The Pharmaceutical Ecosystem Must Work Together to Provide Sustainable Innovation and Ensure Patient Access

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.

Specialty medications often have complex clinical profiles and administration, so while the pharmacy’s traditional role of providing patient education about the therapy and prescribed dosing regimen remains essential, counseling is also needed about topics such as side-effect management, special handling of the medication, and other considerations related to the disease. Additionally, as medications are tailored to individuals and narrow patient populations, and as predictive companion diagnostics play an increased role in the use of therapeutic products, an even greater level of personalization will be required when counseling patients about therapy. Highly skilled pharmacy clinicians provide education and support that supplements patient-prescriber interactions.

Specialty pharmacists maintain specialized knowledge, both clinical and non-clinical, that is needed to ensure the best patient care in times of rapid change. Typically offering 24/7 clinical support, specialty pharmacies serve as a lifeline of information and assistance for caregivers and patients, particularly for those with specific, frequent, and dynamic needs. During these interactions and regularly scheduled clinical assessments, pharmacies gain actionable information about the patient’s progress that can be shared transparently with health care providers, enhancing the ability of the practice and pharmacy to work in a clinically integrated way.

The members of a patient’s health care team provide the highest levels of quality care when they have visibility into the factors that influence access, adherence, and outcomes. Growth in the use of specialty pharmaceuticals demands the evolution of patient care models that leverage the knowledge of all relevant stakeholders.

Specialty pharmacies will continue to play an essential role in driving adherence and persistency, proper medication management, and optimizing response to therapy— all in support of the important pharmaceutical innovation that leads to personalized treatments and targeted therapies for rare conditions. As stakeholders increase transparency and collaboration, the advancement and promise of new, innovative therapies through sustainable, clinically integrated models will help patients realize the full potential of treatment. â—†

About the Author

Kelly Ratliff, DPh, is a NASP board member and president of AmerisourceBergen’s specialty pharmacy, US Bioservices.Rebecca Shanahan is the current NASP president and chief executive officer at Avella Specialty Pharmacy.Burt Zweigenhaft is a NASP board member and health care business opinion leader who has held numerous executive and strategic advisory roles during his career.