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How could an economic downturn in 2023 impact the most vulnerable patients? If we look at lessons from past recessions, we see that increases in unemployment, inflation and financial stress significantly impact access to medicines. Now is the time for pharmaceutical leaders and pharmacy decision makers to consider how the industry could proactively improve outcomes for patients during a downturn, especially patients with rare conditions.
Keena Patel-Moran
How could a recession impact health care in 2023?
While health care may be more recession proof than other industries, it is not immune. We saw a significant patient shift from commercial health insurance to Medicaid in recent economic downturns. In 2008, Medicaid enrollment increased by more than 8%. In 2020, we saw a similar pattern. However, even if patients stay on commercial health insurance, recessions lead to fewer patients being able to afford their existing prescriptions. When patients have a smaller budget for discretionary spending and can’t afford deductibles and co-pays, we also see a drop in medication adherence and persistence. Some patients will even stretch out the time between filling their prescriptions or will drop off therapy altogether.
Patients with rare diseases are among the most vulnerable
One important group to consider: patients with rare diseases. Rare diseases are conditions that impact fewer than 200,000 people. But in aggregate, rare diseases have a significant impact. More than 30 million people in the U.S. — 10% of the U.S. population — have a rare disease. Half of those patients are children. In addition, many rare diseases require chronic treatment, so adherence and persistence are critical.
Rare diseases, especially rare pediatric diseases, have a disproportionate share of patients on Medicaid, even in normal economic conditions. We could see even more patients with rare diseases on Medicaid in 2023. And affordability challenges will be even more pronounced in 2023, especially in rare diseases. Each year, rare therapeutics top the list of the most expensive medicines in the U.S., with the highest-cost therapies reaching upwards of $2 million per patient per year. In addition, rare diseases often require complex care coordination across multiple specialists and caregivers, which exacerbates the economic pressures.
A recession is the right time to solve for rare diseases
Developing drugs for rare diseases is an important public health goal, and the pharmaceutical industry has been making headway in developing treatments for rare conditions. While 90% of rare diseases still do not have an approved treatment, there has been a steady upward trajectory of approved drugs to treat rare or “orphan” diseases since implementation of the Orphan Drug Act in 1983. Today, there are over 599 approved orphan products to treat rare diseases, the majority of which treat only one condition. More than half of the new drugs approved are for rare diseases.
Betty Pio
Due to the high patient and cost burden these important therapies represent, a recession is the perfect time to reimagine the model for treating rare diseases effectively.
Rare disease treatment in action: hemophilia
More often than not, for patients to be healthier and achieve better health outcomes, having a great therapy is just not enough. Hemophilia offers a good example to explore.
Hemophilia is a rare and life-long bleeding disorder in which the blood does not clot normally. While people with hemophilia can lead fairly normal lives with certain precautions to prevent and control bleeds, managing the condition can be challenging. According to the CDC, “the blood of hemophilia patients does not contain adequate amounts of the specialized proteins needed for blood clotting. As a result, people with hemophilia are subject to medical complications including anemia, internal bleeding, damage to muscles and joints, and excessive bleeding after minor injuries.”
Hemophilia primarily affects males and can disproportionately affect a single family if there is a hereditary factor, and the condition is more prevalent within Caucasian and Hispanic populations. Based on estimates from a recent study, as many as 30,000 males have hemophilia in the U.S. This finding was significant, as previous estimates were nearly half of that. The study also found that patients with hemophilia are generally young and that the disease is more common in Northeastern and Midwestern states, with the highest rates found in Iowa, Indiana, Ohio, Vermont, New Hampshire and Maine. An estimated 30% of people with a bleeding disorder are enrolled in Medicaid.
Hemophilia is one rare condition with many new therapies coming to market. The global hemophilia market reached $12 billion in 2021 and will double in less than a decade. Sanofi and Sobi’s Helimbra rival is expected to launch in the coming months; CSL Behring and uniQure’s hemophilia B gene therapy, Hemgenix, has been approved by the FDA. As a result, we will continue to see great scientific achievements, with the potential for high drug pricing but meaningful new benefits reaching affected patients.
Rare diseases require a local care model
Therapy alone will not solve the burden of rare conditions like hemophilia.
The needs of a hemophilia patient can change over their lifespan, and the ability to respond quickly can improve outcomes. One example of this is the risk of developing an inhibitor. An estimated 20% to 30% of people with severe hemophilia A and 1% to 4% of those with severe hemophilia B may develop an inhibitor, a negative immune response to standard replacement therapy treatment. Inhibitors are more likely to develop in early childhood and interfere with response to standard therapies. Treating patients who have developed inhibitors is uniquely complex and costly, and frequent monitoring of any increase in inhibitor levels is important. Hispanic patients with severe hemophilia are about twice as likely to get an inhibitor than non-Hispanic white patients. The health care community must come together to provide long-term personalized solutions by creating partnerships between the pharmaceutical industry, primary care teams, pharmacy teams and other community-based health care workers.
Living with a rare disease can create major financial and emotional stress for families. The National Hemophilia Foundation notes that living at a distance from expert care can impact financial and emotional stress levels, as many families live in areas where therapy requires frequent travel. Parents and caregivers need to take time from work, which is not always an option in times of economic stress.
Typical population-based health strategies and interventions don’t work well for rare diseases. It’s harder to put in effective programs and deliver them at scale when everyone needs a slightly different solution. Local pharmacies may be best positioned to help. A recent study demonstrated that including a pharmacist in the hemophilia comprehensive care model improved health care-related outcomes (reduced overall bleed rates, emergency department visits and hospitalizations) while also reducing drug costs over a 12-month period.
Local care models have already been growing, and the need for them will accelerate in an economic downturn. Today, pharmacists may work directly with patients on health and wellness screenings, flu shots and other immunizations, patient education, and chronic disease management. Local pharmacies can play a critical role in keeping patients on their prescribed therapy, even under financial stress. Strategies for pharmacists could include helping to collect vital patient information and consent to initiate pharmaceutical patient services support, enabling pharmacist-driven patient monitoring programs to generate higher-accuracy clinical patient data capture, medication reminders with a personal touch and patient education tailored for the local community, to name a few. Integrating pharmacies and other local community health workers into the primary care model could create a new and much needed layer to the delivery of health care, improving patient outcomes.
A patient’s local pharmacist has a direct view of the social determinants of health and other factors affecting a community. They’re likely to understand a community’s diverse challenges — economics, culture, language and the regional concentration of heritable diseases, to highlight a few. And as we see fewer doctors in communities, the role of pharmacists will become more critical. In 2019, there were about 228,000 direct patient care physicians in the U.S. That works out to be one primary care physician for about every 1,429 people in the U.S., and one primary care professional (including physician assistants and nurse practitioners) for every 895 people.
When you look at the distribution of specialists, the numbers are even more staggering. For example, there are only 16,000 hematologists and 3,000 pediatric hematologists in the U.S. But an estimated 387,000 licensed pharmacists are practicing in the U.S. Pharmacists also have the advantage of frequent touchpoints with patients. Regular communication with patients means that pharmacists are in a prime position to share critical information about a patient’s treatment with the patient’s provider. Pharmacists can fill a critical gap for all patients, including patients with rare diseases.
No matter what economic conditions are around the curve in 2023, the local care model, with pharmacists on the ground, will play an essential role in keeping communities healthy.
Keena Patel-Moran and Betty Pio are partners in the health practice at Kearney, a global strategy and management consulting firm. They can be reached at keena.patel-moran@kearney.com and betty.pio@kearney.com, respectively. The authors wish to thank Stephanie Young for her valuable contributions to this article.
