Rare diseases, often called orphan diseases, pose unique challenges for patients and pharmaceutical companies. With limited patient populations, developing effective treatments for rare diseases requires a distinct approach. Pharmaceutical commercial models for rare diseases must be innovative, patient-centric, and collaborative to address the complex nature of these conditions. In this article, we will explore the intricacies of the pharmaceutical commercial model for rare diseases, considering key definitions, challenges, and the considerations that set these diseases apart.
The Orphan Drug Act defines rare diseases as those affecting fewer than 200,000 individuals in the United States.1 This designation incentivizes pharmaceutical companies to invest in developing treatments for these conditions, including tax credits and extended market exclusivity. About 7,000 rare diseases have been identified globally, with an estimated 350 million people affected.2 Despite that number, each disease—individually—impacts a small patient population and is often challenging to diagnose.
There are four key stakeholders:
# | Stakeholder | Challenges | Mitigation Strategy |
|
Patients | Limited treatment options, access issues | Patient assistance programs, advocacy groups, and increased awareness |
|
HCPs/hospitals | Limited expertise, diagnostic challenges | Education, collaborative partnerships, and specialized training programs |
|
Payers | Cost concerns, limited evidence | Value-based pricing, evidence generation, and collaboration on access initiatives |
4 |
Patient advocacy groups | Limited resources, awareness | Partnerships with pharmaceutical companies, awareness campaigns, and advocacy for policy changes |
Pharmaceutical companies should focus on patient-centricity while designing different customer-facing roles. Specialized focus and a deep understanding of the product or therapy are paramount while creating a commercial model. Some key responsibilities that commercial or medical teams need to cater to are:
Demand generation: Engage with the doctors and hospitals and drive sales . Many rare disease products (especially in the oncology domain) can only be administered in specialized hospital settings, making account management one of the critical skills of field personnel.
Support or service: Support the customers in the process and challenges of drug treatment, administration, and reimbursements.
Payer-oriented: Engage with the payers and get access to the drug.
Medical expertise: Engage with Key Opinion Leaders (KOLs) and high-value prescribers on deep scientific discussions about different treatment paradigms.
Striking a balance in resourcing is critical as overinvestment could make the operations financially unviable, while underinvestment would lead to a significant loss of opportunity. Understanding the addressable market and related physician networks is crucial for effective resource allocation and deployment.
The geographical element or size of the territory also needs to be baked in while deciding the final Full-Time Equivalents (FTEs). The territory of sales reps in this therapy area will be massive compared to the traditional diseases.
Market access, competition, and ease of diagnosis are other vital factors determining appropriate resourcing levels across roles.
Commercializing via co-promotion and Contract Sales Organization (CSO) collaborations can help optimize resources and increase market penetration.
Orphan drug designation: Seeking orphan drug designation can incentivize pharmaceutical companies, including extended market exclusivity and reduced regulatory fees. This encourages investment in research and development for rare diseases.
Expedited regulatory pathways: Collaboration with regulatory agencies to accelerate the approval process for rare disease treatments is vital. Specialized regulatory pathways, such as the FDA's Breakthrough Therapy Designation3, can accelerate the development and commercialization of promising therapies.
The pharmaceutical commercial model for rare diseases requires a unique and adaptive approach. As the industry evolves, it is essential to recognize the distinct challenges and opportunities of rare diseases and continue innovating to improve patient outcomes. By embracing patient-centricity, fostering collaboration, and leveraging advanced analytics, the pharmaceutical industry can make significant strides in solving the complexities of rare diseases and delivering much-needed therapies to those living with the conditions.