Thirty million Americans, or nearly one in 10, have a rare disease. Of the 7,000 or so identified rare diseases, only 5% have FDA-approved treatments. But the pipeline for orphan drugs is strong. As it grows, so do the hopes of the thousands of patients, who have been waiting for breakthroughs their entire lives.
For manufacturers, this growing category represents opportunities to expand value and drive patient care forward. It also invites critical questions surrounding supply chain, commercialization and the overall impact on the patient journey. For example, rare disease patients can present in many care sites. So how can manufacturers facilitate broad access to medications while still balancing their own, internal concerns?
Availability Versus Accessibility
We see this particular scenario manifested as a paradox of availability vs. accessibility. The availability of the product does not always mean it is accessible for patients. Orphan drug manufacturers must consider not only how to make their products available in the market, but also how patients access them. Meaning, factors such as where patients present and where they might ultimately receive treatment should be considered. Reimbursement considerations may also drive where a patient can reasonably have access to a product.
Some manufacturers may lean toward limited networks in an effort to control the patient experience and develop actionable data that can be useful in telling a value story to payers or refining patient support programs. While well intended, channel decisions like these can lead to unintended consequences in the practical delivery of care.
For example, one hospital customer served as a clinical trial site for the development and ultimately successful FDA approval of a manufacturer’s product. Upon commercial launch of the product, the manufacturer chose a more restricted model for product access, which did not include this hospital (and other investigative sites that participated in the trial). The hospital expressed frustration that it would not have access to the product post-launch, despite its involvement in product development.
The hospital also articulated how many of these restricted models put the most novel and complex products further away from patients’ preferred site of care. This is often counterintuitive to ensuring a patient’s healthcare provider has product access that enables ongoing treatment and close monitoring of the patient’s progress. These are some of the unintended consequences of restricted models that may not be considered in product commercialization decisions. The patient journey, customer convenience, and workflow for patient access are all important considerations. Workflow changes create inefficiency and place additional operational burden on already stretched teams. It begs the question: What’s the value in creating greater burden for providers and patients?
Working with a knowledgeable partner that brings real-time customer and market insight from product launch all the way through market maturity can help manufacturers understand the patient journey and appreciate the role that physicians and pharmacists play in patients’ treatment experiences. Partners that take a disciplined approach to understanding their customers’ experience will help manufacturers gain a richer understanding of how their channel strategies help or hinder healthcare providers’ ability to bring the full benefit of pharmaceutical innovation to their patients.
Patients with rare diseases often are burdened with comorbidities that require multiple treatments and multiple touchpoints with a provider. When seeking to commercialize an orphan drug, manufacturers should consider comorbidities associated with their treatment’s intended indication and how these conditions could affect the patient journey.
Patients might want to go to their preferred pharmacy, or their most appropriate site of care could be a local hospital. Providing options for patients creates more than just choice—it promotes adherence and improved outcomes. For example, a patient with Cushing’s disease who also suffers from cardiac issues like hypertension may remain more adherent if her specialty treatment can be accessed at the same time as her hypertension medication
Conversely, if patients must visit multiple sites of care to receive all of their treatments, drug interactions could be overlooked. Side effects become more difficult to manage. Adherence can be compromised—ultimately affecting outcomes. Intensive coordination should be the new normal, with manufacturers designing their strategies to work toward that goal.
Often a hub model, supported by a case manager approach, is an effective way to help rare disease patients overcome prior authorization requirements and manage affordability constraints. When considering the patient experience and building a channel strategy, key questions to ask include, “Where does the patient present?” and “What’s in each patient’s best interest?” Moreover, following through to “Where are patients receiving care?” and “Will that change over time?” or “What sites of care are relevant for the product’s next potential indication?” A successful patient journey will help yield the positive outcomes that generate critical data to support a therapy’s overall value proposition.
The Value of Data
Data is critical for new, complex drugs—and even more so for those facing pricing scrutiny. The specific, small patient populations associated with rare diseases mean payers might not have a comprehensive view of the total costs of care, making it more important that manufacturers demonstrate the impact of their products in real-world populations and not only in the controlled setting of trials. Manufacturers need to invest in a data approach that will show health economic benefits and prove value beyond the clinical trial stage. The right data and analyses can show the cost of a new treatment compared to the patient’s current costs in the health care system, what costs can be avoided and how quality of life is improved. A well-rounded commercialization partner will have expertise in gathering and interpreting these data sets to create the most compelling value story. This is even more important as rare disease products face increasing competition.
The Impact of Competition
Often a manufacturer’s channel strategy in the orphan drug market is also influenced by potential competition from new treatments entering the market. The more innovative the product, the more control in shaping the market a manufacturer may assume it has. But that is only true until the product faces competition.
While most orphan products are unique and do not go to market in a crowded basket of products for a single indication, there is a rich pipeline in rare disease. Competition will increase. Then, the portion of the market that was not included in the originator’s channel strategy—whether hospitals, specialty pharmacies, or ultimately patients whose voice is often heard through active patient advocacy groups—could advocate switching to an alternative. By working with a partner to design a product and patient-specific channel strategy at least 18 months prior to launch, manufacturers can anticipate and design an optimal plan. The right partner can help develop a commercialization strategy that meets the needs of the patients and providers who will use their products, which then promotes product success and longevity.