Rare Disease Care: The Benefits of a Specialty Pharmacy

21 September 2021


When a biopharma company develops a therapy for a rare or complex disease, selecting a specialty pharmacy to partner with is an important choice. Choosing one that regularly works with patients who have rare and complex diseases means the specialty pharmacy will be familiar with the challenges a biopharma company faces during the orphan drug commercialization process, whether clinical or logistical.

An experienced specialty pharmacy also knows how to help biopharma companies meet patient-related needs, including medication adherence and emotional support.

Understanding the rare disease market

A rare disease is defined as one that affects fewer than 200,000 Americans. While there are currently approved therapies for only 5% of the 7,000 conditions classified as rare diseases1, that is changing. The U.S. Food and Drug Administration has created incentives to help accelerate orphan drug development, encouraging a stream of new therapies for patients with currently untreatable conditions. In 2020, 58% of all new drug approvals were designated for rare diseases, and more than 1,000 therapies are in the pipeline.2

Creating innovative treatments is only part of the equation, though. People with these conditions require specialized support during their treatment journey. They often have difficulty finding the right diagnosis and then may need to follow a complex treatment plan that can feel overwhelming at an already stressful time. A specialty pharmacy that works with rare disease patients acts as their partner on their treatment journey, helping them remain adherent to their medication regimens.

To do this, the specialty pharmacy coordinates care with healthcare providers and offers patients personalized support to address emotional health. In addition, while a therapy may offer life-altering treatment, it may cause side effects that seem insurmountable to a patient. An experienced specialty pharmacy addresses these challenges with urgency, compassion and personalized care.

A specialty pharmacy engages with rare-disease patients based on their personal communication preferences. It also provides extra assistance, as needed, to vulnerable patients and communicates regularly with providers to encourage continuity of care. It is a truly holistic approach.

A meaningful specialty pharmacy partnership

Biopharma companies benefit from partnering with a specialty pharmacy, too. Every orphan drug launch is different. An experienced specialty pharmacy has the infrastructure necessary to help each launch thrive. It is aware of possible risks that may affect different patient populations. It also has the expertise to manage third-party approvals that require confirmation of disease, even when providers are unaware of this process.

Distribution is another challenge. Medications for rare diseases may require special handling needs, such as refrigeration, overnight delivery and shipment tracking. A specialty pharmacy that specializes in rare diseases already uses these techniques to provide uninterrupted therapy.

Because of these benefits, some biopharma companies choose one specialty pharmacy to exclusively distribute a new therapy to a rare disease population. This establishes a consistent brand experience for all patients and provides the biopharma company with uniform patient data and market insights. An exclusive network can help a biopharma company closely manage the patient experience with its new therapy.

A deeper dive

This is just a snapshot of the integral role a specialty pharmacy plays in the orphan drug commercialization process and subsequent patient therapy journey. We go into greater depth on the benefits of partnering with a specialty pharmacy in our whitepaper, “The Value of an Experienced Specialty Pharmacy to Biopharma Companies Treating Rare Diseases.” It illuminates how the right partner excels at providing personalized, compassionate care to patients going through the toughest time of their lives.

PhRMA, 2018

2 Impact Innovation Predictability Analysis, FDA’s Center for Drug Evaluation and Research, January 2021




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