Patients Would Give Up Over 5 Years of Life for Easier Treatments, Study Finds

Adults with cystic fibrosis (CF), an inherited disease that about 30,000 people in the US live with, spend approximately 2 hours per day on all routine treatments according to some studies.  For many, the time spent administering treatment, especially those to treat airway abnormalities and chronic respiratory infections, has increased in recent years as the number and complexity of treatments has grown.

A new study in the American Journal of Pharmacy Benefits by Dana Goldman and John Romley of the USC Schaeffer Center for Health Policy and Economics and others, looks at the relationship between treatment burden and quality of life for these individuals and finds the burden of administering a treatment is a significant factor in patient preference. In fact, they found that nearly 90 percent of adults with CF may be willing to give up a substantial amount of life expectancy to use the newer, less burdensome dry powder treatment option compared to traditional nebulized therapy.

What Are Treatment Options for Patients with Cystic Fibrosis?

People with CF experience multiple chronic symptoms including significant complications with lung functionality and infection eventually leading to death. Though life expectancy has increased substantially in recent decades, it remains shortened with a median life expectancy of about 41 years.

This improvement in life expectancy has arisen in part because of the introduction of new respiratory therapies to treat these chronic airway infections. Traditionally, nebulized therapy has been used and is found to be very effective in clinical trials but adherence to the treatment regimen outside of clinical trials is often low.  Administering the standard nebulized therapy takes 50-70 minutes daily including set-up and clean up, the equipment is heavy and the nebulizer requires a power source. With the progression of the disease, the nebulized therapies become increasingly time consuming and otherwise challenging to administer.

In comparison, the newly introduced dry powder inhaler requires only 10-15 minutes daily to administer, is compact and lightweight.  It also has found to be safe and effective in clinical trials.

To evaluate whether patients value the burden associated with administering treatment, the authors used a validated and widely used survey method to evaluate patient attitudes towards the two treatments by removing other variables. In particular, the survey respondents were told their out-of-pocket costs would be the same between the two options and life expectancy wouldn’t be affected.  Thus the key difference between the two treatments is that the dry powder inhaler takes substantially less time to use and is otherwise considered to have less of a non-economic cost for the patient.

How Do Patients Value Treatment Burden?

Quality of life for patients with a chronic disease is important to consider when analyzing treatment burden and other health care factors.  Through surveying 73 adult CF patients who had previously been diagnosed with a chronic lung infection and used a nebulized therapy for treatment, the researchers found 89% would prefer the dry powder inhaler over the nebulized therapy if their out-of-pocket costs were the same. Furthermore, 53% would be willing to give up 1.5 years of life (out of a hypothetical life expectancy of 40 years) to have the dry powder therapy over the nebulized therapy option.  When looking across the full distribution of responses, the average adult with CF was willing to give up 5.3 years of life to have the less burdensome treatment.

What are the Policy Implications?

These results indicate that the burden of a treatment factors heavily for patients with CF and might indirectly affect patient outcomes.  The researchers note that their findings, “raise the possibility that use of a dry powder might lead to improvement in adherence to chronic therapy, a critical issue in CF care.” Improved adherence could lead to improved health outcomes.

Broadly, the US health care system has been moving towards prioritizing the patient within frameworks of care. Research studies like this one provide important insight into the patient perspective and the patient’s valuation of non-clinical components of treatment. Furthermore, as this study indicates, incorporating patient preference into disease management may ultimately impact health outcomes, making it a factor to consider in clinical decisions.