With shrinking pipelines, increasing treatment costs, stiffer competition, and stricter regulations, pharma companies struggle more than ever to make their drugs succeed. With orphan drugs (ODs), commercialization is even more difficult given the lack of knowledge about the disease state, fewer analogs to derive lessons from, and little understanding of the patient experience. Thus, pharma companies must invest early in the drug development lifecycle to understand market requirements and implement a robust product launch strategy. A well-planned go-to-market framework should follow an integrated approach from developing a winning commercialization strategy to operationalizing it.

Challenges in commercializing orphan drugs

Pharma companies face unique challenges when commercializing ODs. Due to these factors, the rare disease (RD) and OD commercialization model differs significantly from non-RDs and non-ODs.

Here are four of the top challenges

Diagnosis issues

Often, doctors struggle to diagnose RDs as patients face long evaluation times and multiple physicians before diagnosis. Further, patients may struggle to find physicians with the right expertise to provide an accurate, speedy diagnosis.

Medical and economic burdens

RDs may cause patients chronic or progressive physical deterioration, disability, or early death. This causes patients, caregivers, and their families significant financial burden. The latest reports show that in 2018, the mean annual OD patient cost was $150,854 versus just $33,654 for non-OD patients.

Pricing and value assessment issues

Rare diseases bring challenges involving research and health technology assessment, clinical outcome assessment, drug valuation, and use of traditional health economic models of outcome assessments that may not apply or require adjustments when applied to Orphan Drugs.

Government role and rare disease patient organizations (RDPOs)

Globally, government reimbursement for Orphan Drugs vary as questions arise regarding the return-on-investment of spending significant resources on RDs affecting few people. The number of non-OD indications for OD drugs is also increasing, likely causing the tax credit cut for OD clinical trial costs from 50% to 25%, as the FDA reports. Lastly, RDPOs, defined as non-profit organizations representing the needs of patients with RDs, play an increasingly important role in advocating research for RDs, prioritizing RD research by governments and pharma companies, and promoting greater involvement of patients and related individuals in research.

Commercialization elements for orphan drugs

Successfully commercializing ODs requires a range of strategic and tactical elements for pharma companies to implement. These elements are listed below in approximate chronological order according to the life cycle of the product, from the clinical trial stage through to post-launch

Patient development for RD clinical trials: Pharma must work closely with patients and RDPOs to recruit clinical trial patients.

Improvement in RD diagnosis and treatment: Pharma companies must collaborate and share data on RD mechanism discovery and accelerate pathways for patients to accurate diagnoses. Further, they must educate physicians, engage patients, and create an RD physician registry for patients.

Market access and patient affordability: Pharma companies need to develop strong RD health economic models to demonstrate value to payers to subsidize patient healthcare costs.

Pre-launch preparations: Pharma companies should increase efforts pre-launch to ensure a successful OD launch.

Sales and marketing activities: Pharma should harness a solid digital and social media presence for patient, caregiver, physician, and key opinion leader (KOL) engagement. Further, companies should provide caregivers greater support, conduct patient-journey analyses, and prioritize informative sales and marketing strategies over traditional persuasive approaches.

Specialized supply chain development: Pharma companies must understand the distribution of ODs for RDs through specialty pharmacy and buy-and-bill channels. Specific OD treatments may require specialized supply chains.

Engagement with government agencies and policy decision-makers: Pharma companies must actively engage with government agencies and policy decision-makers to address the economic and social impacts of RDs.

Greater cross-functional collaboration with internal pharma organizational units: Pharma tends to run initiatives in silos and stick to legacy systems. These behaviors cause commercial initiatives to trail behind clinical and result in poor launches. To break these barriers, the industry must think through initiatives well, adopt cross-functional approaches, and leverage data and analytics to help launches succeed.

Rare disease drug launch use case

A specialty pharma company was in the advanced stage of launching a novel drug for a RD. The business team struggled to answer the basic questions around the target population, top treaters, and the treatment journey. Together, we collaborated with the client to develop a commercialization strategy after a thorough analysis of the patients’ treatment journey.

This rare disease had a high misdiagnosis rate, which made determining the specific patient population difficult. In addition, the disease had a complex diagnosis and treatment process involving multiple healthcare professionals (HCPs), causing difficulty in determining the correct targets.

The analysis involved using multiple datasets, including patient-level data and HCP affiliation data. Given the disease’s complex treatment and diagnosis patterns, several dataset instances involved the alignment of a single patient with multiple HCPs and led to the difficulty in the HCP segmentation and valuation process resulted. Targeting also became challenging as the exercise required identifying HCPs with the most recently treated or diagnosed patients. The company also faced difficulty identifying high-value HCPs as the ZIP code information in the patient and HCP data differed.

For the go-to-market strategy, we followed a five-step approach to segment and target the HCPs, which included:

• Patient journey and segmentation schema
• Customer segmentation
• Customer valuation and targeting
• Salesforce size, structure, and deployment
• KOL identification

Each of these fives steps involved specific activities including using longitudinal patient-level data to examine how HCPs treat specific patient segments that classified as priority targets; defining high-value targets and ensured optimized salesforce coverage using a robust go-to-market approach, including salesforce size, structure, and alignment; and using a local, regional, and national sphere of KOL influence (while indexing distances for population density).

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