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Rare Diseases: Building Long Term Relationships


Therapeutic companies that work with rare diseases must take early steps to build relationships with patients, caregivers, and HCPs.

Natalie Douglas

It is widely recognized that people living with a rare disease have significant obstacles on their path to the right treatment. The diagnosis process is often slow as more common diseases are “ruled out” one by one. Once a rare disease is diagnosed, informational and other resources are often limited, and (in some cases) access to therapy may be restricted.

Companies that develop and commercialize therapies for the treatment of rare diseases must take steps as early as phase II of clinical trials to build an enduring relationship with patients, caregivers, and physicians. This article outlines two key actions—the development of both a Managed Access program and a “concierge” approach to support and service—and the recommended timing.

Phase ll: Development of a Managed Access Program

Mary Anne Greenberg

Once a rare disease is diagnosed, informational and other resources are often limited, and (in some cases) access to therapy may only be available through clinical trial or a Managed Access program.

Early planning ensures a Managed Access program will be in place once the product is approved and commercialized. This maximizes the program’s significant benefits, including providing product to patients who otherwise could not receive it—either because they do not fit the clinical protocol or live in a country where it is not yet approved.

In addition to helping patients, Managed Access programs can also be beneficial as strategies to collect data and ultimately obtain country-by-country approval for the product. Companies can benefit from having a partner to navigate each country’s regulatory landscape and help deliver Managed Access (and medicine management) programs in an effective and compliant way.

Phase lll: Development of a concierge approach

For patients

When a clinical trial for a rare disease therapy enters phase lll, it’s time for the company to begin active planning for the post-approval handling of their Medical Information, Pharmacovigilance, and Patient Support solutions. The availability to interact with patients (and caregivers) should be in place and ready-to-go as of (and contingent upon) the product’s approval date.

Rare diseases by nature have smaller patient populations, with generally fewer patient and caregiver resources to tap into. Applying a concierge team approach is valuable, as it ensures that every element of patient support is handled in an integrated way, by a dedicated team—including medical information, reimbursement assistance, and general customer care support. A portion of the team, which typically reports into the company’s Medical Affairs group, is composed of nurses and pharmacists who have extensive knowledge about the disease state and the drug. Other team members, who may report to commercial contacts within the organization, handle the reimbursement approvals and general customer support. All are trained to recognize adverse events and handle reporting according to company and regulatory guidelines.

The concierge team is there to educate and provide support through every aspect of the patient’s journey—answering question they have at the beginning of therapy, recognizing and coping with potential side effects, affordability concerns, and any other issues or obstacles that may arise along the way.

The concierge team must be available to meet a multitude of needs, and must do so using multiple channels (e.g. calls, email, video, chat, and text). Patients newly-prescribed a self-injection drug may want to make their first couple of injections while on a video call. Patients traveling out of town can text a member of their team to ask a quick question about how to maintain optimal storage temperatures. A patient who has a question about their prescribed drug’s compatibility with a certain type of food may benefit from a chat on the phone.

The concierge team approach provides patients with the confidence to adhere to their therapy. It also provides caregivers with information they need to be an effective source of support to the patient.

For HCPs

The concierge team also extends their reach to the patient’s health care team. Specialists may have treated few (or even no) patients with the disease, and it can be challenging for them to recognize an expected side effect or other nuance of the disease or therapy. Having the concierge team as a resource eases the burden on the specialists and supports a good outcome for patient.

An added benefit for HCPs is the inclusion of PharmDs who can act as “virtual” MSLs. HCP demand for the services of MSLs is expected to grow by a minimum of 20 percent in the next three years. And because of busy schedules and COVID-19, HCPs—including those that treat rare diseases—are increasingly requesting virtual engagements.

One overarching point: A non-siloed approach to the support of patients and HCPs is advantageous, and (because of their size) is easier for rare disease companies to implement and maintain than it is for larger companies.


As companies launch clinical trials for a rare disease therapy, early and ongoing planning about the programs that will be in place post-commercialization is crucial for creating enduring and outcome-focused relationships with patients, caregivers, and HCPs.

Natalie Douglas, Co-Founder, RareiTi, ndouglas@rareiti.com, Mary Anne Greenberg, CEO, Diligent Health Solutions, LLC, mgreenberg@diligenthealthsolutions.com

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