Introduction

For specialty pharmacies, winning a rare disease drug contract is not just about expanding business - it’s about solidifying your role as a key healthcare provider, ensuring sustainable revenue, and making a direct impact on patient lives. But with increasing competition, evolving reimbursement models, and pharmaceutical manufacturers becoming more selective, securing these contracts requires more than just operational capabilities - it demands strategic positioning, patient engagement, and data-driven value demonstration. 

Background

The landscape of specialty pharmacy is evolving rapidly, and nowhere is this more evident than in the rare disease space. As pharmaceutical companies develop groundbreaking therapies for conditions affecting small patient populations, the role of specialty pharmacies has never been more critical. Winning a contract to distribute these life-changing medications requires pharmacies to demonstrate unparalleled patient care, operational excellence, and strategic alignment with drug manufacturers' goals.

Larger, established specialty pharmacies are facing increased competition from smaller, independent pharmacies. Securing exclusive or preferred distribution contracts can mean long-term sustainability, increased market credibility, and a deeper impact on patients who rely on high-touch services. Competition in this space is fierce and pharmaceutical companies are selective. 

As of 2023, there were approximately 1,651 accredited specialty pharmacy locations in the United States, with the market expected to expand rapidly at a projected compound annual growth rate of 35.4% from 2022 to 2027 [8]. Pharmaceutical companies seek partners who can ensure seamless patient access, provide real-time data on adherence and outcomes, and navigate complex reimbursement landscapes. Specialty pharmacies need to continuously iterate and improve their strategies to successfully compete for and win rare disease drug contracts. 

We’re here to help - with this guide you can explore the factors that influence manufacturer decision-making, the critical capabilities that set winning pharmacies apart, and actionable steps to strengthen your positioning in this competitive market.

Whether you’re an independent pharmacy looking to break into the rare disease space or an established player aiming to expand your portfolio, this report will provide the knowledge and tools necessary to thrive in an industry where every partnership can change lives.

Challenges in bidding for contracts

Specialty pharmacies face a unique set of challenges when bidding for contracts. These challenges stem from the complex nature of rare diseases, the high cost of orphan drugs, and the specialized services required to support patients effectively. To better understand these challenges in order to overcome them, let's examine in more detail:

Limited market size

• The small patient populations associated with rare diseases create a limited market for orphan drugs, making it challenging to predict sales and revenue.

• This can affect a pharmacy's ability to demonstrate financial viability and secure contracts.

Complex distribution needs

• Many rare disease treatments require specialized handling, storage, and delivery, posing logistical challenges for pharmacies.

• Ensuring timely and efficient distribution to geographically dispersed patients is crucial for patient care and contract fulfillment.

High drug costs and reimbursement issues

• Orphan drugs often come with high price tags, leading to concerns about affordability and reimbursement.

• Pharmacies need to navigate complex reimbursement landscapes and demonstrate value to payers to secure contracts.

Competition from larger pharmacies

• Specialty pharmacies may face competition from larger pharmacies with greater resources and established networks.

• Differentiating their services through personalized service, deep community ties, or a focus on specific rare disease areas, and demonstrating specialized expertise is essential for bid success.

Value-based care models

• The shift towards value-based care models presents challenges in the rare disease space because the small patient populations can lead to statistically insignificant or highly variable results when assessing the value and impact of treatments. This makes it difficult to demonstrate consistent, predictable outcomes necessary for these models.

• Pharmacies need to develop strong value propositions that demonstrate the clinical and economic benefits of their services.

Regulatory challenges

• Pharmacies must be mindful of FDA regulations on pre-approval communications and product promotion when interacting with patient advocacy groups.

• This includes providing clear information on the benefits and side effects of treatments while adhering to guidelines around interactions with healthcare providers.

• Additional regulatory considerations include HIPAA compliance, state pharmacy regulations, and requirements for handling specialty medications.

Best practices for enhancing bid success

To enhance bid success, specialty pharmacies must focus on patient-centric care, specialized expertise, and strategic partnerships. Below are key best practices with real-world examples.

1. Patient-centric approach

A winning bid strategy prioritizes patient needs, improving outcomes and strengthening payer relationships.

Key tactics:

• Develop personalized patient support programs for adherence and education.

• Integrate digital health tools (AI reminders, remote monitoring) to enhance engagement.

• Partner with advocacy groups to align services with patient needs.

Example: UVA Health Pharmacy

• Specializing in rare neurological and rheumatologic conditions, UVA Health provides tailored support, fostering strong payer partnerships and improving treatment success.

2. Specialized expertise

Expertise in rare disease management differentiates specialty pharmacies in a competitive market.

Key tactics:

• Highlight specialty accreditations (e.g., URAC, ACHC) and staff certifications.

• Collaborate with key opinion leaders (KOLs) and research initiatives.

• Offer specialized pharmacist-led support and medication management.

Example: Amber Specialty Pharmacy

• Known for disease-specific education and adherence programs, Amber strengthens provider collaboration and payer trust, enhancing bid competitiveness.

3. Strategic partnerships & branding

Building relationships with manufacturers, payers, and advocacy groups boosts credibility and market presence.

Key tactics:

• Showcase expertise in managing rare disease therapies to secure limited distribution drug (LDD) access.

• Provide robust adherence tracking and therapy persistence data to payers.

• Support disease-awareness initiatives to enhance credibility and patient trust.

Example: PantherRx Rare Pharmacy: 

• PantherRx excels in LDD access, payer collaboration, and patient advocacy partnerships, securing contracts for ultra-rare therapies.

4. Innovative drug pricing models

Pharmacies can differentiate themselves through value-driven pricing structures.

Key models:

• Outcomes-Based Agreements (OBAs): Payment tied to patient outcomes.

• Subscription Models: Fixed pricing for broad patient access.

• Risk-Sharing Agreements: Manufacturers absorb financial risk for non-responsive treatments.

• Financial Assistance Programs: Co-pay assistance and patient support increase adherence.

Example: Novartis' Kymriah

• Novartis ties reimbursement to CAR-T therapy effectiveness, requiring specialty pharmacies to track real-world patient responses.

5. Leveraging technology & data analytics

Data-driven insights improve operational efficiency and bid credibility.

Key tactics:

• Implement AI-driven adherence tracking to optimize patient engagement.

• Utilize blockchain for secure, transparent drug supply management.

• Integrate with electronic health records (EHRs) for seamless payer-provider communication.

Example: Orsini Specialty Pharmacy (ORBIT System)

• ORBIT tracks adherence, collects real-world data, and automates reporting for enhanced contract negotiations.

6. Patient-reported outcomes (PROs)

Capturing PROs strengthens payer and manufacturer partnerships by proving real-world therapy effectiveness.

Key tactics:

• Deploy digital tools for real-time patient feedback.

• Utilize validated PROMs (e.g., EQ-5D, PHQ-9) for structured data collection.

• Showcase PROs in bid proposals to highlight improved health outcomes.

Example: Optum Frontier Therapies

• Optum integrates PROs into patient support, improving treatment success and payer engagement.

7. Financial assistance support

Helping patients access cost-saving programs enhances adherence and payer relationships.

Key tactics:

• Offer dedicated financial counselors to navigate co-pay assistance and grants.

• Partner with manufacturers and advocacy groups to streamline financial aid.

• Advocate with payers to secure better reimbursement models.

Example: Accredo Health Group

• Accredo integrates financial assistance with clinical services, ensuring affordability does not hinder treatment.

By adopting these best practices, specialty pharmacies can strengthen their bids, secure more contracts, and position themselves as trusted partners for manufacturers and payers in rare disease management.

The importance of patient communities in the bidding process

Patient communities are not merely recipients of care; they can be active partners in the healthcare ecosystem. Their involvement in the bidding process can significantly enhance a pharmacy's chances of success. By demonstrating a strong understanding of patient needs, preferences, and priorities, pharmacies can create bids that resonate with decision-makers and showcase their commitment to patient-centric care [12].

Engaging with patient advocacy groups and incorporating their input can provide valuable insights into the patient journey, treatment challenges, and unmet needs [13]. This information can be used to tailor service offerings, develop patient support programs, and demonstrate the pharmacy's commitment to improving patient outcomes. Patient advocacy groups can also help pharmacies identify potential clinical trial participants and raise awareness about trials [14].

Furthermore, patient communities can serve as valuable partners in disseminating information about the pharmacy's services and raising awareness about rare diseases [15]. This can help to expand the pharmacy's reach and build trust within the rare disease community.

Why does engagement matter?

• Pharmacies that collaborate with patient groups see higher prescription retention rates due to stronger patient trust.

• Active involvement in patient communities increases brand visibility, leading to higher manufacturer preference in bids.

• Data-sharing agreements with advocacy groups can provide real-world evidence to manufacturers, strengthening pharmacy bids.

How to effectively engage with patient support groups, build trust within the community and gain visibility among manufacturers

In the competitive landscape of specialty pharmacy, securing successful bids requires a holistic approach that demonstrates a deep understanding of patient needs and a commitment to community engagement. A strong track record of collaboration needs to be visible to manufacturers. Here are some key strategies to enhance your bid success:

1. Cultivating strong relationships with patient support groups:

• Active engagement: Go beyond passive sponsorship. Attend patient advocacy events, participate in online forums, and offer educational workshops.

• Tailored support: Understand the specific needs of each patient group. Offer customized programs, resources, and services that address their unique challenges.

• Feedback loops: Establish open communication channels to solicit feedback and incorporate patient perspectives into your service delivery.

• Collaborative initiatives: Partner with patient groups on initiatives that raise awareness, improve access to care, and empower patients.

2. Building trust within the rare disease community:

• Transparency and empathy: Communicate openly about your services, pricing, and patient support programs. Demonstrate genuine empathy and understanding of the challenges faced by rare disease patients.

• Personalized care: Provide individualized care plans that address the unique needs of each patient. Offer dedicated patient navigators and pharmacists with specialized expertise.

• Reliable access: Ensure reliable access to medications, including those that are difficult to obtain. Offer flexible delivery options and assistance with insurance and financial aid.

• Educational resources: Provide comprehensive educational resources on rare diseases, treatment options, and patient support services.

Did you know, AllMyHealth has an audience of 15,000+ subscribers, including 450 patient support groups? Speak to our team to find out how you can engage more with your community today.

3. Gaining visibility among manufacturers:

• Demonstrate expertise: Showcase your expertise in managing complex therapies and providing specialized patient support.

• Data-driven outcomes: Track and report on key performance indicators, such as adherence rates, patient satisfaction, and clinical outcomes.

• Collaborative partnerships: Proactively seek opportunities to collaborate with manufacturers on clinical trials, patient assistance programs, and educational initiatives.

• Showcase technological capabilities: Highlight your ability to manage data, track inventory, and provide secure and efficient medication delivery.

• Proactive Communication: Maintain regular communication with manufacturer representatives, providing updates on your services and capabilities.

4. The value of targeted information and support:

• In the realm of rare diseases, access to accurate and timely information is paramount. Specialty pharmacies can significantly enhance their value proposition by providing curated content and resources.

• This includes access to specialized news, research updates and support networks. Platforms like AMH (AllMyHealth) play a vital role in this space, focusing on rare disease communities. AMH's model of providing curated content and resources for underserved rare disease communities, improving access to specialized news, research, and support, shows the power of focused data and community building. Integrating this type of resource or similar resources into your patient offerings, speaks volumes to your dedication to the patient.

• By partnering with or utilizing resources similar to AMH, specialty pharmacies can demonstrate their commitment to patient empowerment and improved access.

Examples of successful bids by rare disease pharmacies and recently won contracts

Accreditations and distinctions:

• Orsini Specialty Pharmacy: On January 6, 2025, Orsini Specialty Pharmacy partnered with PicnicHealth to enhance rare disease patient care and real-world study execution. ​[18]

Exclusive distribution and provider contracts:

• X4 Pharmaceuticals and taiba rare: On February 19, 2025, X4 Pharmaceuticals entered into an exclusive agreement with taiba rare for the distribution and commercialization of XOLREMDI® (mavorixafor) in select Middle East countries, targeting WHIM syndrome, a rare immunodeficiency. [19]

• PANTHERx® Rare & Ionis Pharmaceuticals (TRYNGOLZA™): On December 23, 2024, PANTHERx® Rare was selected by Ionis Pharmaceuticals, Inc. as the specialty pharmacy for TRYNGOLZA™ (olezarsen). TRYNGOLZA™ is the first and only FDA-approved treatment, as of December 19, 2024, designed to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare genetic disorder affecting fat metabolism [20]

These strategic partnerships and accreditations highlight how specialty pharmacies continue to lead in the rare disease space, ensuring access to life-changing therapies for patients with complex conditions.

Key takeaways

The specialty pharmacy landscape is increasingly competitive. Winning bids requires a clear strategy, strong partnerships, and innovative patient-centric approaches. Here’s what you need to focus on:

1. Winning more contracts: stand out in a competitive market

• Differentiate your pharmacy: Focus on niche rare disease treatments where large chains have less expertise.

• Highlight patient outcomes: Use real-world data to prove how your pharmacy improves adherence and patient well-being.

• Master prior authorizations: Streamline approval processes to reduce delays for patients and payers.

• Be proactive, not reactive: Approach manufacturers before contracts are released to build relationships early.

2. Building stronger relationships with manufacturers & payers:

• Showcase your value beyond dispensing: Provide adherence programs, financial support services, and direct patient engagement.

• Leverage outcomes-based agreements (OBAs): Demonstrate how your pharmacy can track and improve patient outcomes for high-cost therapies.

• Invest in data analytics: Payers and manufacturers prioritize pharmacies that can provide robust reporting on treatment success.

• Engage early in drug commercialization: Work with manufacturers during clinical trials and drug launches to position yourself as a preferred distributor.

3. Enhancing patient-centric services

• Partner with advocacy groups: Strong patient engagement increases manufacturer trust and bid success.

•  Offer personalized support programs: Pharmacies that provide disease-specific education and adherence coaching stand out.

• Expand financial assistance services: Manufacturers and payers favor pharmacies that reduce patient financial barriers.

4. Using technology & data for competitive advantage

• Implement AI for predictive adherence monitoring: Proactively intervene before patients stop treatment.

• Leverage blockchain for secure supply chain management: Prove compliance with DSCSA and improve manufacturer trust.

• Integrate with EHRs: Provide seamless data exchange with providers and payers.

5. Mastering pricing & reimbursement models

• Adopt flexible pricing strategies: Subscription models, OBAs, and risk-sharing agreements make high-cost therapies more attractive to payers.

• Demonstrate cost-efficiency in bids: Pharmacies that can show reduced overall healthcare costs have a competitive advantage.

• Highlight success with value-based care models: Show how your pharmacy aligns financial incentives with patient health improvements.

Next steps: Take action now

Winning contracts isn’t just about having the right credentials - it’s about proving your value, building trust and staying ahead of industry trends.

What will your pharmacy do next?

• Identify at least one area where you can improve your bid strategy.

• Set a 6-month action plan to strengthen relationships with payers or manufacturers.

• Invest in one new technology or patient support service that differentiates you in the next bidding cycle.

By focusing on these key areas, your specialty pharmacy can secure more contracts, improve patient outcomes, and position itself as a leader in the rare disease space.

Conclusion

Winning rare disease contracts isn’t just about meeting requirements - it’s about proving value, building trust, and staying ahead of industry shifts. What steps will your pharmacy take today to stand out in the competitive specialty pharmacy landscape? Whether it’s refining your patient engagement strategy, strengthening manufacturer relationships, or integrating new data-driven approaches, the time to act is now.

Speak to AllMyHealth to refine your patient engagement process today. Email info@allmyhealth.io for more information. 

References

1. Patient-driven initiatives for prioritizing drug discovery for rare diseases - PMC

https://pmc.ncbi.nlm.nih.gov/articles/PMC6607820/

2. Listening and Learning: Advancing Equity in Rare Disease Care - Sanofi U.S.

https://www.sanofi.us/en/sanofi-today/your-health/listening-and-learning-advancing-equity-in-rare-disease-care

3. Emerging roles and opportunities for rare disease patient advocacy groups - PMC 

https://pmc.ncbi.nlm.nih.gov/articles/PMC10184204/

4. Navigating Key Challenges in M&A Deals in the Rare Disease Sector - Morgan Lewis

https://www.morganlewis.com/blogs/asprescribed/2025/02/navigating-key-challenges-in-m-a-deals-in-the-rare-disease-sector

5. Navigating distribution challenges for rare disease drugs - Pharmaceutical Technology

https://www.pharmaceutical-technology.com/features/navigating-distribution-challenges-for-rare-disease-drugs/

6. The Rare Disease Market: Policy Changes and Coverage Trends - Avalere https://avalere.com/insights/rare-disease-market

7. Pharmacy's Role in Treating Rare Disease: Connecting Patients, Providers, Payers, and Pharma

https://www.pharmacytimes.com/view/pharmacy-s-role-in-treating-rare-disease-connecting-patients-providers-payers-and-pharma

8. Shaping the Future of Specialty Pharmacy: Gaining Insights from Industry Leaders on Rare Disease Therapy Commercialization - GRG Health

https://www.grgonline.com/post/shaping-the-future-of-specialty-pharmacy-gaining-insights-from-industry-leaders-on-rare-disease-the

9. Rare Disease Clinical Trials: Challenges, Strategies for Success - Allucent

https://www.allucent.com/resources/blog/rare-disease-clinical-trials-challenges-strategies

10. 5 Ways Pharma Can Support Patients with Rare Diseases - AllazoHealth,

https://allazohealth.com/resources/5-ways-pharma-can-support-patients-with-rare-diseases/

11. Advocacy, Empathy, and Support for Rare Disease Patients in Clinical Trials - mdgroup

https://mdgroup.com/blog/advocacy-empathy-and-support-for-rare-disease-patients-in-clinical-trials/

12. Establishing Patient Registries for Rare Diseases: Rationale and Challenges - PMC

https://pmc.ncbi.nlm.nih.gov/articles/PMC7286934/

13. Contributor: The Critical Role of Patient Engagement in Patient-Centered Outcomes Research and Health Technology Assessment of Rare Disease Treatments - American Journal of Managed Care

https://www.ajmc.com/view/contributor-the-critical-role-of-patient-engagement-in-patient-centered-outcomes-research-and-health-technology-assessment-of-rare-disease-treatments

14. Realizing the Full Value of Patient Centricity in Rare Disease: 5 Ways to Maximize Your Engagement Efforts | Premier Research

https://premier-research.com/perspectives/realizing-the-full-value-of-patient-centricity-in-rare-disease/

15. Patient involvement in rare diseases research: a scoping review of the literature and mixed method evaluation of Norwegian researchers' experiences and perceptions

https://pmc.ncbi.nlm.nih.gov/articles/PMC9158134/

16. Rare Disease Pharmacies: The Quarterbacks for Orphan Drug Regimens

https://www.specialtypharmacycontinuum.com/Operations-and-Management/Article/02-25/Rare-Disease-Pharmacy-Management/76223

17. Successful launches in rare diseases - McKinsey

https://www.mckinsey.com/~/media/McKinsey/Industries/Pharmaceuticals%20and%20Medical%20Products/Our%20Insights/How%20to%20successfully%20launch%20a%20rare%20disease%20drug%20in%20a%20patient%20centric%20world/Successful-launches-in-rare-diseases-vFinal.pdf

18. Orsini and PicnicHealth Partner to Improve Rare Disease Patient Care and Real-World Study Execution

https://picnichealth.com/blogs/orsini-and-picnichealth-partner-to-improve-rare-disease-patient-care-and-real-world-study-execution

19. X4 Pharmaceuticals and taiba rare Announce Exclusive Agreement for the Distribution and Commercialization of XOLREMDI® (mavorixafor) in WHIM Syndrome in Select Middle East Countries

https://investors.x4pharma.com/news-releases/news-release-details/x4-pharmaceuticals-and-taiba-rare-announce-exclusive-agreement

20. PANTHERx® Rare Selected by Ionis as Specialty Pharmacy for TRYNGOLZA™ (olezarsen)

https://www.biospace.com/press-releases/pantherx-rare-selected-by-ionis-as-specialty-pharmacy-for-tryngolza-olezarsen