This article originally appeared in Medium on May 23, 2016.
Last year, at a board meeting, I presented a video about the value of medical technology. The video focused on a mom struggling with caring for a toddler with type 1 diabetes. I distinctly remember a moment when the young child looks incredulously at his mother, who is supposed to protect him, as she approaches him with a needle.
Little did I know, my family was about to be faced with a similar reality. Mere minutes after presenting that video, my wife called to tell me our son, Chris, who had been suffering from fatigue, weight loss and unusual thirst, had just been diagnosed with type 1 diabetes. The moment bordered on the surreal.
Having spent the bulk of my career in life sciences, I’ve met hundreds of patients in my work to improve patient health and have become close with many of them and their families. I previously thought I had a pretty good handle on what patients go through every day as they interact with doctors, nurses, insurance companies and the various other pieces of the health care system. It turns out I really didn’t have a clue about what it’s like to care for someone with a chronic medical condition.
Type 1 diabetes occurs when the body’s immune system mistakenly attacks its own cells in the pancreas, eliminating its ability to produce insulin, a hormone we need to convert food into energy. Chris has to check his blood sugar throughout the day and take injections of insulin before every meal and at bedtime, and make sure that he has had enough food to match his insulin dosage and activity level — and vice versa.
If you have preteens at home, you understand discipline and vigilance are not top on the list of traits often used to describe their behavior. This is the time in life when kids should be carefree, but Chris’ favorite foods like pizza and ice cream make staying on top of regulating his blood sugar particularly challenging. Participating in sports adds another layer of complexity.
Chris' diagnosis has without a doubt deepened my professional commitment to making sure medical progress continues to save and improve lives. Thanks to Chris’ treatment team, the vigilant attention of my wife and the medicines he administers, Chris is well-equipped to confront the challenges of living with type 1 diabetes. Of course, this hasn’t always been the case.
Scientists figured out how to create insulin from animals in the 1920s, but it wasn’t until the 1980s that more advanced insulin, made from human proteins, became available. Because they were made from human proteins, they were less likely to cause allergic reactions, and safer for patients. This was a life-saving innovation, and even today, researchers continue to innovate and deliver more stable forms of insulin for patients with type 1 diabetes.
Over the last half-century, new medicines, advanced technologies and better scientific understanding have truly revolutionized how we treat diseases. Millions of patients now live longer and healthier lives because of this progress.
But that’s not the exciting part. That’s the history. What I’m most passionate about is what is yet to come.
America is on the cusp of a golden era of medical innovation. We have the building blocks to revolutionize the treatment of costly and debilitating diseases like cancer and Alzheimer’s disease. Our ability to harness the human body’s natural defenses to fight disease is getting better and better, as is our capacity to create medicines tailored to an individual’s genetic makeup. For patients like Chris, the science is moving toward smart insulin, which turns on and off as needed to ensure greater glucose control throughout the day, or new oral medicines that would prevent the need for regular needle injections. And I’m confident that someday we’ll restore the function of the pancreas through beta cell replacement therapy.
We are also bringing patients into the process of making medicines, honing our understanding of how treatments impact people’s daily lives and making them easier to take.
But in order for this golden era to become a reality, we need sound policies in place to foster it. We need policies that make the patient a priority, improve how our health care system works and make it affordable. That’s why my organization recently introduced new recommendations and solutions to ensure our policy environment continues to support delivering these innovative treatments to patients like my son.
Modernize the process
It still takes too long and costs too much to develop a medicine, test it and get it to patients. The scientific process for making medicines is far more advanced today than the tools the U.S. Food & Drug Administration (FDA) has to evaluate those medicines. The FDA should be empowered to use the latest technologies and methods, such as biomarkers, real world evidence and patient-reported outcomes. That would speed the delivery of innovative treatments to patients, enhance competition and keep costs down.
Get more value out of health care
Current rules prohibit discussions between insurance companies and biopharmaceutical companies about medicines that are on the horizon. This doesn’t give insurers the opportunity to plan their budgets, causing uncertainty about insurance premiums and other planning tools. Similarly, once a medicine is available for patients, current rules prevent manufacturers from sharing research — including FDA-requested research — with insurers and doctors, including the results of trials, data on specific patient populations use of the medicine and more. In order for biopharmaceutical companies to become partners — rather than simply vendors — we need to be able to communicate with payers about the performance of our products.Removing these barriers will promote efficiency and affordability and ensure the right drug is getting to the right patient at the right time.
Engage and empower consumers
A well-informed consumer is an engaged and empowered patient. And yet consumers too often are frustrated by hurdles that make it hard to access information about their insurance benefits and out-of-pocket health care costs. Patients need to know whether the physician or hospital they visit is in network. They need to know whether the medicine they need is covered or if all medicines for their disease are placed out of reach by their health plan. We need to make this information available to patients. In addition, the most vulnerable patients should be protected by commonsense rules that prevent discrimination and pull down barriers that prevent them from accessing the medicines they need.
Address market distortions
The U.S. health care system is largely market-based and has worked well over time, but more can be done to help it work even better. We need to address policies that distort the market-based system, such as the 340B program or risk adjustment systems that do not pay plans appropriately for high risk patients. Helping the market work at its best can help preserve the safety net and improve affordable access to medicines for patients.
I've always been passionate about fostering a health care system that works better for patients, and my son's diagnosis has only strengthened that conviction. As grateful as I am for the treatments Chris has available to him today, I'm hopeful that the future holds even more promise. With the right policies in place to encourage and advance innovation, I see a bright future ahead – for Chris, and for all patients.