Brandon Salke
Pharmacist in Charge, General Manager of Operations
Optime Care

Reengineering cells, repairing or replacing genes: this is the new frontier of medicine. For many patients with rare and orphan diseases, cell and gene therapies may offer new hope. They also require what these patients know so well—the need for individual attention.

Each Individual Cell Therapy and Gene Therapy is Unique to the Patient

A cell or gene therapy patient is literally a population of one. Every treatment must be tailored to the patient’s precise needs. For example, CAR-T (chimeric antigen receptor) cell therapy could be promising for many cancer patients when traditional approaches fail. With this therapy, the patient has her own
T-cells removed, reengineered and then reintroduced into her body. The reengineered cells are irreplaceable. And when her life hangs in the balance, there’s little room for error.

Contrast this with a far more common condition such as diabetes. Roughly one in 10 Americans have it, and as many as 95 percent have the type-2 version. A first-line treatment for type-2 diabetes is metformin; according to one study, more than a half billion metformin prescriptions were dispensed in the U.S. from 2000 to 2015.

Imagine the difference in dispensing millions of identical prescriptions versus tailoring treatment for a few patients—or a single one. A mass market approach simply can’t be customized at that level because it requires speed, efficiency and above all, “sameness.” The goal is for every patient encounter to be treated as a uniform transaction. That may work when dispensing metformin. But when every patient is an “outlier,” the mass market approach begins to falter.

In our experience, applying a mass-market strategy to a small patient population can lead to less-than-optimal results. But that should come as no surprise. Far more than filling a maintenance prescription, the specialty pharmacy must stay closely engaged with the patient, family and caregivers—answering questions, managing side effects and ensuring adherence and persistence as applicable. No technology can take the place of a knowledgeable, reassuring voice when a patient calls worried in the middle of the night.

For rare and orphan disorders, the patient—not the process—must come first. These conditions require a patient-first strategy.

Prepare now for the new frontier of Cell and Gene Therapies

The need for a patient-first mindset has never been more important than today, as cell and gene therapies emerge for some of the most challenging diseases.

The FDA is approving more and more of these therapies. Employers and payers are asking for them—but they’re concerned about access, cost and the availability of data to know the treatment is working. How will manufacturers address these concerns? Will they take the traditional path or a truly patient-first approach?

Optime Care was founded on our patient-first philosophy. From day one, we have customized care for every patient we serve. As more cell and gene therapies come to market, we’ll be ready to play our part in optimizing treatment for rare disease communities.

To learn more, download our white paper, “How to create a patient-first strategy.”

Or contact us below, or call 888-287-2017.

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