Medicine for the future: Innovation and patient access

By Debbie Hart and Robert Goldberg
New Jersey | May 17, 2018 at 9:00 am
Op-Ed

Kathleen Arntsen, the CEO of the Lupus and Allied Diseases Association, contracted shingles on her face over four years ago. The virus spread to her right eye, where the inflammation caused incredible pain and constant pressure.

Over several months, she tried several medicines — which only made the pain and pressure worse and led to glaucoma and a cataract. Then, she tried a sample of a new drug that reduced the eye pressure within days.

Kathleen’s health plan refused to cover the new medicine. She had to fail first with two more types of eye drops. Those drops failed her indeed. In fact, the inflammation worsened and now she is blind in the affected eye. As Kathleen notes: “The insurer’s decision was not only bad for my health, but penny-wise and pound-foolish for the company. Instead of covering the initial drops that my physician prescribed for less than $200, the carrier has now covered years of numerous specialty appointments, diagnostic tests, procedures, several devices and lenses costing over $15,000 to date.”

Unfortunately, the barriers patients face in obtaining personalized medicine are being undercut by the way medicines are covered and paid for. Currently, entities called pharmacy benefit managers, or PBMs, administer prescription drug benefits for the 85 percent of all Americans whose medicines are covered through health plans. And, in an effort to maximize the use of medicines that cost the least or generate the most rebates, PBMs are imposing high out-of-pocket costs, prior authorization and step therapy on the sickest patients with the most complex diseases. As a result, in far too many cases, the health of patients is at risk and health care costs are higher.

At the same time, even as PBMs, insurers and employers benefit from rebate dollars paid by biopharmaceutical companies, more and more patients are being forced to pay up to 50 percent of the retail price of a treatment. While passing rebates on to patients is a laudable act, that alone is not enough. It still begs the question as to whether the right patient is getting the best treatment at the right time. Thankfully, there is a surge in the number of new therapies, devices and diagnostics targeted to specific patients or disease mechanisms. But that means there will be pushback on the cost of medicines and a growing demand by payers, physicians and the patients who are going to use the medicines for evidence that new treatments improve health and well-being.

Today, the quickening pace of transformative, scientific discoveries is resulting in increasingly more disease-altering treatments for patients with unmet medical need. Based on an understanding of specific mechanisms and pathways that shape our individual risk of disease and response to medicine, such innovations can and will be used “to enhance health, prevent disease, track its development, intervene early and manage disease most effectively if it occurs,” according to Dr. Ralph Snyderman, James B. Duke Professor of Medicine and chancellor emeritus of Duke University. Medical innovation that once took centuries to advance, and more recently decades, are progressing through the system in only a few years, or even months. The list is growing, from checkpoint inhibitors and chimeric antigen receptors, or CAR T cells, to PD-1/PD-L1 inhibitors.

Our understanding of disruptive science continues to build on itself, leading to more unconventional treatment approaches, and better results at a never-before-seen pace. Significant advances in technology are allowing us to more accurately and efficiently prevent, diagnose and treat diseases. People are living longer, better and healthier lives because of the continuous stream of medical innovation driving new discoveries at a seemingly breakneck speed. Indeed, since 2000, new cancer drugs contributed to a 20 percent decline in cancer death rates, a 43 percent decrease in lost productivity and a 70 percent reduction in the days spent in hospitals.

However, speed slows and the pace of advancement stalls when medical innovation moves faster than the health care delivery system’s ability to adapt to new scientific realities and possibilities. Successfully leveraging our most important breakthroughs requires efficient regulatory processes and collaboration between payers, regulators and innovators to identify flexible pricing models that allow for continued investment in cutting-edge science and unencumbered patient access, that will increase competition and choice in the marketplace so that all stakeholders will benefit.

Last December, BioNJ brought together patients, biopharma companies, insurers and others to discuss what specifically has to change. The participants concluded that innovator companies must do more and invest more in matching people to treatments that work best for them. Health care insurers must do more to eliminate all hurdles that encumber patient access. PBMs must do more to improve on outdated formulary structures, to ensure that the right patient is getting the best treatment at the right time, and that the decision is ultimately being driven by the doctor together with the patient. Regulators must do more to accelerate the approval of innovative medicines, expanded label opportunities for rare and debilitating diseases and support discussion with payers around real-word evidence.

Moving from basic science to medical discovery and translating that into clinical practice is hastening because we’re building relentlessly on our successes and using knowledge to advance from idea to breakthrough at a breathtaking pace. The question remains: Can we evolve our approach to care quickly enough to maximize benefit to patients and society and reap full benefits afforded by today’s medical innovation?

Over the next year, BioNJ will be collaborating with some of the most innovative and entrepreneurial health care leaders in our state to reduce barriers to access and demonstrating that a prospective solution delivers better value. We invite everyone to join us in this exciting venture. The time to start is now, “Because Patients Can’t Wait.”

Debbie Hart is CEO and president of BioNJRobert Goldberg is co-founder and vice president, Center for Medicine in the Public Interest.

ROI-NJ Staff | editorial@roi-nj.com | @ROINJNews