5 Commercialization Hacks for a Rare Disease Treatment

When it comes to commercializing rare disease products, there are no shortcuts to success. But there are proven ways to avoid time- and resource-consuming pitfalls. My colleagues and I at AmerisourceBergen have partnered with hundreds of pharmaceutical companies—including many in the orphan space—to create end-to-end commercialization strategies. And after more than 20 years of experience, we’ve identified a few hacks that can help manufacturers improve product performance and patient outcomes.

Hack #1: Think like a payer…or find someone who can.

As market baskets become more competitive, payers place greater scrutiny on the budget impact of these treatments—even if the treatments are only accessed by a very small percentage of patients. Payers are also evaluating the potential impact of expanded indications, and how larger patient populations would increase the budget impact on their plans. The cost-prohibitive price of many orphan drugs contributes to restrictive behaviors for pharmacies, payers, and patients alike. Orphan drugs often have far more complex education and evidence hurdles to overcome. The manufacturer serves as a critical resource for every stakeholder in the patient journey and must clearly communicate the product’s value in a way that addresses how each stakeholder prioritizes and perceives value.

The right commercialization partner, like AmerisourceBergen’s Xcenda, can help biopharmaceutical firms that lack infrastructure in health economics and outcomes research (HEOR) or market access. Through HEOR outsourced staffing, a part-time or full-time market access expert works hand-in-hand with manufacturer leadership to design product HEOR plans or develop evidence-based tools to support that strategy. The staff member often works on-site at the manufacturer’s location and is supported by a scalable team that has authored scientific articles in national and international peer-reviewed publications and presented product evidence to national health technology authorities. This solution enables access to the real-world experience needed to support rare disease products worldwide.

Hack #2: Think globally. Execute locally.

Advances in identifying genetic markers for diseases have created opportunities to conduct clinical trials with smaller, targeted patient populations. This often requires trials in multiple countries, including emerging markets. Yet recruiting and retaining patients within small populations is quite daunting. As a result, a patient-centric clinical trial design is critical and requires a partner with multi-national scale and local expertise.

Whether you’re choosing a specialty logistics partner to manage clinical trial phases, or you’ve left this decision to your CRO, ask your partner about its global reach and quality standards, as excellence in these areas reduce risk and improve treatment access for patients worldwide. During the pre-commercialization phase, ask your partner about how they evaluate the ways in which a product’s characteristics influence storage and transport. These learnings, executed with a partner experienced in global transport, will help the manufacturer to transition logistical and distribution support into the commercialization phase. A partner should have the ability to offer local knowledge from experts on the ground in various countries to enable a fully GxP-compliant quality system through the full product lifecycle.

Hack #3: Address what patients value the most.

A Shire Rare Disease Impact Report1 revealed that patients suffering from orphan diseases typically visit up to eight physicians and receive two to three misdiagnoses before an accurate one is reached. The lengthy path to diagnosis and treatment takes a significant emotional and financial toll on these patients and their caregivers. Partners such as AmerisourceBergen’s Lash Group, design high-touch programs to help patients and caregivers navigate critical product access scenarios that exist most acutely for small patient populations.

In rare disease, addressing ongoing care is equally important and the role of case manager within support programs can make all the difference. As the patient’s single point of contact, case managers can provide continuity of care, confirming coverage and providing reimbursement support, overseeing medical appointment scheduling, working with specialty pharmacies to ensure timely administering of drugs, and coordinating with physicians to provide updates on patient progress. This latter aspect is especially important in capturing health outcomes data from support programs and helping manufacturers demonstrate the drug’s real-world effectiveness and value.

Hack #4: Enhance patient care with the right specialty partners.

The specialty pharmacy strategy for an orphan product is critical for commercialization success and should be evaluated based on product attributes and patient needs. Network design decisions must balance two patient-centered priorities: access and safety.

Support programs for orphan and ultra-orphan drugs exist due in large part to cooperative relationships with specialty pharmacies. Specialty pharmacies, like AmerisourceBergen’s US Bioservices, serve as patient advocates, leveraging their expertise to advocate when payers attempt to limit access. Specialty pharmacies help find financial assistance to enable broad access and improve speed to therapy, and are equipped to collect and report adverse events, patient-reported outcomes, and other data elements that help manufacturers and providers improve care. Comprehensive inventory management and pharmacist quality checks ensure that orphan medications are dispensed properly, while technology solutions enable manufacturers to access detailed program information and strategic insights throughout the product lifecycle.

Hack #5: Seek guidance to shape strategies.

The right partner should guide your team through each decision and make proactive recommendations. Questions to ask:

  • What works well for this product’s attributes/this patient population?
  • What is going on across the market that might affect product launch?
  • How will the product’s clinical roadmap affect channel strategy?
  • What is the best way to reach hospitals/health systems that specialize in rare disease?

An experienced commercialization partner will know how questions like these will influence your decisions and help you adapt as market dynamics, policy, or patient needs change.

In closing…

With more than 7,000 rare diseases identified, and most lacking treatment options, it’s important to recognize that even with the rapid pace of innovation, patients will continue to face a high unmet need. And for manufacturers, finding the right partner who can help overcome obstacles that separate products from the patients who need them will greatly simplify the complex road to successful commercialization.


1. “Rare Disease Impact Report: Insights from Patients and the Medical Community.” Shire. April 2013. Accessed 26 September 2016. Available online at https://globalgenes.org/wp-content/uploads/2013/04/ShireReport-1.pdf.

  • Amy Grogg

    Amy Grogg, PharmD is Senior Vice President of Strategy and Commercialization at AmerisourceBergen Specialty Group. Dr. Grogg develops strategies to advance AmerisourceBergen’s leadership in the pharmaceutical services industry and position the company as the preferred commercialization partner for specialty pharmaceutical manufacturers. She also holds operational responsibility for Xcenda, part of AmerisourceBergen, which provides strategic consulting services for life sciences manufacturers commercializing products in the U.S., Germany, and key global markets.