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Do's and Don'ts for Orphan Drug Support Programs

By Nancy Pilcher

Complex. It’s the word most often associated with the orphan drug market. Commercialization success for rare disease drugs isn’t easy. There are challenges and subsequent risks from development through post-launch. But there are also proven solutions for each obstacle—solutions that enable the right commercialization partner to help manufacturers succeed where complexity exists. I work with experts every day who leverage our experience with orphan drug manufacturers for a deep understanding of obstacles, challenges and best practices for commercializing rare disease products. And as it relates to building patient support programs for rare disease patients, we’ve simplified the process with a few key do’s and don’ts.

DO have a patient-centric adherence plan. 

The often high costs and lifelong treatment associated with orphan diseases can exacerbate the negative health psychology that leads to medication non-adherence in rare disease patients. Traditional adherence models focus on the product and its side effects, typically supported by a frequent and repetitive communications plan between clinician and patient. But more recent support models focus not on the product’s attributes, but on the patient experience. 

Built upon patient empathy and empowerment, these individualized adherence solutions take into consideration the patient’s understanding of how their rare disease is treated and their willingness to support their therapy. It’s less about monitoring patients, and more about engaging them in their own healthcare. Equally important, these adherence strategies bend to the path of the patient journey, which for rare disease patients can have many turns and touchpoints. Successful adherence programs focus on educating patients on their disease and connecting them with advocacy groups or charities. These offerings can aid in curbing feelings of isolation, bridging affordability barriers, managing modifications in therapy or transitioning from hospital-based treatment to home care.  

Approximately 50 percent of people affected by rare diseases are children.

Perhaps most unique to patient-centricity for orphan drugs is the critical role of the caregiver. Approximately 50 percent of people affected by rare diseases are children, 30 percent of whom will not live to see their fifth birthday.1 These alarming numbers emphasize that not only will communication and education take place primarily with a parent or other caregiver, but that the caregiver will have significant emotional needs that must be met as well. Programs should be built with the caregiver in mind.

DON’T leave anything to the imagination as far as the level of support a patient needs. 

When one considers the very small patient populations inherent to rare diseases, it’s understandable that every single patient would mean the world to an orphan drug manufacturer. This, compounded by the fact that patients with rare disease often have few places to find support, should compel manufacturers to provide a higher number of wraparound services on a per patient basis than their traditional small molecule counterparts. As well, considering how little time providers may have to explain support offerings, partnering with advocacy groups can prove invaluable. These groups can spread the word about the value of programs; they can also be important collaborators in patient-centric program design.

While robust support services require more investment, they also deliver more value—particularly when it’s a case management support program or hub model. The most successful programs offer rare disease patients access to a knowledgeable, single point of contact from the very beginning of the treatment journey.

Successful programs offer access to a single point of contact from the very beginning of the treatment journey.

That role can be clinical (e.g., a nurse navigator to help manage side effects), a reimbursement professional or an experienced social worker. This role can take on a variety of activities from patient benefits verification and prior authorization to managing site of care operations by overseeing the education and training of clinicians to ensure the drug will be delivered correctly or coordinating home-health visits from a qualified nurse—whichever best meets the ongoing needs of the patient.

Whatever the mix, the message manufacturers convey to patients through these support services is consistency. After all, in the lifespan of the rare disease patient, many things may change—their location, their physician, their insurance. The branded program never changes, nor does the 1-800 number patients use to access its benefits. In this way, the patient support services make up the bones of the orphan drug brand, one which patients can enjoy a direct and meaningful relationship with throughout the treatment journey. 

DO consider site of care.

Site of care is an important component when building support programs for orphan drugs. Field nurses or clinical educators can be engaged and put it into the field as an extension of the hub to provide patient education, site education and pull-through to ensure there aren’t going to be any limitations to access when split coverage happens. As some payers cover orphan drugs under the pharmacy benefit and others put them under the medical benefit, manufacturers want to be sure that they are positioned to support both types of coverage as well as the sites of care that manage drug purchasing.

Transitions in site of care are common occurrences in the rare disease patient journey. As such, manufacturers will want to ensure patients have support through transitions from hospital to home and associated transitions from medical to pharmacy benefits, offering services like bridge programs to fill gaps in coverage.

Field representatives are the community representation of the manufacturer’s brand and product, and need to be directly connected with the hub program to make sure that they are aligned with the message and communications. This will ensure they can be the first line of defense in addressing affordability issues (field reimbursement specialists) and access barriers (field nurses and clinical educators).

DON’T ignore affordability issues.

The average drug cost per patient, per year was almost $112,000 in 2014.2 While that alone is a daunting figure, it can grow exponentially based on where the patient is relative to the location of the specialty physician, site of clinical administration or specialty pharmacy. For this reason, it’s critical orphan drug makers address every affordability issue, big or small, that a rare disease patient might face. 

That means providing commercial co-pay assistance as well as supporting a foundational strategy for patients who are covered by government insurance. Additional assistance related to travel reimbursement or time lost from work should also be accounted for. Finally, a contingency plan: a free-drug distribution program for those rare disease patients who don’t qualify for financial assistance, but desperately need access to the orphan drug. 

The ultimate DO: be a qualified partner.    

Whether an emerging player or a card-carrying member of big pharma, no one organization has the in-house expertise required to effectively support every aspect of a support program for rare disease patients. Aligning commercialization goals with internal resources and experience can be a humbling event for many pharmaceutical companies. The right partner, enlisted early in the commercialization process, can help a manufacturer map the patient journey, build a program that supports patients and propels product success, and determine the right distribution strategy to ensure appropriate access.  


1 Rare Diseases: Facts and Statistics. Global Genes. Accessed November 2016. Available online at
2 Orphan Drug Report 2015. EvaluatePharma. October 2015. Accessed October 2016. Available online at


About The Author

Nancy Pilcher
Director, Business Development
Lash Group
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