Not everyone in the clinical space is familiar with health economics and outcomes research (HEOR). Dr. Jay Jhaveri, lead investigator for Ferring Pharmaceuticals, has a background in clinical medicine. His residency was in urology and minimally invasive urologic surgery. As a practicing physician entering the clinical space, HEOR was a new concept for him.
“I came into the pharmaceutical industry about four years ago,” says Jhaveri. “The first time I teamed up with our HEOR team, they had to explain to me how clinical outcomes were used to emphasize financial outcomes.”
Jhaveri explains that HEOR is an attempt to put a dollar value on a patient experience. For example, in the oncology space, those experiences could be tied to death, overall survival, development of a metastatic disease, or any other condition that impacts a patient’s life.
When a pharmaceutical company gets a drug approved, the next step is typically getting payers to agree to cover it. That is where HEOR can come into play.
“When we talk to companies like Aetna, Humana, and UHC, they will ask very similar questions,” says Jhaveri. “They want to know why the drug exists, how it fits into the treatment paradigm, what is the overall cost of the disease, and why they should make the treatment available to millions of their patients. A good HEOR may help us to answer those questions.”
Create An HEOR
Jhaveri wanted to determine the cost of nocturia to the U.S. economy. Nocturia is a medical term that refers to excessive urination at night. Unfortunately, no HEOR existed for nocturia. For that reason, Ferring decided to produce its own.
To get the real-world information it needed on the prevalence of the condition, the company turned to the National Health and Nutrition Examination Survey (NHANES). NHANES is a large and representative survey of a U.S. cross-sectional, non-patient population. It is sponsored by the Center for Disease Control and surveys 5,000 individuals per year. The survey combines interviews and physical examinations to assess the health and nutritional status of adults and children.
One part of the survey contained questions related to urinary symptoms. The findings suggest that timely diagnosis, followed by appropriate nocturia management, may alleviate both adverse health-related consequences as well as associated economic ramifications to patients and society.
Ferring looked at data for the years 2005 through 2014. The data encompassed approximately 51,000 people. Of those, 22,300 met the study criteria. The company decided, for the purposes of its study, that a patient had nocturia if they got up to urinate two or more times per night. The study found that 24.4 percent of the individuals examined had nocturia. Additionally, the individuals completing the survey were not known to have the condition and were not being treated for it by a physician.
“That was a measurable outcome,” says Jhaveri. “People either urinated more than twice a night or they didn't. Using that outcome, we were able to quantify how many people have nocturia. We then used that information to determine the economic losses related to it.”
A Huge Expense
Using the NHANES data, study investigators were able to determine that patients who urinate two or more times at night incur up to $62.9 billion more in direct expenditure to the healthcare system. They also incur up to $151.7 billion more in indirect costs, for a total cost of $214.5 billion.
“This is a problem that is very important to patients,” says Jhaveri. “Getting up two or more times a night is a nuisance. It creates sleep fragmentation and causes them to be more tired during the day. People who urinated two or more times at night were associated with 60 percent more hospitalizations and 19 percent more outpatient visits. This is where the cost figures come from.”
Once Ferring ascribed a dollar value to the cost of a single hospitalization and a single outpatient visit, it was able to create a dollar value for everyone who experiences nocturia. The direct costs were measured by hospitalization and outpatient visits while the indirect costs were attributed to work productivity, work loss, absenteeism, and presenteeism.
The costs associated with lost productivity were also staggering. Ferring discovered that workers who experienced nocturia produced 3.91 less hours of work per work week. That is almost ten percent of their total work week, indicating that these patients are experiencing issues at work. Forty six percent were less likely to report being in “very good” or “excellent” health. Those with nocturia were also 30 percent to 35 percent less likely to be employed.
A Cost-Effective Treatment
Although the economic costs of nocturia are quite high, the cost of treating it is not. For patients with nocturnal polyuria, one of the causes of nocturia, treatment is similar to that for patients with an overactive bladder. Ferring calculated the cost of that treatment and found it to be $3,491 per patient per year. Those costs are significantly less than the cost of hospitalizations, outpatient visits, and lost productivity.
“That is information that we can now share with payers,” states Jhaveri. “Payers must decide where their money will go. They use HEORs to get a real-world snapshot of what's going on with their patients. I understand medicine and clinical outcomes. I know what patients are going through. But that does not help me communicate with payers. Payers understand HEORs. That is the language that they speak. It is the language that allows the two sides to communicate with each other.”
Nocturia has many different causes and is associated with many different conditions. Jhaveri hopes this research, which was published in the Journal of Managed Care & Specialty Pharmacy, will also raise awareness of the condition. He notes 88 percent of nocturia cases are caused by nocturnal polyuria, which is treatable.
“That is something no one really learns in medical school,” he adds. “Physicians also do not learn about it in residency or fellowship. That is why pharmaceutical industry medical education exists. When there are advances in various areas, we can appropriately communicate them to the relevant stakeholders. Real-world evidence allows us to know what actually happened in a population. When we know that a disease contributes up to $214.5 billion in costs to the economy, we can start taking the cost of treating a single patient and seeing what happens to them over the course of time. That allows us to see what the cost savings are.”
Finally, Jhaveri believes HEOR is one of the only ways for pharmaceutical companies to show payers the true value of a treatment option. HEOR helps payers understand the economic burden of a disease and the cost of that burden to the payer. By knowing what the cost of the disease is, and the cost to treat it, payers can make a more informed decision.
“Payers will often not know how much money they're saving by making a treatment option available to patients,” adds Jhaveri. “They may also not know the potential market size. The HEOR helps to provide them with dollar values. It also tells them how much they can save. If 88 percent of nocturia cases are caused by nocturnal polyuria, and a drug exists to help eliminate even half of the cost of the disease, that could potentially save them tens of millions of dollars. If I'm the payer, I'm looking at this data and seeing the benefits of the treatment. It can save me a lot of expense and will create a better quality of life for the patient.”