Most people think of pharmaceuticals as the last prescription they had filled in CVS or another major chain retailer. However, recently the industry has seen a major shift in the industry toward precision medicine, increased innovation, shrinking patient populations with higher prices, and an increased focus on rare and orphan therapies.

In fact, there are approximately 7,000 rare diseases impacting close to 30 million Americans, yielding an average patient population of 4,300. This has driven an increase in orphan drug development and approvals; orphan drugs now account for close to 21% of all brand-name drug sales worldwide. Despite all of this, 95% of rare diseases lack a commercially-available therapeutic treatment.

Why is Commercialization So Difficult?

There are many reasons why commercializing a therapy for rare/orphan diseases is so difficult, but they tend to fall into one or more of the below categories:

Timing. Most manufacturers start their commercialization and launch planning too late. Governmental approval and payer coverage can take years, something these patients don’t have. As a result, providing some type of “experimental access” program is often necessary to give patients access to treatment. Even when commercial access is granted, these types of programs can evolve into common “30-day starter” bridge programs.

Delays in the Patient Journey. Patients face unique challenges too. ​​For patients, the journey to an accurate diagnosis is bleaker than any number can reflect. It frequently takes years and many consultations and tests (by some estimates 6 years and 7.3 doctors on average), during which time they are passed from doctor to doctor, spending weeks and months waiting for appointments, tests, and test results. 

Operational Complexity. Distribution strategies for rare disease therapies are often far more complex compared to more traditional drugs. Emerging hybrid specialty/retail or “SpecTail” designs are increasingly common as are non-office-based settings (hospitals, clinics, etc). For cell-and-gene-based therapies the equation is even more complex with requirements for specialized treatment sites, data collection capabilities, immunotherapy manufacture, and shipment, etc. 

Limited Expertise. Usually, when a large company introduces such a treatment, it is entering the relevant therapeutic area for the first time. It is therefore likely to lack both expertise in the disease and in-depth understanding of the health ecosystem and of patients’ experience of the disease. Because the condition is rare, the launch team will have few, if any, analogs from which to draw lessons. And because the company may have bought the drug from a small biotech company at a late stage of development, it might allow too little time to prepare for its launch. In addition, Rare disease manufacturers run lean, making them more dependent on a consolidated vendor and system landscape than other organizations for innovation and operational support 

Healthcare Provider (HCP) Education. Patients with orphan and rare diseases often seek treatment in clinics where the condition has never been seen before, and absent, masked, misunderstood or confused symptoms can contribute to delayed diagnosis. For one-third of individuals with a rare disease, it can take between one and five years to receive a proper diagnosis.

Data Issues. Pharmaceutical manufacturers need to learn how to aggregate, refine, and analyze every piece of data that might help them better target specific healthcare providers (HCPs) and Integrated Delivery Networks (IDNs) who are treating these specific rare diseases. The small patient population means that more accurate insights and analyses are required and no data point is too small. Since the data set is small, the quality/accuracy of the data is even more important. Additionally, Benchmarking against other rare disease manufacturers is critical.

Financial Risk. Pricing (e.g, payer interactions, contract types, price protection) continues to be the #1 area of risk/concern for manufacturers. Orphan drugs tend to have more coverage restrictions than non-orphan drugs, leading to higher per-unit price. Under Medicaid Part D, 85% of plans covered orphan drugs, which were placed in the highest cost-sharing tier, only covering 25 to 33% of the full cost of the drug. Lastly, this is especially true for cell-and-gene therapies where their high costs and complexity of administration often create challenges for reimbursement agencies who need to assess safety and efficacy factors against value for money in terms of improvement in patients’ quality of life and the practicalities of rolling out these therapies into routine commissioning. 


What Can Manufacturers Do to Overcome These Challenges?

Start with the data. For emerging companies launching their first therapy, the key is to begin drug commercialization strategizing 2-3 years prior to targeted launch. This provides sufficient time to create a solid data management and governance strategy that can support Day 1 readiness and ongoing growth. More mature manufacturers are increasingly moving away from basic data aggregation capabilities (often provided by 3PLs) to more advanced, data science-driven solutions that can handle the broadening set of distribution models, provide deeper analytics and insights, and more rapidly identify areas for optimization. 

With truly actionable data, companies have the insight necessary to address many of the challenges listed above and are better able to answer questions like:

  • How long is it between diagnosis and treatment initiation? How does it vary by indication? Therapy? HCP?
  • Where are we seeing the need for patient intervention?
  • What are the main reasons for the lack of therapy initiation? 
  • What are the main reasons for poor adherence to therapy?
  • What trends are we seeing with payer restrictions?
  • How does the prior authorization activity compare with our contracted levels?

“Instrument” the Patient Journey. Companies need to identify the crucial leverage points and what reports, dashboards, and other visualizations are required to intervene and help prevent or ease roadblocks that can impede diagnosis and treatment. To address this, manufacturers need to have a clear strategy, long before launch, on how they are going to use data to “instrument” or monitor the patient journey. From diagnosis to first-fill to renewals and all points in-between, market access teams need to identify:

  • What data is necessary? How will it be used?
  • How frequently? 
  • From whom? 
  • In what format? 

Commit to Gross-to-Net Excellence. The small number of patients means the margin of error regarding accurate net-price forecasts based on projected diagnosed patients, from a financial standpoint, can be substantial. Many manufacturers underestimate the level of discounting, fees, free goods, copay programs, chargebacks, and rebates necessary to get a new rare disease therapy accepted in the marketplace. As a result, it is common for companies of all sizes to design and implement a comprehensive gross-to-net process and methodology to ensure an accurate, transparent view into net-price predictability. 

Empower Field Teams with Insights. Best-in-class companies are able to aggregate channel and patient data, refine it, flow it through their commercial data warehouse (CDW) and into their business intelligence tools in a day or so, a far cry from the weeks that many companies are still facing. This rapid flow of insight to the field means national account, reimbursement, and other field teams know patient- and HCP-level detail in a matter of hours. The end result is better and faster support of HCPs, trading partners, and patients.

Prioritize Cross-Functional Alignment. Cross-functional collaboration is important to ensure the timely, accurate linkage between the pieces of information accrued by medical and commercial field representatives and thus to generate the patient ecosystem insights required to create the launch strategy. A key element in achieving this alignment is to have a unified view of the contracting, pricing, inventory, patient status, and financial performance data.

Continually Add External Perspectives and Expertise. To address the combination of drug commercialization complexity and lean operations, manufacturers are turning to partners who can bring both strategic insights across all of the facets of market access along with operational excellence to the table. 


If you would like to gain more perspective on how IntegriChain can help solve the challenges associated with commercializing a rare disease therapy, download our ebook.

About the Author

Dave Weiss

Dave Weiss

Vice President, Industry Solutions

David Weiss, a software industry veteran, is charged with leading IntegriChain’s effort to provide pre-sales business consulting to life sciences manufacturers in the areas of needs assessment, analytics design, and value engineering. Prior to IntegriChain, David led the solutions and product marketing organizations at Model N, SAP, and IDS as well as spent five years as a management consultant for PWC, KPMG, and Knowledgent.