When the Biomedical Industry Can’t Prioritize Diseases, Private Money Can Save Lives

Type 1 diabetic Omar Elbarky, 8, holds his insulin pump at his home in Spring Hill, Tenn. AP

My daughter was six when she was diagnosed with type 1 diabetes. Like others parents confronted with such life-altering news, I threw myself into learning everything about the condition. What I found was that this autoimmune disease has no cure, requires relentless glucose and insulin monitoring and can quickly lead to serious health complications—even death—if not properly managed.

I also learned that my daughter was not alone. Nearly two million people in the U.S. suffer from type 1 diabetes (T1D), and millions more are affected worldwide. We, the global community affected by T1D, need better treatments and a cure.

With so many affected, you might assume drug and medical device makers are racing toward solutions. But for many pharmaceutical and medical device companies, two million is a small figure. (Type 2 diabetes, by contrast, affects at least 25 million Americans.) Given the expense of research, development, and bringing a new device or drug to market—which can easily reach $1 billion—a relatively small potential market discourages investment by large companies.

David Panzirer

David Panzirer is a parent of a child with type 1 diabetes and a trustee at the Helmsley Charitable Trust, the largest private foundation funder of T1D-related research, treatment and support services in the nation.

Fortunately, when the market doesn’t provide the right incentives, philanthropic groups have a unique opportunity to spur advances in treating diseases that affect millions but might otherwise be ignored. We all saw how the ALS Association, with its viral Ice Bucket Challenge, quickly amassed more than $100 million to expand its fight against a once little-known, but deadly, disease. And in a sign of the flexibility and resources unique to philanthropic groups, the Gates Foundation recently donated $50 million to fight Ebola in Africa, a sum that eclipses the commitments of many large western nations.

In these situations, from rare diseases to mass epidemics, philanthropic donors and disease advocacy groups can catalyze the development of new therapies and incentivize governments, pharmaceutical companies, and medical device makers to step in and support new treatments.

The benefit of partnering with private companies and government is not just efficiency or economics: patient voices and early stage funding have the power to minimize risk in the development process and create new markets. Encouragingly, foundation giving has surpassed its pre-recession peak, and funders have become less passive and more results-oriented in their grantmaking. As a result, opportunities for collaborations are greater than ever.

Let me give you an example. For five years, the philanthropy I’m a part of, Helmsley Charitable Trust, has worked with the National Institutes of Health and the Juvenile Diabetes Research Foundation (JDRF) to accelerate the creation of new technology for T1D patients. In one project, called the bionic pancreas, investigators supported by our organizations integrated smartphones with continuous glucose monitors and insulin pumps to create the first generation of wearable systems that automatically regulate blood glucose levels. Through several studies during the last two years, including one recently reported in the New England Journal of Medicine, the device has demonstrated strong potential to be a game-changer. By freeing patients and caregivers from the 24/7 cycle of manual blood glucose monitoring and insulin injections, it has the potential to help those with T1D live longer, healthier, normal lives.

The financial model that enabled researchers to develop the bionic pancreas is noteworthy, too. By embracing the bold ideas of those like Boston University engineer Ed Damiano, whose son with T1D inspired him to create the bionic pancreas, we have accelerated innovation. And through early-stage funding, philanthropy and government have been able to de-risk the financial burden of creating a new product, increasing the likelihood that a medical device company can bring it to market.

Kalydeco

And there are many other examples. For instance, philanthropy helped create the cystic fibrosis drug Kalydeco. Through three phases of trials, as well as research investments with other leading pharmaceutical companies, the Cystic Fibrosis Foundation successfully accelerated the discovery and development of the new drug and earned FDA approval in 2012. Over the course of a decade, philanthropy was able to align itself with government and a pharmaceutical company, Vertex, to invest millions of dollars in research and establish a new treatment for an underlying genetic cause of a disease that affects tens of thousands of children and adults.

In too many cases the standard path of creating new treatments through FDA approvals and the reimbursement process is slow and costly.

Even when a cure to a chronic illness can seem far off, and when the market can’t accommodate the necessary R&D to make meaningful strides, there is still an urgent need to improve the lives of those who suffer from these diseases. Type 1 diabetes is often deceiving and if not well controlled can take a slow but devastating toll on those affected. It can lead to blindness, amputations, heart disease, and kidney disease. Diabetes (types 1 and 2) is the seventh leading cause of death in the U.S., and the risk of premature death for people with diabetes is nearly double that of similarly-aged people without it.

In too many cases, however, the standard path of creating new treatments through FDA approvals and the reimbursement process is slow and costly. Only industry can fund large enough trials to secure a sign-off from the FDA, and only industry can produce drugs and devices at a scale enough to be widely available and affordable.

With a shift in the way we think, however, philanthropy can be a strategic player in the process by partnering with nonprofit organizations and private companies, funding research, and taking on under-prioritized projects, as Helmsley has done with Medtronic, Dexcom, Xeris and other companies. More than funders, we can be partners building a bridge from academic research to industrial production. We can also leverage internal and external expertise, resources and support, mobilize the power of networks of grantees, and serve as unbiased patient advocates throughout the development, regulation and reimbursement processes.

This summer, the bionic pancreas project underwent another important trial, as the investigators tested the system on active T1D kids ages 6-11 in the open air of a summer camp. Parents across the country were eager to learn how their children can participate, having read about the promising results from past studies.

Still, even with these successes, there is a great deal of work to be done in system and product development, education and preparation for commercialization. For example, only 30 percent of people in the U.S. with diabetes use an insulin pump, and less than 10 percent use a continuous glucose monitor, technologies integral to the bionic pancreas. By working to expand awareness and the pool of potential system users, our hope is to create a market while, most importantly, improving health outcomes for those with this disease.

We’re moving in the right direction. When the market alone can’t drive medical advancements, philanthropy plays a critical role for those affected by acute and chronic diseases. These creative, collaborative partnerships are driving innovation and providing hope for millions of Americans to live happier, more fulfilling lives.

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