Rare Disease Situation Analysis is aimed at providing a high level glimpse into the rare disease drug development and commercialization. Rare disease drug development has been an increasingly attractive proposition for biopharmaceutical companies.

In this report, we have analyzed and showcased the 2008-17 trends, the current situation and most recent FDA guidance into perspective. We conclude by looking into the emerging rare disease drug archetypes, changes in FDA’s regulations and proposing a collaborative commercial model that’s likely to be more competitive and successful than the traditional monolithic commercial model that biopharmaceutical companies tend to deploy.

10 Points Analysis and Key Take Away

1. Cost to Compete: Beyond the standard R&D, filing and launch expenses, an OD developer has to account for the expenses associated with market preparation including but not limited to science, patient identification, diagnostics and patient endpoint/outcomes management
2.  Business Model: A successful developer has to shift its business model from a monolithic structure to a patient centric, decentralized model that is predicated on partnering throughout the bench-to-market value chain
3.  Commercial Model: it requires a cradle-to-grave one-stop-shop model predicated on expertise in biology, clinical and healthcare aspects of the disease, including supply chain and patient management
4.  Need of Early Diagnosis: Along with comprehensive awareness campaigns, the sponsors need to adopt innovative technologies to identify yet-to-be diagnosed patients and ensure early, accurate diagnosis
5.  Clinical development: It requires a significantly higher level of analytics driven patient and physician identification, outreach, recruitment and engagement
6.  Access to Capital: Given the „orphan” status of the disease (i.e. high unmet need) and a highly targeted approach towards disease biology, a rare disease “venture” is likely to be well capitalized across the bench-to-market value chain
7.  Higher Probability of Success: ODD development is likely to be successful because of
   1. Prior marker based identified patients
   2. A well-defined map of patients, physicians and KOLs
   3. Pre-IND interactions resulting in better trial designs
   4. And a well-established network of Patient Advocacy Groups
8.  Market Incentives: At least in the US, FDA has created a highly negotiable regulatory pathway to encourage parallel/deferred development (acknowledging the difficulties associated with natural history studies, patient identification, disease biology, lack of treatment options) along with tax credits and seven year market exclusivity
9.  Competitive Pressure: given that the bench-to-market value chain, Center of Excellence driven commercial model and a likely rapid revenue plateau (which could run counter to Big Pharma Shareholder Return Pressures)
10. Market Accessibility: Given that the ODD development generally accompanies a well-defined patient population, facilitated regulatory path and prevalent unmet needs, we anticipate a relatively easy price negotiation