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Rare Disease Pharmacy: Navigating REMS, Hub Services, and Single-Source Manufacturing

  • 72 minutes
  • 38 slides
What we'll cover
  • 0:00 Rare Disease Access Reality
  • 8:00 REMS as Workflow Architecture
  • 20:00 Hub Services Decision Points
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What you'll take away

Practical shifts you can apply this week

  • Identify the operational choke points created by REMS requirements in rare-disea

    Spot where enrollment, labs, and documentation slow therapy so teams can reduce avoidable delays.

  • Evaluate when hub services reduce friction versus when they add handoff risk and

    See when a hub clears payer and onboarding work, and when it mostly adds another inbox.

  • Compare distribution and fulfillment models for therapies dependent on single-so

    Weigh control, resilience, and reach across exclusive, limited, and hybrid channel designs.

  • Design cross-functional controls for continuity of care during supply, authoriza

    Leave with a practical way to assign owners before a denial, shortage, or missed lab disrupts care.

  • Decide which metrics best reveal access failure before patient abandonment appea

    Track the few signals that surface hidden drop-off earlier than shipment counts or revenue trends.

Agenda

What we'll cover

  1. 0:00

    Rare Disease Access Reality

    Why small populations and high-consequence delays make operational misses more damaging here.

  2. 8:00

    REMS as Workflow Architecture

    How certification, enrollment, labs, and documentation shape who can start and stay on therapy.

  3. 20:00

    Hub Services Decision Points

    Where hubs remove payer and onboarding friction, and where they create bounce-back and delay.

  4. 29:00

    Single-Source Manufacturing Exposure

    How one disrupted path can ripple into scheduling, allocation, and patient communication.

  5. 39:00

    Distribution Model Tradeoffs

    A practical comparison of exclusive, limited, and hybrid models for ultra-rare products.

  6. 47:00

    Failure Modes and Recovery

    Playbooks for REMS lapses, prior auth denials, shortages, and missed monitoring events.

  7. 55:00

    Metrics, Recap, and Q&A

    The operational measures to review first, plus closing guidance on mapping your weakest handoff.

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