“There’s so much evolution, in terms of advances in the rare disease space,” says Kelly Ratliff, the president of US Bioservices, a specialty pharmacy company that’s part of AmerisourceBergen. “Continued investment in research and innovation is driving significant growth in treatment options. The pipeline of drugs in the rare disease space is incredible.”

Patients that pay attention

While it can take a long time to get drugs to market, when it happens, these patients are ready.

“They know the drug is coming, they’ve read about the drug and they may have been involved in clinical trials,” says Ratliff, a pharmacist herself with over 25 years of experience in the health care industry. “They are the experts in their own right. That challenges us in the pharmacy profession to raise the bar as well, and to be sure we’re informed and ready to be their partner in care the moment new therapies are approved.”

Ratliff says patients with rare diseases are very well informed, active in their care and share treatment information with their peers. She encourages them to “continue to ask questions, seek answers and demand exemplary care and service.”

'“Our role is to be an extension of that already developed patient relationship with their physician.”'

When answers are rare

Dealing with a rare disease diagnosis is never easy. But patients and doctors can be further challenged by the intrinsic obstacles that accompany complex and costly treatments.

“Our role is to be an extension of the relationships that patients have already developed with their physician,” says Ratliff, adding that specialty pharmacies can work with patients and physicians to ensure access to vital medicines, as well as resolve concerns around insurance coverage and financial assistance opportunities. Specialty pharmacies also offer clinical support throughout treatment, such as 24/7 availability of nurses and pharmacists for questions and consultations.

“We work on behalf of our patients to remove any barrier to care and access that we can. We want to lift burden off the physician’s office,” says Ratliff. “The more we can do to streamline the process on behalf of the patient, the better their outcome will be — and the less stress we place on them and their caregivers.”