For many patients with rare diseases, reaching the first step of an accurate diagnosis can be a lengthy journey, riddled with pain, difficulty, and trial and error—and that’s before they begin the challenge of fighting and treating their conditions.
Once diagnosed, patients with rare diseases often face a new set of challenges: affordability, adherence to complex regimens, side effect management, and coordination of follow-up appointments that may involve providers far from home. And yet, innovative therapies are emerging that offer patients, including those who had previously exhausted all other options, new opportunities for improved health outcomes.
Highly targeted treatments allow key stakeholders to apply more personalized approaches to disease states, even those as broad as cancer. This specificity requires new levels of coordination from all involved—manufacturers, wholesalers, distributors, payers, prescribers, and pharmacies—to effectively support the patient and remove barriers to access and adherence. Within the pharmaceutical ecosystem, specialty pharmacies are primarily recognized for delivering education and medication to patients, but they also provide high-touch support that extends to many other areas of patient care.
Patients with rare, complex, and chronic conditions rely on specialty pharmacies to help them overcome the unique and difficult challenges that arise when gaining access to new therapies. Approvals of specialty medications are on the rise, but to manage costs and make coverage decisions, insurers are requiring greater pharmaco-economic evidence from manufacturers, and stringent prior-authorization requirements before reimbursement approval for patients.
Working collaboratively with the prescriber and patient, specialty pharmacies help remove affordability barriers by coordinating submission of complete clinical information based on payer requirements and investigating all potential sources of funding. Both processes can be overwhelming for patients to navigate on their own, and can ultimately delay treatment. Specialty pharmacies assist patients with insurance benefits and authorizations, manufacturer co-pay assistance programs, and foundation assistance, helping to expedite the start of therapy.
Once therapy access is secured, and especially as innovative therapies come to market, collaboration between specialists and specialty pharmacies is critical to optimize the patient’s response to treatment. These stakeholders must work in partnership to educate and empower the patient to take the medication safely and appropriately.