As costs for orphan drugs rise, how can we better the patient experience?

Drug prices are climbing across the board, but the cost of orphan drugswhich are used to treat approximately 7,000 rare diseaseshave superseded that of generics. Data reported by EvaluatePharma reveals that the annual cost for orphan drugs in 2016 was $140,443, compared with $27,756 for a non-orphan drug. Patients who suffer rare diseases spend 5.5 more for orphan drugs than patients requiring non-orphan prescriptions.

“If the orphan drug industry is to continue to thrive it must continue to generate innovations that justify the huge cost of these life-transforming treatments,” concludes EvaluatePharma.

Incentivizing orphan drugs

Fewer than 200,000 Americans are afflicted with the conditions which can only be treated by orphan drugs.

Created more than 30 years ago to encourage development of drugs to treat rare diseases, the Orphan Drug Act offers incentives such as tax credits, access to federal grants, and market exclusivity, for companies who develop these niche drugs. The program has given patients living with rare and often fatal diseases life-saving treatments where previously there were none. For example, over 40 years ago, there were fewer than 10 treatments approved for rare diseases. Since the law took effect, over 400 orphan drugs have been brought to market.

Despite being utilized by a much smaller patient population, orphan drugs are very profitable. “A $50,000 drug taken by 50,000 patients could bring in $2.5 billion per year for a company. A $300,000 orphan drug taken by only 5,000 people could net $1.5 billion a year,” reported the Huffington Post. Sales of orphan drugs are expected to grow 11 percent over the next five years to $209 billion; in 2016, sales climbed more than 12 percent, to $114 billion according to STAT.

Market forces driving high costs

Companies point to the high price point to develop an orphan drug. It’s quite costly to develop any drug—orphan or branded—but some feel that because an orphan drug is treating a much smaller number of people, as opposed to a branded drug that would treat hundreds of thousands, high price points are necessary to recoup on research and development.

A lack of competition can fuel drug prices—not just for orphan drugs, but generic and brand drugs as well. “People expect a generic to be inexpensive, but the reason it is inexpensive is that there is reasonable competition,” Dr. Aaron Kesselheim, associate professor of medicine at Brigham and Women’s Hospital in Boston and at Harvard Medical School, told the New York Times.

Innovating for patient support

Living with a rare disease, or caring for a loved one who does, takes an unimaginable toll—physically, emotionally, and financially. Arriving at a diagnosis can be a long, grueling process with many patients experiencing a reduced quality of life or facing limited life expectancy before even starting a treatment. If a drug is not covered by insurance, or patients cannot afford to pay out-of-pocket expenses, further distress is added to already extraordinarily difficult circumstances.

Personalized medicine is becoming not just a trend, but a priority for consumers; it will be critical for orphan drug manufacturers to not only keep up the pace with groundbreaking therapies but embrace innovative solutions to ensure their patient populations are being guided through the process from start to finish. Patients need support beyond their treatment.

Creating innovative support solutions in addition to treatments nurtures customers beyond the pill, ensuring a more impactful and positive patient experience. Pharmaceutical patient programs are not limited to treatment adherence—they address gaps in care, answer patient concerns, supply adherence recommendations, and coach them through their journey—offering guidance and understanding to patients navigating their conditions. A positive customer experience will yield healthier, engaged patient outcomes, and transparent data and brand loyalty for drug manufacturers.

Having a rare diseaseone which can only be treated by orphan drugsis a unique and sometimes lonely situation. Providing an exemplary support system for patients can mitigate some of the alienation and confusion they experience, reducing the hardships of their condition.