Unlocking the Full Potential of Rare Disease Drug Launches with Digital PSPs

The Rare Disease Launch Challenge

Rare disease drug launches are fundamentally different from traditional launches due to the unique challenges they present. Small, dispersed patient populations make identification and engagement difficult. Many rare diseases are underdiagnosed, leading to delays in treatment initiation. Additionally, relying solely on healthcare provider (HCP)-driven identification slows uptake and creates friction in patient access. With fewer available treatments, ensuring long-term patient adherence is critical but challenging.

‍
One of the biggest barriers to rare disease drug adoption is the clinic-only acquisition model, which limits patient outreach. Traditional methods depend on patients seeking care proactively, often after experiencing significant disease progression. This results in delayed diagnoses, treatment gaps, and low adherence rates.

• Small, dispersed patient populations: Patients are often spread across various regions, making engagement difficult.
• Limited diagnosis and awareness: Many rare diseases are underdiagnosed, delaying treatment initiation.
• Clinic-only acquisition barriers: Dependence on HCP-driven identification slows uptake.
• Low adherence rates: Ensuring long-term adherence is difficult due to limited treatment options.

How Digital PSPs Can Change the Game

Digital Patient Support Programs (PSPs) are transforming rare disease drug launches by providing scalable, patient-centered solutions. They significantly reduce time-to-uptake by enabling direct patient engagement, bypassing traditional bottlenecks. Additionally, they enhance market access by generating real-world data (RWD) that supports reimbursement discussions. Furthermore, they drive adherence through personalised digital interventions that empower patients and improve long-term outcomes.

Digital PSPs create a proactive patient pathway, where education, symptom tracking, and medication reminders help ensure treatment continuity. Patients are no longer passive recipients of care — they become active participants in their health journey.

• Reducing time-to-uptake: Direct patient engagement bypasses traditional bottlenecks.
• Enhancing market access: Real-world patient data supports reimbursement discussions.
• Driving adherence: Personalised digital support helps patients maintain treatment adherence.

‍

PHuman: A Proven Solution in Rare Disease PSPs

Avegen’s PHuman - a digital PSP for pulmonary arterial hypertension (PAH) - has demonstrated significant success. It supports PAH patients across Europe by providing education, tracking, and engagement tools. The program has:

1. Already onboarded 5.64% of diagnosed PAH patients in active regions.
2. Over 60% of users track symptoms and vitals, and 45% share reports with HCPs. 
3. Real-world insights from over 22,000 patient-recorded symptoms and vitals are helping improve patient outcomes.

PHuman also serves as a bridge between pharma, patients, and HCPs, providing data-backed insights that facilitate treatment decision-making. Patients gain greater control over their condition, while pharma gains access to meaningful, real-world evidence supporting drug efficacy and adherence.

‍

Key Pharma Learnings from PHuman’s Success

• Digital PSPs enhance patient self-management and reduce treatment drop-off rates.
• Real-time patient data strengthens clinical decision-making and reimbursement arguments.
• Scalability: A digital-first approach supports global market expansion with minimal incremental cost.

‍

Why Digital PSPs Should Be a Launch Priority

A well-executed digital PSP accelerates market success by enabling faster patient acquisition, leading to stronger early market traction. Digital solutions offer scalable, cost-efficient support for niche populations, ensuring that rare disease patients remain engaged beyond initial treatment initiation. Additionally, real-world data (RWD) collected from digital PSPs provides a competitive advantage in market access and reimbursement discussions.

• Faster patient acquisition → Stronger early market traction
• Scalable, cost-efficient support for niche populations‍
• Real-world data enhances market access and reimbursement

What Next? How to Build a High-Impact Digital PSP for Rare Disease

For pharma teams planning a digital PSP, defining objectives is a crucial first step. Identifying key outcomes such as adherence, engagement, and RWD generation ensures a clear focus. Leveraging proven digital platforms helps maintain compliance and scalability. Ensuring seamless integration with HCP workflows optimizes the impact of PSPs.

The most effective digital PSPs follow a structured implementation roadmap, ensuring a seamless transition from strategy to execution. Avegen’s proven approach ensures rapid deployment while maintaining compliance and high patient engagement.

• Define objectives: Identify target outcomes (adherence, engagement, RWD generation).
• Leverage proven platforms: Use validated digital solutions to ensure compliance and scalability.
• Ensure seamless HCP integration: Align PSP functionality with clinical workflows.

‍

How Avegen Supports Pharma

Avegen helps pharma companies with rapid deployment and localisation for different markets. The PHuman solution ensures GDPR-compliant patient engagement and data collection while continuously optimising based on real-world usage insights.

Conclusion

Digital PSPs represent a transformational opportunity for rare disease drug launches. By accelerating patient engagement, improving adherence, and generating valuable real-world insights, these solutions help pharma achieve market success faster and more efficiently.

Avegen’s PHuman has already proven its impact in PAH, and the same principles can be applied across multiple rare disease indications. As pharma companies look to the future, investing in digital-first patient support will be the key to unlocking rare disease success.

Is a tailored digital PSP for your rare disease drug launch a part of your strategy? Let’s talk.