Orphan Drug & Rare Disease Specialists

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

Special Resources for Orphan Markets

Infusion’s orphan drug effort is underpinned by a senior-level consulting team thoroughly experienced in the orphan and ultra-orphan drug markets. We bring the insight, experience and judgment required to help biopharma management teams succeed in this complex environment and to bridge internal differences of opinion in a setting where few hard guideposts are available.

Our consultants speak at major orphan drug meetings and are in ongoing contact with payors, providers, channel members, regulators and others important orphan drug decision makers. Infusion consultants are backed with a proprietary orphan drug pricing analytics database, a real-time set of orphan product candidates, and
orphan-specific tools to support client engagements.

Infusion - Support For Your Orphan Drug

Whether you are an experienced orphan drug manufacturer or embarking on your first product, we offer a range of services to support your efforts. These include:

  • Orphan Drug Market Sizing – Determine the size and structure of the addressable market and the realistic prospects for diagnosis, uptake and promotional reach requirements.
  • Orphan Strategic Pricing and Market Access – Develop orphan pricing recommendations grounded in robust market and value assessment. Provide reimbursement, channel access and market preparation planning support. Guidance on the strategic repricing of in-market brands where required. Substantiation of value for key stakeholders.
  • Multi-Indication Brand Optimization – Optimize the value of products with two or more potential orphan/non-orphan indications where different dosages, price/value points, and market potentials may apply. Provide guidance on dosage form(s) development strategy.
  • Business Development – Support in-house business development efforts in the search and screening of orphan product opportunities. Commercial due diligence and valuation.

For additional information on Infusion Orphan Drug services, please click here