genomic sequencing. —Patrice Milos, PhD, President and CEO, Claritas Genomics
7. Interpreting genomic information
As genomic sequencing becomes more available, we’ve been seeing a growth in the “ome” of false-positive incidental findings—the incidentalome. Through sequencing of the genomes of large populations, 2015 will reveal that an increasing number of genomic variants previously considered to be highly predictive of specific diseases are actually much more weakly associated with those diseases. This already has been shown for some mutations thought to be causal in cystic fibrosis, hemochromatosis and cardiomyopathy. Through “big data” science, we will begin to learn the real clinical implications of each mutation. At the same time, the incidentalome will make insurers more reluctant to pay for genetic testing on individuals without any clinical symptoms.
A new kind of problem will become manifest in cancer genomics. Despite the growing relevance of genomic sequencing for choosing individualized therapies for cancer, none of the major EHR companies will deploy to their major customers software that stores and interprets whole-genome or whole-exome sequencing data for clinical decision support. This gap will leave the door wide open for clinically oriented genomic interpretation platforms to disrupt the current market offerings in cancer care (or to be acquired by the current EHR companies). —Isaac Kohane, MD, PhD, Chair, Children’s Hospital Informatics Program (CHIP)
8. Pluripotent stem cells will drive new treatments
Around the globe, therapeutics derived from human pluripotent stem cells are entering early clinical testing. For example, ViaCyte has made mature human beta cells—using both embryonic stem (ES) cells and induced pluripotent stem (iPS) cells—which promise to restore insulin production and normalize blood sugar levels in diabetics. Groups in Japan, the U.K., and Massachusetts are attempting to revert vision loss in macular degeneration using retinal cells derived from human ES or iPS cells.
At the Boston Children’s Center for Cell Therapy, we make hundreds of stem cell lines from patients using state-of-the-art reprogramming and gene correction technologies. These patient-derived stem cell lines help us to study diseases and find new drugs, and by turning these stem cells into tissues, we hope to repair diseased organs. For example, we can now generate blood cells that could be used as an alternative to blood or platelet transfusion, and create muscle progenitor cells that could be used for muscular dystrophy treatment. —Leonard Zon, MD, Director, Stem Cell Research Program, Boston Children’s Hospital
9. Mesenchymal stem cell therapies will approach fruition
This year, efforts will intensify to develop therapies using adult mesenchymal stem cell (MSCs), which give rise to a variety of connective tissues. While hundreds of MSC-based clinical trials are currently registered at ClinicalTrials.gov, many of the results have been underwhelming. This has been attributed to multiple factors, including insufficient understanding of the administered cells’ pharmacokinetics and ability to engraft in the body.
Nonetheless, recent studies give important additional insights into cells’ biological attributes and their communication with other cells in the body, and should lead to more rational therapeutic efforts. New trials will likely continue to target a plethora of conditions, including cardiovascular diseases and immune-mediated disorders, but with the increasing recognition that MSC-based therapies need to be more specific and target their tissue of origin (such as using bone-derived MSCs to treat bone related disorders). We will also see an increasing appreciation of cell-to-cell communication in stimulating regenerative processes (see here, here, and here). To date, most clinical trials have transplanted just one cell type, but mounting evidence indicates that long-term regenerative capacity after transplantation is impaired if stem cells are separated, even transiently, from the cells that naturally support them. This year we will see a more systematic use of multicellular protocols—and hopefully improved outcomes of stem cell transplantation. —Juan Melero-Martin, PhD, Cardiac Surgery Research, Boston Children’s Hospital
The vibrant field of perinatal regenerative medicine should bring some interesting developments in 2015, including advances in trans-amniotic stem cell therapy using MSCs isolated from amniotic fluid. Our group, for example, demonstrated that a single injection of amniotic MSCs into the womb caused skin to grow over the spinal cord in a rat model of spina bifida, sometimes completely covering the defect, and minimized the chiari malformations that often accompany spina bifida. Clinical applicability of fetal stem cell-based therapies will edge closer to reality. —Dario Fauza, MD, PhD, Surgery Research, Boston Children’s Hospital
10. New partnership models will share risks, marry strengths
Academic, industry and philanthropic organizations will explore new, creative partnership models based on risk sharing. Several weeks ago, Royalty Pharma purchased the Cystic Fibrosis Foundation’s royalty rights for Kalydeco, a drug developed by Vertex Pharmaceuticals with the foundation’s support. This news is a reminder that alliances (sometimes between improbable partners) can create powerful synergies and resources to bring to market much-needed therapies that are too costly or risky for any one group to develop on its own. I have seen this trend in my work leading Boston Children’s Technology Development Fund (TDF).
One of our most successful projects—the MAPS vaccine platform—is the product of a multiparty collaboration. Recently licensed to startup Affinivax, MAPS was supported not only by TDF, but also by the hospital’s Translational Research Program and the Bill & Melinda Gates Foundation. Other projects under development are leveraging resources and expertise from multiple academic centers, foundations and pharmaceutical companies. This trend will continue and even increase in 2015. —Monique Yoakim-Turk, PhD, Partner, Technology Development Fund and Associate Director, Technology and Innovation Development Office, Boston Children’s Hospital
As pharmaceutical companies retreat from internal research and development efforts in the neurosciences, we will see more focus on collaborative program-building with academic medical centers to maintain a broad neuroscience portfolio. The need for exploratory programs rooted in human genetics will lead to programs that target genetically defined, well-characterized patient populations at academic medical centers where patients are seen in sufficient numbers to support clinical trials.
Tissue samples from which pluripotent stem cell lines can be induced will facilitate the development of human neuron-in-a-dish models of neurodevelopmental genetic disorders and phenotype-based drug screens. As new targets emerge from these screens, we will see hybrid drug discovery programs where academic partners contribute biology and disease expertise, while industry partners contribute expertise in medicinal chemistry, pharmacokinetics and drug disposition, formulation and preclinical toxicology. —Robin Kleiman, PhD, Head of Preclinical Research, Translational Neuroscience Center, Boston Children’s Hospital
11. The year of 3D printing in medicine
In 2015, the 3D glasses of yesteryear will be replaced with the most realistic 3D imaging possible—actual 3D-printed models of tumors, aneurysms and even whole organs from specific, individual patients for better visualization of disease. A combination of higher resolution printers, faster production times and increasingly detailed patient imaging has now made construction of overnight “bespoke” clinical models practical.
Building models goes beyond just making a copy of a scan in 3D. With physical models, clinicians can take scans from any patient, change the size, orientation or color of a part of the body and see things in ways not possible on a screen. They then can practice surgical approaches, teach trainees or safely rehearse difficult techniques with real tools.
Combined with advances in tissue engineering, 3D printing will allow doctors and scientists to custom-build new or replacement parts for patients. We will truly be able to build our patients a better future. —Edward Smith, MD, Director, Pediatric Cerebrovascular Neurosurgery, Boston Children’s Hospital
We have just scratched the surface of capabilities of 3D printing technologies in medicine, ranging from Organovo’s bio-printed liver tissue to the Boston Children’s Simulator Program’s life-like 3D models for preoperative planning. Just in the last year we’ve seen dramatic improvements in prosthetics and surgical outcomes from knee cartilage to tracheomalacia. We’ve also seen an explosion in the creative use of consumer 3D printing technology for rapid prototyping of medical devices. For example, accuTrach, a team formed out of this year’s Hacking Pediatrics event, wants to create on-demand custom tracheostomies for children.
2015 will bring the next generation of 3D printing using new materials and combinations of materials. Printing resolution will jump dramatically, down to the nano-scale. The accompanying software will become more powerful and easier to use, such that novices will be able to easily print any idea they come up with. Biocompatible matrices for tissue engineering and nano-robots with medical applications will soon become a reality. —Michael Docktor, MD, Clinical Director, Innovation Acceleration Program, and Director of Clinical Mobile Solutions, Boston Children’s Hospital
12. Soft robotics and conductive fabrics enter medicine
Robotics has been in the health care environment for many years, in forms ranging from robotic assisted surgery to food tray delivery. In 2015 we will see a new class of robotics expand into health care: soft co-robotics—robots created with soft materials that cooperate with humans.
Soft co-robotics leverages wearables and conductive fabrics and brings these technologies to another level, incorporating conductive fluids with sensors and actuation that can adapt to changing environments. The resulting robots will be able to perform like an artificial muscle, opening endless possibilities for science and health care. Think, for example, of an active brace that stiffens and softens based on the user’s mobility requirements.
It will also be exciting to see soft co-robotics “hacked” by combining the technology with low cost materials and 3D printing technology for rapid prototyping. —Kate Donovan, MBA, Education Innovation Technology Coordinator for Medical Patient Services, Boston Children’s Hospital, and co-founder of Hacking Pediatrics
13. Convergence across platforms
Stand-alone biological research will increasingly give way to interdisciplinary collaborations: biologists and engineers, biologists and mathematicians, biologists and computer modelers. More work will be done on the computer and less at the bench. —Bruce Zetter, PhD, researcher, Vascular Biology Program, Boston Children’s Hospital
Big data, based on mining medical records, will begin to produce research data and lead to “real life” drug trials. —Walter Kaufmann, MD, co-director, Fragile X and Down Syndrome programs and clinical co-director, Translational Neuroscience Center, Boston Children’s Hospital
14. Mixed progress ahead for cancer
Increasing progress will be made on a few cancer types, such as breast cancer and prostate cancer where funding is greatest, whereas progress on some of the most aggressive tumors such as lung cancer, liver cancer, ovarian cancer and pancreatic cancer will continue to lag behind. —Bruce Zetter, PhD
15. Funding: a mixed forecast
Congress will see the light of day and increase the NIH budget in order to prevent the U.S. from losing its position as a global leader in health research, a threat born out by analyses from the NIH’s Fogarty International Center. —Bruce Zetter, PhD
There should not be any meaningful improvement in the federal funding climate. A “valley of death” still separates biomedical discoveries from the patients that need them. Leadership in research institutions will need to fill the gap and rethink how they look at the return on investment in research. The old days of looking at costs versus revenues as the main measuring stick for research are over. These measures never correlated with clinical relevance or clinical applicability. —Dario Fauza, MD
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