Orphan disease Consultants

Orphan drugs are making great strides in the treatment of rare, often previously untreatable diseases while offering attractive business opportunities for biopharma companies of all sizes. Since the passage of the Orphan Drug Act in 1983, more than 350 orphan drugs have been approved in the US with over 2000 orphan designations granted. Previously the domain of smaller companies, industry leaders have increasingly established dedicated orphan drug units.

Although the populations they serve are comparatively small, orphan product developers face increasing competitive pressure in an industry segment that until recently has been largely overlooked. Today, expectations have been raised as manufacturers of orphan drugs compete for the same resources as traditional products.

Developers must be attentive to balancing the interests of patients, regulators, payors and shareholders alike while safeguarding against any action that may compromise future product value. Infusion Pharma Consulting can help.

Orphan Drugs – Distinct Commercial Challenges

Orphan products present fundamentally different commercial challenges than those of traditional biopharma products. Patient markets are relatively miniscule, often poorly characterized and difficult to gauge and value accurately. The commercial tools and strategies routinely used for traditional products are often poorly suited for orphan and ultra-orphan drugs.

Pricing and market access pose especially critical challenges. With research costs spread across relatively few patients, required prices are invariably higher and can be the source of intense public scrutiny. Successful orphan drug pricing strategies must recoup large, high-risk research investments, ensure patient access regardless of financial situation, and be recognized as equitable and justifiable by key stakeholders.

Further commercial challenges abound. Patients are often misdiagnosed, delaying therapy. Stocking of rare use products may be problematic. Orphan products are frequently injectable, with advanced patient support required to realize successful outcomes. Specialty channels of distribution may be required. And since many rare diseases are treated by a small core of disease specialists but initially diagnosed by far more, the level and scope of commercial initiatives must be calibrated appropriately.

Infusion – A Decade of Orphan Experience

For over a decade, Infusion consultants have provided strategic direction for some of the most challenging orphan and ultra-orphan drug products worldwide. Our experience includes products administered in the hospital, in the physician’s office, and in the outpatient setting and span almost every reimbursement situation.

We have developed and continue to refine distinctive approaches to orphan drug pricing and access strategy. But, experience has also taught us that every orphan drug situation is unique and that biopharma companies can easily go very wrong when they try to imitate others

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