Securing longer, healthier, happier lives—securing better health for all Americans—is the fundamental goal of the vision President Trump has for our healthcare system.
As Prepared for Delivery
Thank you, Peter [Saltonstall], for that kind introduction, and thank you all for having me here today. It's an honor to be with you.
We face daunting challenges in American healthcare, but it is heartening to look across this room and see so many passionate people who are determined to conquer the rare diseases that cause too much suffering for too many Americans and their families.
The work you do as researchers, stakeholders, and advocates is vitally important to helping us understand, combat, and, one day, end rare diseases.
I know that many of you are here today because you suffer from a rare disease or you have a loved one, or someone very close to you, who does.
Raise your hand if that represents you.
I'd like to personally thank you for the remarkable perseverance you demonstrate every day.
Your hope and your tenacity are contagious. I think you all deserve a round of applause for the commitment and courage you show.
Today, I want to talk to you about the Trump Administration's commitment to ensuring that those suffering from rare diseases receive the support, attention, and care they need.
Securing longer, healthier, happier lives—securing better health for all Americans—is the fundamental goal of the vision President Trump has for our healthcare system.
He understands the vital importance that health holds for every American, and that is especially true for Americans suffering from rare diseases and the families who support them.
The President has a particular vision for healthcare: a system with affordable, patient-centric, personalized care, a system that puts you in control, provides peace of mind, and treats you like a human being, not a number.
That kind of personalized, patient-centered system can be essential, as I'm sure you all know, to managing and eventually curing so many rare diseases.
Such a system will provide you with the affordability you need, the options and control you want, and the quality you deserve.
The President has a clear approach to delivering on this vision: He's promised to protect what works in our healthcare system, and fix what's broken.
When it comes to rare diseases, that means protecting the parts of our system that undergird innovation toward the treatments and cures we need, while trying to fix the market failures that have led some diseases to be neglected for far too long.
There are three cross-cutting platforms where we're working to deliver on the President's vision: reforming how we finance care, delivering better value from that care, and improving health in specific, impactable areas.
As you'll see, each area is important in our battle against rare diseases. I'll touch on each of these areas in turn.
First, we have to think about how our financing system can protect those with serious and rare illnesses.
As many of you know, having a rare disease can be extremely expensive.
Effective treatments are often hard to come by, requiring years of expensive maintenance therapies. When successful therapies are developed, they're not cheap. We need to ensure that Americans who suffer from rare diseases have ways to finance their care—while also making sure that our financing system can support innovation toward the cures we need.
In part, that means keeping the promise President Trump has made to Americans with preexisting conditions.
He's been unequivocal: We will always protect Americans with preexisting conditions—a guarantee we will maintain at the federal level. That's not changing.
We want to protect what works in our system of financing.
Medicare and private insurance have made it possible for Americans to receive the most cutting-edge medical treatments, typically faster than anyone else in the world.
President Trump is dedicated to protecting Medicare and private insurance because they work—and we're going to continue making them work even better.
Another key financing challenge for rare diseases is how to handle high-cost therapies. We have already seen our system struggle with high-cost therapies for some more common diseases, like cures for Hepatitis C.
State Medicaid programs in particular struggled with the fact that they wanted to provide the cure, but simply couldn't afford providing it to all the patients who needed it, all at once. State budgets simply couldn't handle it.
So CMS actively worked with states on these challenges, because we need a financing system that supports these exciting public health developments.
CMS has approved waivers for two states to use subscription-based models for Hepatitis C drugs, and we continue to look at ways that we could offer flexibility, including around price-reporting mechanisms, to support innovative ways to finance high-cost, high-value drugs.
This same kind of innovative thinking may eventually be needed for financing the cures of rare diseases, too.
Lots of discussion around healthcare in Washington often focuses on financing, but the President's vision is broader than that: He also seeks to improve the actual delivery of care, and secure better value from that care.
This takes a number of forms, from transparency and health IT reforms to put patients in control of their own care, to paying for outcomes and bringing down the price of prescription drugs.
There can also be tremendous value created through scientific and medical breakthroughs. New curative therapies often mean that an individual can live a better, longer life, while eliminating the need for high-cost maintenance therapies.
To secure that value, we're building on the work that CMS and FDA have already been doing to streamline how we go about drug and device approvals, and how we reimburse for these technologies.
You just heard from Dr. Sharpless about the strong commitment FDA has to these efforts, particularly as they relate to rare diseases.
CMS is hard at work on the same goals, accelerating coverage for the latest technologies in Medicare.
We know there's potential for curative therapies for rare diseases—because we've already seen some recent breakthroughs.
But we've also seen them come at an incredibly high cost.
A value-based approach in this area may sometimes mean spending a lot of money up front to find and fund a cure, instead of paying for maintenance and management.
But that is not a reason to accept excessive, abusive pricing of drugs that patients need, and President Trump is committed to introducing more competition, negotiation, and better incentives throughout our drug pricing system.
Government has a major role to play in helping finance treatments and cures for rare and neglected diseases.
It's well understood that drug and medical device companies have little financial incentive to spend a lot of money to produce a drug or device that's only expected to benefit a very small segment of the population—even when it may mean years and years of better life for that population.
That is one reason why the National Institutes of Health has been so dedicated to combating rare diseases for decades.
It has been a longstanding priority and a point of pride for the institution.
NIH's work has helped us better understand the underlying genetic foundations of disease, opening up the promising field of gene therapy.
That work continues to grow in ambition and scope, as we advance NIH's All of Us project, which now has an unprecedentedly diverse group of nearly a quarter-million Americans enrolled.
NIH has also invested in translational science, to help develop the knowledge we need about rare diseases to support clinical trials.
Earlier this month, NIH's National Center for Advancing Translational Sciences made new grants to continue the Rare Diseases Clinical Research Network, which works all across the world to understand the progression of rare diseases and develop treatment approaches.
We're also proud to support the gathering of data through newborn screening programs, like those supported by the Health Resources and Services Administration in cooperation with state governments.
The sooner we identify cases of rare diseases, the earlier babies can be treated, potentially preventing death or disability and enabling them to reach their full potential.
The final platform of the President's vision I mentioned is impactable health areas, and I want to discuss one rare disease in particular, sickle-cell disease, where we believe we can make a large impact, quite soon.
Making a meaningful impact on this terrible disease will require thinking across each of the platforms I mentioned: reforming financing, value-based care delivery, and focusing on particular challenges.
This chronic, inherited, and debilitating disease afflicts approximately 100,000 Americans, disproportionately African-American and Hispanic populations, along with millions around the world. Beginning in childhood, the disease can cause severe disabilities and recurring, unpredictable episodes of crippling pain.
Caused by a single genetic mutation, sickle cell disease results in red blood cells' acquiring an abnormal crescent shape during low oxygen conditions. These cells subsequently cling to the walls of blood vessels, blocking the flow of blood and depriving the body's tissues of oxygen.
Along with repeated episodes of excruciating pain, SCD can cause infections, lung damage, blindness, heart and kidney failure, and strokes. Depression is also common in patients with sickle cell disease.
But, while sickle cell disease has been neglected for far too long, today, there are many, many reasons for hope.
In fact, it is one of the single most promising areas for biomedical research and public health impact we have in front of us over the next decade.
In 2018, President Trump signed a bill to reauthorize SCD prevention and treatment programs and provided funding for continued research for SCD and other similar diseases.
His administration has made the disease a priority. At HHS, we've set a goal of extending the lives of Americans with SCD by 10 years, within 10 years—and we're committed to reaching the ultimate goal of a cure for the disease.
Through an HHS Sickle Cell Disease Workgroup, we're looking at how to address the challenges patients face when transitioning from pediatric care to finding providers who treat adults with the disease.
We're also looking at the expansion of data collection efforts, such as the Sickle Cell Data Collection System at the CDC.
Sickle cell, as I mentioned, is also a global health challenge.
More than 300,000 babies are born with it in sub-Saharan Africa every year, and 50 to 80 percent of them will die before the age of 5.
We've begun to work with African health leaders to understand how we can help deliver better access to the treatments we have, and understand how an eventual cure could be delivered around the world.
A value-based approach to care delivery, including for sickle cell, means greater collaboration between doctors, researchers, and patients—especially patients in rural areas, underserved areas, or areas with few SCD patients.
Today, only 1 in 4 patients with SCD receives the standard of care for the disease, often because providers don't recognize the disease or have the expertise needed to provide proper care.
That's why we're also interested in using innovations like telehealth to spread expertise about the standard of care for SCD. This focus on improving care delivery is a key piece of the President's vision.
But we're going well beyond just better use of the treatments we have today. We are now seeing unprecedented levels of research, across HHS and aligned institutions, on SCD treatments.
Over the past few years, the FDA has also begun to review a number of new investigational drugs to combat SCD. In the last century, there have been only two new drugs approved to treat SCD in the United States. But that's starting to change.
As of today, there are about 40 drugs to help patients better manage pain and other complications currently being tested in clinical trials.
Even more excitingly, the National Heart, Lung, and Blood Institute has launched the Cure Sickle Cell Initiative, to accelerate gene therapies for the disease.
The institute believes that a cure for sickle cell is within our reach over the next five to ten years.
I've seen with my own eyes how close we are: I was able to meet a patient whose blood was now sickle-free as a result of the gene therapy he'd received from an NIH clinical trial.
This is an incredible triumph of medicine—and you all should be proud of how the rare-disease community has helped get us to this point.
But, as you all know, there are still challenges.
In particular, gene therapies have typically carried unprecedentedly high prices—and the same will likely be true for a cure for sickle cell.
Progress on sickle-cell disease, and so many other diseases, must prompt us to think about how we are going to finance the delivery of these cures.
The cures are coming—and thank God they are. But when they arrive, we must be ready to get them to the patients who desperately need them.
All of the actors involved—HHS, private payers, innovators, legislators, and patient advocates—need to be thinking about how to build a system, together, that can support these cures.
The whole point of a health insurance system like we have is to ensure that, if you're struck by a serious illness, let alone one that is rare and highly unlikely, our system is there to care for you.
A healthcare system isn't worthy of its name if it cannot provide the care needed by those who are struck by the most uncommon, most serious illnesses.
So, all across HHS and the Trump Administration, we are dedicated to preparing our system to support this exciting new era of innovation.
The particular focus I've laid out for sickle cell demonstrates President Trump's commitment to making progress on some of the most serious health challenges we face.
He knows that a comprehensive approach, with collaboration from many of you in this room, will heal and change lives for many Americans—and for many beyond our borders.
So I want to conclude by thanking you for the work that has been done by the rare disease community to get us where we are today.
As someone who has worked at high levels of healthcare for almost two decades now, it is common to know both hope and despair: the wonderful hope provided by new cures, and the occasional despair that comes when prospective treatments don't pan out.
But we all persevere because of you: those who are on the front lines of confronting diseases in your families.
You give us hope—and, in return, we're committed to doing everything we can to provide you with reasons for hope, too.
Some of you may have heard NIH leaders refer to the NIH as the National Institutes of Hope.
Well, when you put all of HHS together, we can do even better: we have two Hs. We're all about hope.
So thank you for joining with us in hope, thank you for your commitment to such important work, and thank you for having me here today.
