Medical Breakthroughs Require a New Kind of Innovation
Sickle cell disease is a devastating genetic disorder that can lead to stroke, organ damage, pulmonary hypertension, and even heart failure. More than 2 million Americans carry the genetic mutation that leads to sickle cells developing in the body, and yet research for treatment of the disorder is chronically underfunded, and it receives far less attention from the media and research community than other genetic disorders. While it’s unclear exactly why, some have noted that this could be yet another example of health disparities in the U.S. today, as the disorder primarily affects African Americans.
Racial minorities continue to be underrepresented in clinical trials for cancer treatments, and access to care remains an overwhelming challenge for low-income black communities.
Still, recent medical breakthroughs hold promise for improving the health of all Americans. Some medications have made previously fatal diseases treatable, and technological advances have reduced patient morbidity and mortality during surgery.
Now, with the advent of gene and cell therapies, diseases such as hemophilia, sickle-cell disease – both of which disproportionately affect minorities – and even rare forms of cancer, can often be cured.
The Food and Drug Administration approved the first gene therapy for an inherited disease, to treat a rare form of genetic blindness, in December 2017. Patients who had been blind their whole lives were suddenly able to see after receiving one-time injections. This is truly a miracle cure: 93 percent of patients reported at least some improvement.
Because gene therapies alter the underlying genetic cause of the disease, their curative nature, versus long-term treatment of chronic symptoms, make them fundamentally different than traditional pharmaceutical options. Of course, gene therapy represents decades of medical research and innovation, and the treatments are likely to be expensive – some have been priced at nearly half a million dollars, and others are likely to cost even more.
The current debate over high drug prices is a valid one, especially when considering that failure to cover medications disproportionately prices out minority populations. However, when examining the cost of these therapies, we must prioritize their effectiveness, value to patients, and long-term cost savings of curing a disease, rather than treating it.
For example, hemophilia, a rare disorder which causes blood not to clot and minor bruises to become life-threatening injuries, is more prevalent among black and Hispanic men than among the general population. It’s a debilitating disease that makes physical activity difficult or impossible, and costs individual patients and the healthcare system millions of dollars every year. Hospitalizations and surgeries for hemophilia patients can cost $1 million annually, without factoring in an average of $250,000 to $300,000 per year for clotting proteins to prevent internal bleeding.
A gene therapy for hemophilia is currently undergoing clinical trials, and one patient in Michigan has been symptom free since his treatment. This is significant for both the patient and his family members who may also suffer from the genetic disorder. The value of curing it, even if the upfront cost is large, should not be disregarded when considering price and insurance coverage.
The challenge is that our system is not prepared for one-time payment for medications or treatments. Historically, patients take the pill or have the surgery, and an invoice immediately goes to your insurer.
But this does not accommodate potentially high-cost, but extremely effective, one-time therapies. In order for minority populations to overcome access gaps and obtain these transformative treatments, private companies should be encouraged to find innovative payment solutions.
So far, drugmakers and insurers have focused their efforts on outcomes-based pricing, including “money back” guarantees if therapies don’t work, payments spread out over time, or an up-front payment with staggered reimbursements tied to clinical effectiveness and measures such as reduced hospital admissions. This allows insurers and hospitals to capture the long-term value of curing these deadly diseases, while focusing on what’s best for the patient.
If pharmaceutical companies and payers are given the room to innovate in the development of and payment for cures, more patients will be able to realize the benefits of these miracle treatments.
Minority patients can and should be able to be a part of the gene therapy revolution. Time will tell.
Dr. Gary A. Puckrein is President and Chief Executive Officer of the National Minority Quality Forum. The Forum is dedicated to improving the quality of health care through the creation of evidence-based, data-driven policy and initiatives.
