February 28 marks Rare Disease Day – a global event to raise awareness for the 300 million people worldwide living with rare diseases, their families, and their caregivers. As part of Avalere Health’s commitment to rare disease patients globally, we are proudly partnering with the National Organization for Rare Disorders (NORD), the leading advocacy group for rare diseases in the US, sister organization to EURORDIS-Rare Diseases Europe.

NORD was set up by rare disease patients and caregivers, who were instrumental in the passage of the US Orphan Drug Act. The Act is celebrating its 40th anniversary this year and since its inception, more than 7,000 rare diseases have been identified and over 1,100 orphan indications for treatments have been approved by the FDA.

At Avalere Health, we are proud of our steadfast dedication to rare disease patients and caregivers. Across our disciplines and geographies, our teams work on initiatives to solve unmet needs and support our clients in improving outcomes for patients.

We champion therapies grounded in the most exciting scientific discoveries

In addition to being deeply meaningful work, rare diseases are an area of continued growth, exploration, and evolution. As our own Dan Zaksas told PharmaLive, “The sheer number of diseases being addressed and therapies in development are pretty staggering compared to where we were even a decade ago. A third of the industry’s R&D pipeline is now in rare disease, and we’ve had almost 400 therapies approved exclusively in orphan indications. So, this is great news for patients who have waited for years or decades to be diagnosed or be treated, but there are also new challenges.”

Among these challenges is the pace of change, and we know physicians and other healthcare professionals are hungry for information. They’re being asked to understand, diagnose, and treat many more diseases, including diseases that they might have heard mentioned once at a lecture in medical school—or never! Our work with therapies at the cutting edge of innovation supports those at the front lines supporting rare disease patients, caregivers, and the wider community affected.

We develop strategies to educate and create awareness

From building the right story for the right audience, through insight from multi-stakeholder audiences to excellence in publications and omnichannel delivery, Avalere Health is a key partner in helping clients navigate the complex landscape of rare diseases and build immersive programs designed to help healthcare professionals better understand a disease.

For patients and caregivers, we have developed practical, easy-to-understand disease state and patient support programs to help guide them toward more empowered patient journeys.

In multiple instances, we’ve supported our clients’ commercialization plans with development of healthcare professional and patient brand launches, helping clients to establish leadership in numerous rare disease categories.

Earlier this year, our team developed a comprehensive launch experience for a new oral Amyotrophic Lateral Sclerosis (ALS) treatment, telling the story of how treatment could easily fit into a patient’s daily routine and allow them to keep experiencing moments that were vitally important in their lives.

We support our clients to build the strategies and solutions needed to drive connections with the community

Across a host of rare diseases—spanning hematology, pulmonology, central nervous system (CNS), endocrinology, cardiology, and ophthalmology—our Consulting Capability team members are supporting clients to gain crucial insights into the experiences of patients and healthcare professionals, including analysis from clinicians across our Expert Partnership in these complex and data-heavy disease areas.

Building on these insights, we develop launch and portfolio strategies and solutions, including supporting our clients to build internal capabilities that drive connections with the community and improve outcomes for rare disease patients.

We partner with clients to ensure life-changing therapies reach the hands of those who need them

We must uphold two key pillars when doing this: health equity and access.

Health equity means ensuring rare-disease patients are able to obtain and afford life-changing treatments efficiently—the same as any other type of patient. This includes ensuring our clients’ products are covered and reimbursed sufficiently.

To help ensure equity, our colleagues helped a client understand the evolving reimbursement landscape—including coverage, reimbursement, and coding considerations across sites of care—for cell and gene therapies (CGTs) ahead of a product launch.

The other pillar, access, means making sure a treatment is available to be prescribed to a patient who needs it.

To help with access, colleagues helped to develop lifecycle management strategies for two rare disease brands. These strategies played a part in the client to seeking label and formulation expansion which expands access to target patient population.