Friday, February 07, 2025

Today’s Rare Disease Launches Require the Right Specialty Pharmacy Partner

Today’s guest post comes from Richard Faris, Chief Strategy & Growth Officer at PANTHERx Rare Pharmacy.

Richard outlines the key factors for commercialization success when a manufacturer launches treatments for rare and orphan diseases. He explains the key considerations for choosing a specialty pharmacy network design, selecting the right pharmacy partners, and establishing patient services HUBs.

To learn more, download PANTHERx’s latest white paper and SP decision-support resource: Selecting the Right Specialty Pharmacy Partner for a Successful Launch.

Read on for Richard’s insights.

Today’s Rare Disease Launches Require the Right Specialty Pharmacy Partner
By Richard Faris, Chief Strategy & Growth Officer, PANTHERx Rare Pharmacy.

Rare diseases, defined by the Orphan Drug Act as affecting fewer than 200,000 individuals, require highly specialized treatment and support. The complexities surrounding these therapies demand that manufacturers invest significantly in their commercialization strategies, particularly in selecting the right specialty pharmacy (SP) partner. This important decision can influence patient access to therapy, treatment outcomes, and ultimately, a product’s financial performance.

COMPLEXITY ALSO IMPACTS LAUNCH PLANNING AND EXECUTION

Do manufacturers launching their first rare-disease or orphan therapy realize that partnering with the right SP can significantly influence commercial success or failure? Even if brand leaders have experience with SPs outside of rare diseases, they may not understand that rare-disease-focused SPs are not like traditional SPs, and that the usual pharmacy considerations simply don’t apply.

In addition, launch planning for rare disease treatments must start much earlier due to the complexity of therapies and their administration, patient populations, supply chain, access and reimbursement, and other factors. And partnering with experts in an SP that specializes in rare and orphan launches can speed a manufacturer’s learning and reduce costly mistakes. This means a materially better launch, with more patients getting access to a new life-saving treatment, more personalized and consistent patient support, and more real-time feedback for the patient’s care team.

CHOOSING THE RIGHT SPECIALTY PHARMACY MODEL

Manufacturers invest decades and hundreds of millions of dollars developing therapies but get only one opportunity to launch. Setting up the right SP distribution model well before launch is critical for the success of your product. Your SP partner(s) will be with you for the long run, providing comprehensive support and actionable insights for market positioning, so it’s critical that your due diligence enables you to objectively assess their strengths and shortcomings as not all pharmacies are created equal.

KEY CONSIDERATIONS FOR SELECTING A SPECIALTY PHARMACY

The SP is a critical partner on a brand’s extended launch team, and the cost of switching to another SP if you make a mistake is high. Here are some high-level considerations to help reduce risk and identify SP partners who may be a good fit:
  • Patient journey: Identify SPs that understand and support the patient journey using personalized, data-informed services.

  • Collaborative relationships: Assess SPs in meeting your specific data and insights needs to help understand treatment uptake, prescriber and payer trends, and navigate strategy evolution.

  • Narrow or Exclusive Arrangements: Many manufacturers have pivoted from large SP networks to smaller or exclusive SP models. The expertise of smaller, highly-focused, and nimble SP providers—rare-only pharmacies, capable of delivering tailored solutions for rare populations—is now the gold standard.
The decision to choose a single, exclusive SP versus two or more has implications for launch as well. Figure 1 shows product characteristics that may impact SP distribution options.

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ROLE OF PATIENT SERVICES HUB IN RARE DISEASES

Due to the high complexity and cost of rare treatments, many manufacturers decide to offer HUB services. A HUB provides capabilities that enable a manufacturer to have a single point of contact with patients and may assist in delivering patient services, thus impacting patient and prescriber satisfaction.

While a HUB can enhance patient care and adherence, it also adds complexity and cost. Figure 2 details important considerations when adding a HUB:
  • Patient & prescriber experience: It’s critical to align care using clear communications with the appropriate language and channels for each audience, at the right frequency.

  • Manufacturer control: Tradeoffs may need to be made when partnering with one SP vs. several. When partnering with more than one it can be difficult for the manufacturer to orchestrate a cohesive experience across SPs for patients and prescribers.

  • Cost: HUBs often charge for “add-on” services that may be core services included by SPs. Use of an external HUB in an exclusive network is not recommended.

  • Staffing expertise & continuity: Level of staffing for your brand may not always be guaranteed, and neither is their expertise that can ultimately affect patient care quality and satisfaction.

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PANTHERX RARE IS THE RARE DISEASE PHARMACY®

Since PANTHERx was founded in 2011, it has been one of THE leading specialty pharmacies for rare and orphan diseases. We proudly distribute rare and orphan therapies nationally and focus on providing access and support services to the people who need them most. And we’re recognized as the expert in patient experience and care, earning a record seven MMIT Patient Choice Awards.

PANTHERx has launched nearly four dozen rare-disease therapies with our biopharma partners—often as the exclusive SP—and has helped many emerging companies launch their first rare-disease product. PANTHERx consistently delivers for patients, healthcare professionals (HCPs), and manufacturers—from launch through lifecycle management.

Click here to download our latest white paper and SP decision-support resource, Selecting the Right Specialty Pharmacy Partner for a Successful Launch.


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