What We Do

Health Advances has built and maintained a strong understanding of the different development and commercialization challenges faced by companies targeting orphan markets. Our involvement in orphan diseases dates back to the late 1990s when the orphan disease field was still nascent. To assist clients in developing strategies to succeed in orphan diseases, Health Advances leverages its vast project experience and key relationships both with industry and patient advocacy organizations. Health Advances has access to top key opinion leaders across markets as well as payers to provide insights into treatment and diagnostic paradigms, unmet needs, and pricing and reimbursement considerations. Health Advances’ consultants are thought leaders in the orphan area through their publications, moderation of panel discussions, and participation at top orphan drug and biopharma events.


Carrie Jones, Manager: “Health Advances recently supported a client that is preparing to launch an orphan drug which will be the first approved therapy for its target disease. We benchmarked pre-launch disease awareness and education campaigns and marketing tactics across six different markets to provide our client with ideas on what they could do to educate the market about their orphan disease before their drug’s launch.”

Andrew Funderburk, Partner: “Our client was questioning how they should price their orphan drug, especially since it was a cellular therapy with unique dosing considerations. To guide their decision, we did a detailed system economics analysis for them looking at the potential cost savings their drug would offer in addition to talking to payers and benchmarking other relevant orphan drug price points.”

Carrie Jones, Manager: “Health Advances developed a worldwide market forecast for a cell-based gene therapy targeting an ultra-orphan indication. Countries were classified based on standard of care, reimbursement considerations, and regulatory processes. The team combined epidemiologic data from multiple sources with physician-level patient volumes from interviews to establish the number of eligible patients in each country. The resulting flexible model allowed the client to evaluate various commercialization and distribution strategies. In addition, the team advised the client on the unique manufacturing and distribution requirements for their therapy.”

Indications of Interest

Lysosomal storage disorders (Gaucher disease, Fabry disease, Pompe disease, MPS1)
Pulmonary arterial hypertension
Idiopathic pulmonary fibrosis
Paroxysmal nocturnal hemoglobinuria
Hereditary angioedema
Alpha-1 antitrypsin deficiency
Cystic fibrosis

Urea cycle defects
Non-tuberculous mycobacterium
Cushing’s disease
Excessive Daytime Sleepiness
Numerous orphan oncology indications

Related Areas

You may also be interested in our expertise in oncology and biopharma.

View our Orphan Diseases case examples.

Contact the Biopharma Team