The launch of any new drug is complex, however, in rare disease it has its unique challenges. Here are our top four considerations to think about when planning for a rare disease drug launch.
Diagnosis is an odyssey
The challenge of diagnosis
By definition, rare diseases are rare. Not only is there a small patient pool to start, but also the majority of healthcare professionals are unfamiliar with rare disease and its diagnosis. Reports say patients see, on average, 7.3 physicians before receiving an accurate diagnosis. Thus resulting in a patient journey that is long and significant with extended delays to treatment, if available.
Educating patients, their caregivers and healthcare professional is an obvious first step to facilitating diagnosis. However, our experience is that having a thorough understanding of the diagnosis pathway will help focus efforts in areas that will be most impactful. This could be identifying a specific healthcare professional target or developing assessment tools that will aid in diagnosis. Improving the path to diagnosis is critical element to identifying patients quickly and expediting their access to suitable treatment.
A motivated patient audience
Possibly because of the challenge of diagnosis that patients and families endure, their engagement in their disease community is generally very high. They comprise passionate and committed advocacy organizations that provide a valuable support network and a voice for their rare disease community. These groups should be recognized as influential stakeholders in the rare disease ecosystem.
Working with grassroots advocacy organizations in advance of drug launch can help organizations better understand the patient pathway and identify areas requiring support. Developing a strong partnership is a first step in helping to establish the organization’s commitment to and support of the community.
Limited data platform
Often a challenge in rare disease is the breadth of clinical data that exists to support the brand. While in therapeutic areas such as cardiovascular there may be hundreds of clinical studies to reference, it’s not uncommon that in a rare disease there are only a handful. Ensuring treaters are familiar with the data and science is important to building confidence in the treatment.
Facilitating global knowledge exchange with experts around the world is one way to bring the information and expertise needed to the treaters in your market. Information gaps can be addressed through peer-to-peer education and the exchange of knowledge and experiences between treaters.
Access and reimbursement
Access to rare disease treatment is an ongoing challenge in Canada, as well as around the world. In rare disease, where treatment costs are generally very high in order to offset research investment across a small patient population, reimbursement is paramount if patients are actually to receive treatment.
Building a case for reimbursement is a multifaceted approach that in some ways can be described as “economics meets medicine.” A treatment is assessed based on its “value” and how it compares to other treatments, or lack thereof. This takes into consideration aspects such as cost-effectiveness and overall benefit to the patient. Engaging stakeholders such as medical economists, physicians, patients and their families in the process can help strengthen the case and hopefully result in positive recommendation for reimbursement.
About 1 in 12 Canadians, two-thirds of them children, are affected by a rare disorder. And right now, according to CORD (Canadian Organization for Rare Disorders) only 60% of treatments for rare disorders make it into Canada and most get approved up to six years later than in the USA and Europe. There is a need to increase this number, and with these four considerations in mind, it might be a bit easier getting your product to the people it can help the most.