Clinical pharmacologist Harry Shirkey observed more than 50 years ago that children are often “therapeutic orphans” in biomedical research. Today we are witnessing a continuous 5-10 year lag in pediatric biomedical progress relative to adult populations.
In pediatric health care, approximately 90% of health resources are used by 5-10% of children suffering from complex and chronic medical conditions. In many cases, these 5,000 pediatric-only rare diseases, or “orphan diseases,” do not have a large enough market to drive intervention, which permeates both the pharmaceutical and biomedical device industries.
Over 80% of Food and Drug Administration (FDA) approved biomedical devices have no tests or indications for use in children under the age of 18. To support the development and adaptation of pediatric devices, the FDA provides exemptions for humanitarian devices (HUDs) for target groups of fewer than 8,000 individuals per year, and there are encouraging examples. Indiana-based Cook Medical has a long history of developing pediatric medical devices, starting with the Harrison Fetal Bladder Stent Set in 1997, the first humanitarian device approved in the U.S. However, 25 years later, there are only 79 approved devices and only a small fraction of them are for pediatric use.
American universities have the opportunity to serve as a development force to conduct research in therapeutic and biomedical technologies for children and adolescents. Our academic institutions can approach this in two ways: by carrying out federally funded fundamental research on these orphan diseases; and partnering with biomedical device companies to scale, modify, test and implement life-saving pediatric technologies.
For example, Purdue University and Indiana University are forming a strong partnership between engineering and medicine. The Weldon School of Biomedical Engineering at Purdue University, the Department of Pediatrics of the Indiana University School of Medicine, and the renowned Riley Hospital for Children in Indianapolis have created an interdisciplinary nexus to address the long-standing lag in diagnostic devices, technologies and in pediatric therapies. This research includes the development of wearable sensors for continuous monitoring and point-of-care diagnostics; imaging technology and advances in data processing; neuroengineering; and micro and nano devices to monitor metabolic diseases and drug delivery to children.
Take Duchenne muscular dystrophy, for example, which is a pediatric cardiovascular disease that causes a progressive weakening of the heart due to a deficiency of dystrophin, a protein necessary for the proper functioning of muscle fibers. Early diagnosis can improve life expectancy through prophylactic treatment with heart medications. To combat this ailment, and others like it, the aforementioned partnership brings together engineers and doctors to imagine the heart using non-invasive 3D and 4D methods. These are used to form custom files and records that can calculate heart function and determine important parameters that provide a more detailed look at the underlying condition. Many of these parameters are more common in a fluid mechanics course than in a doctor’s office, such as maps and measurements of forces, stresses, strains, and velocity profiles that can be extracted to predict heart conditions and prognosis that inform plans. of treatment. In some cases, structures near the heart are printed on 3D printers for further study or to communicate prognosis and treatment plan to families through 3D models. Examples like these show the benefit of collaboration between engineering and medical skills.
This combination of engineering and customization can also play a role in the development of new drugs. An example of personalization is the mRNA technology that enabled COVID-19 vaccines; it is expected to form the basis of a third of all new drugs by 2035 and will be applied to develop personalized cancer vaccines capable of targeting patient-specific tumor mutations. Personalization implies that medicines should be produced in much smaller volumes for groups of patients with similar needs. Another example is the portable and modular production of medicines; these can be transported and used anywhere. The megatrend of miniaturization is finally reaching pharmaceutical manufacturing.
Unlike some innovations, however, medical engineering breakthroughs cannot simply blossom in someone’s garage, due to the highly regulated environment and vital nature of their end products. The disruptive changes in personalization, miniaturization and automation that are on the horizon for the pharmaceutical industry were the focus of this month’s inaugural Indiana Life Sciences Manufacturing Summit, which was attended by leaders such as Indiana Secretary of Commerce Brad Chambers. My native Indiana traces its strong legacy to pharmaceutical manufacturing in the 1950s, when Indianapolis-based Eli Lilly and Company helped end the American polio epidemic. Medicines and medical devices are still the largest export category for Indiana, totaling more than $ 11 billion annually. Indiana also ranks second in the United States in world life science exports and offers the highest per capita concentration of life science jobs.
We need more of these conversations bringing together state and local government, industry leaders, university researchers and community college educators. The goal is to forge global solutions with regional sources and the workforce development needed to keep pace with onshoring and acceleration of medicine and medical device technologies. Such an ecosystem of innovation in engineering medicine will be a key part of a long-term strategy to strengthen U.S. manufacturing sovereignty and competitiveness.