By Brandon SalkePharmacist in Charge, General Manager of Operations, Optime Care

For patients who have nowhere else to turn, cell and gene therapies can be a second chance. They are the future of the medicine. And they are destined to fall short of their full potential to save lives—unless manufacturers see what they’re missing.

To be sure, the light of innovation can be blinding. Consider the patient whose rare immunodeficiency is finally managed by correcting a dysfunctional gene. Or the cancer patient who has exhausted all traditional forms of treatment, and then a cell therapy called CAR-T saves her life.

From engineering therapeutic cells outside the body to transforming genetic material into a drug itself, innovation is exciting. But so are outcomes. And it takes more than talk for the former to lead to the latter. For their cell and gene therapies to succeed, manufacturers must take a new approach as radically different as the therapies themselves. Manufacturers must reengineer the way they think about administering and monitoring care over the long term. At stake are the very lives of the patients we are all committed to serve.

Indeed, I’m talking about adopting a patient-first strategy. Its value to all stakeholders—from manufacturer to payer, provider to patient—is well established and proven to succeed. It’s the very reason Optime Care was founded. You can learn more about this patient-first strategy by downloading our white paper, “How to create a patient-first strategy.” You’ll see how this approach not only applies to cell and gene therapies, it was literally built for them.

Where traditional thinking falls short

When it comes to cell and gene therapies, it’s easy to fall into the trap of thinking short term, simply identifying the patient and delivering the treatment. But diagnosis and distribution are just the beginning.

How are you monitoring and tracking the patient experience one, five—even 10 years and beyond? With that data set, you would be able to establish the long-term value of the therapy to all stakeholders. You could answer such critical questions as, Has the product improved the patient’s quality of life? Has the outcome justified the product’s cost (which for cell and gene therapies can be in the millions of dollars)?

Next, you have to understand how very different the monitoring of the patient experience is between cell and gene therapies. With gene therapy, there’s a one-time treatment to modify the genes. Following that treatment, what are you doing to ensure your product demonstrates its long-term value? Do you have the data to support that conclusion?

With a cell therapy, the treatment regimen is much different. A patient may have multiple doses every year for life. What is the patient’s status in between doses? Who is monitoring that and reporting back to the manufacturer, payer, provider and patient year in and year out?

Without this visibility and data collection throughout the patient experience, how can you expect to justify your therapy’s cost?

Multiple vendors: another roadblock to success

Manufacturers of cell and gene therapies typically use multiple resources to deliver patient care; for example, having a separate specialty pharmacy, specialty distributor and hub services provider. Then, a manufacturer may use additional, separate vendors for patient financial assistance, billing administration and scheduling lab work. These resources all work independently, don’t communicate efficiently (if at all) and create bottlenecks that can delay care.

If a patient needs blood pressure or type 2 diabetes medication, perhaps the delays would have less of an impact on health. But cell and gene therapies can be lifesaving. A CAR-T patient may be lying in the hospital and have a matter of days—not months—to live. If you can’t deliver the therapy quickly, you may lose the patient.

Where manufacturers obtain these multiple services can be just as detrimental. A manufacturer accustomed to working with a particular “big box” specialty pharmacy and specialty distributor for a mass market drug may automatically turn to them for a new cell or gene therapy.

That manufacturer may then decide to create its own internal hub services, thinking it would improve control over patient care. The manufacturer might hire an outside “expert” to create the hub and believe the problem was solved. Nothing could be further from the truth.

Manufacturers aren’t built to be patient services organizations. They don’t have the infrastructure or expertise to deliver the patient experience required for cell and gene therapies.

Different therapy, different approach

The bottom line: you can’t rely on a traditional, multi-vendor approach to work for your unique cell or gene therapy. Your patient’s needs are as individual and unique as your product. Taking a mass market approach simply won’t lead to success.

At Optime Care, we’re built to work with small patient populations. Indeed, they can number in the hundreds, or even less, for rare and orphan disorders. But the number of patients receiving a particular cell or gene therapy can be far lower. These are the smallest of the small patient populations we see (in many cases, a population of one).

As a result, the requirements you must meet are the most daunting you’ll likely ever face, such as the time-critical nature of distribution—obtaining approval and gaining reimbursements from payers—and data collection over the long term (perhaps over the entire life of the patient).

Let’s focus on reimbursement for a moment. For years, payers have talked about requiring positive outcomes data to justify the price of a therapy. Now, with the extraordinary cost of some cell and gene therapies, this data will be more critical than ever.

If you fail to collect it, you won’t have a case for continuing to keep a patient on your therapy over the long term. That’s bad for the patient—not to mention your bottom line.

In our experience, a patient-first model solves these issues. If your goal is to deliver your therapy faster, using multiple vendors will only slow you down. Instead, rely on a “one-stop shop” of interconnected services working together to streamline the patient experience.

The patient-first model is a unique and proven combination of specialty pharmacy, specialty distribution and patient hub services—with a care coordinator connecting it all. For example, with a cell therapy the care coordinator would schedule cell pickup, lab delivery and then shipment back to the provider for administration to the patient. This level of oversight can cut precious time out of the process and potentially improve outcomes.

With this model, you’re also collecting data across the entire patient experience—sometimes for many years. So you can monitor and confirm the long-term value of the therapy to all stakeholders.

That’s why it’s essential manufacturers leave traditional thinking behind. I can’t say it strongly enough. Stop believing the job ends with diagnosing patients and distributing therapies. When a therapy is literally customized patient by patient, you’re well beyond the capabilities of a typical specialty pharmacy or distributor.

To succeed, you must see what you’re missing. Think holistically and adopt a patient-first strategy coordinated by a single source—not scattered across multiple, disjointed vendors.

In our experience, manufacturers know that taking a traditional approach with their cell and gene therapies isn’t working. But they don’t know there’s a better way. I can assure you there is. The patient-first approach works.

To learn more, download our white paper, “How to create a patient-first strategy.” Or contact me directly at or call 888-287-2017.