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

The FDA establishes strict regulations to help ensure the quality, safety and efficacy of medications.

For rare and orphan drug manufacturers, the patient-first model simplifies compliance with major regulatory challenges — including Black Box Warnings, REMS and DSCSA.

1. Black Box Warnings (or Boxed Warnings)

These are the FDA’s highest safety warnings to make patients aware of the serious or even life-threatening risks of certain medications. Hundreds of drugs have Black Box Warnings—such as antidepressants, which can cause suicidal thoughts or behaviors. When a medication has a Black Box Warning, it’s critical that the patient is made aware and follows any instructions.

The foundation of the patient-first approach is communication and building strong, lasting rapport with patients. With this personal relationship established, the specialty pharmacy can better educate patients — and they’ll be more inclined to understand and manage the implications of the Black Box Warning.

2. Risk Evaluation and Mitigation Strategies (REMS)

These are FDA drug safety programs to help ensure the proper use of medications with serious patient safety concerns. REMS programs help manage risk by establishing procedures for the drug’s safe administration, which can include:

• Additional provider and patient education
• Entering prescription information in an online registry

• Extra precautions to manage potential adverse events — such as administering the medication only in a facility that can provide the necessary interventional care during an adverse event
• In the most extreme cases, requiring additional lab work before prescriptions can be filled

For example, let’s say a medication is known to cause significant birth defects. Per the drug’s REMS, the prescribing physician must confirm that the patient isn’t pregnant before giving the prescription to the specialty pharmacy. The REMS would include a rigid, step-by-step procedure to ensure this safety protocol is met before the medication is ever in a patient’s hands.

Rare and orphan diseases can have very significant REMS requirements, and there’s no one-size-fits-all strategy for meeting them. Optime Care works closely with manufacturers to tailor their approach to each REMS, helping to ensure regulatory compliance while supporting quality patient outcomes.

3. Drug Supply Chain Security Act (DSCSA)

This FDA regulation requires that each pharmaceutical have a unique serial number for electronic tracing. The goal is to help protect patients from drugs that could be contaminated, counterfeit, stolen or otherwise be harmful. The serial numbers improve detection of these drugs so they can more readily be removed from the supply chain.

For manufacturers, the patient-first model significantly simplifies DSCSA compliance. With this model, a single versus multiple specialty pharmacies distribute drugs. This helps manufacturers simplify the supply chain and make it easier to track individual serial numbers, which significantly reduces the burden of DSCSA compliance.

In addition, using a single SP greatly improves visibility into drug inventory and utilization. All of the drugs (and serial numbers) that a manufacturer needs to monitor are stored in and distributed from one place. That makes product returns, recalls and expirations easier to track. Plus, manufacturers can better align inventory levels with actual utilization, which can lower inventory holding costs. Clean utilization data, originating from a single source, make it possible.

Ensuring regulatory compliance, supporting patient care.

Manufacturers should look to their specialty pharmacy to assist with developing the most effective strategies for managing Black Box Warnings, REMS and DSCSA regulations.

While the regulations themselves are rigid and can’t be tailored, your approach to ensuring compliance can be. At Optime Care, our patient-first model is designed for deep customization by manufacturer, product and patient. In fact, the model thrives on tailoring the best approach to meet the unique needs of individual patients with rare and orphan diseases — and the manufacturers who serve them.