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2025-07-28

PSP Services: The Most Effective Investment by Pharmaceutical Companies in Rare Disease Care

PSPs encompass a wide range of services, including insurance navigation, financial assistance, patient education, nursing support, and medication adherence guidance. As rare disease therapies become more prevalent and AI-driven drug development advances, the importance of PSPs continues to grow—especially given that over half of the new drugs approved by the U.S. FDA are now for rare diseases.


The investment value of PSPs in rare disease management can be measured through key performance indicators such as adherence, patient satisfaction, treatment accessibility, clinical outcomes, and cost-effectiveness. In recent years, leading pharmaceutical companies in the rare disease space—such as Takeda, Sanofi, Novartis, and Pfizer—have increasingly refined and optimized their investments in PSPs to maximize impact.

Impact of PSPs on Key Metrics and Return on Investment

In 2023, the pharmaceutical mergers and acquisitions (M&A) market began to recover from the impact of the pandemic, with both deal value and volume returning to pre-pandemic levels. Notably, while the number of deals saw a slight year-over-year decline of 8%, the total deal value surged by 37%, surpassing USD 200 billion. This increase can be attributed to the prolonged delays in negotiating major transactions during the pandemic. With restrictions now fully lifted, the industry has shifted its focus toward targets with higher potential value and lower risk, driving up the overall deal value.

The trend of "the big getting bigger" is becoming increasingly evident in the pharmaceutical industry. In terms of scale, the largest deals include Pfizer’s USD 43 billion acquisition of Seagen, Amgen’s USD 27.8 billion purchase of Horizon Therapeutics, Bristol Myers Squibb’s USD 14 billion acquisition of Karuna Therapeutics, and AbbVie’s USD 10.1 billion deal for Immunogen. Pfizer’s acquisition exemplifies several defining features of today’s M\&A landscape: pharmaceutical giants with strong cash reserves pursuing high-growth-potential targets.

Which Therapeutic Areas Are Attracting Investment?

From the perspective of pharmaceutical companies, investing in PSPs for rare diseases can yield several tangible business benefits：

  • Increase patient retention and loyalty： By helping patients adhere to treatment and feel supported, PSPs can foster patient loyalty to both the product and the company. Patients who receive emotional, financial, and medical support are more likely to continue their treatment and trust the brand, thereby building long-term loyalty. For example, AbbVie offers an extensive support program called "Humira Complete" for Humira patients (although not a rare disease drug, it treats complex immune conditions). Studies on this program have demonstrated concrete benefits: patients enrolled showed approximately a 29% increase in adherence at 12 months and maintained better adherence over 2 to 3 years. The risk of discontinuation was 30% lower compared to non-participants, and hospitalizations were significantly reduced. AbbVie has leveraged these results to demonstrate that its patient services improve real-world outcomes and patient retention. This case is frequently cited as evidence that PSPs can simultaneously drive both health and business results, extending the duration of treatment per patient.
  • Expand market penetration and accessibility： PSPs effectively increase market penetration by removing barriers to treatment initiation. They provide insurance navigation, financial assistance, and education, which boost the proportion of new patients starting therapy. Metrics such as shortened time to first dose and higher treatment persistence demonstrate the value of PSPs to prescribing physicians. Moreover, doctors are more inclined to prescribe medications that come with patient support programs, knowing their patients will receive comprehensive care beyond the clinic. For instance, Sanofi’s programs for certain rare diseases include free counselor services for patients and families, which is especially important for caregivers managing pediatric rare conditions. This psychosocial support enhances patient satisfaction and indirectly improves adherence, as supported families are more likely to follow complex treatment regimens. It also differentiates Sanofi’s products by emphasizing the company’s commitment to overall patient well-being—not just drug supply.
  • Data Generation and Real-World Evidence (RWE)： PSPs generate a wealth of real-world data (RWD) on how patients use medications, adherence patterns, achieved outcomes, and reasons for discontinuation. This real-world data is highly valuable to pharmaceutical companies. By analyzing PSP data, companies gain deeper insights into patient needs and treatment effectiveness outside of clinical trial settings. This helps provide stronger evidence to regulators and payers—for example, long-term outcome data collected through PSPs can support regulatory approvals for new indications or fulfill post-marketing study requirements. Currently, several companies have integrated real-world evidence (RWE) collection into their PSPs—for instance, PSP providers pair patient support with electronic health record data to continuously gather outcomes with patient consent. For pharmaceutical companies, this rich RWE resource informs ongoing development (drug lifecycle management) and strengthens the evidence base for their therapies in real-world practice.
  • Enhance patient satisfaction and brand reputation： A well-operated PSP can significantly enhance the patient treatment experience, thereby boosting the company’s reputation. High patient satisfaction—typically measured through surveys or Net Promoter Scores (NPS)—indicates that the pharmaceutical company delivers value beyond the medication itself. Such positive experiences increase the likelihood of patients continuing treatment and advocating for the therapy within their communities. In the rare disease field, word-of-mouth and community advocacy hold tremendous influence; pharmaceutical companies investing in patient-centered services can position their brands and products as truly patient-friendly. This goodwill can translate into stronger relationships with patient advocacy groups and regulatory agencies, which are increasingly encouraging patient-centric drug development.
  • Better alignment with value-based care models：As healthcare shifts toward value-based care, pharmaceutical companies must demonstrate that their therapies improve outcomes in a cost-effective manner. PSPs support this by enhancing real-world drug-related outcomes, such as adherence and complication avoidance. Improved adherence means payers can see greater value in expensive rare disease treatments. In fact, increasing treatment adherence through PSPs "strengthens the product’s value proposition" and reduces payer uncertainty about outcomes.Some innovative arrangements directly link PSPs to value-based contracts. For example, a global model exists where manufacturers provide financial rebates if patients receiving costly therapies fail to achieve expected results (outcome-based rebates). These agreements are only feasible with robust patient support and monitoring to track outcomes.By investing in PSPs, pharmaceutical companies align their services with the goals of value-based care. In one case, Novartis obtained approval from the U.S. Office of Inspector General (OIG) to offer travel and lodging support for low-income patients receiving its CAR-T gene therapy (Kymriah), improving accessibility while remaining compliant. Such support not only aids patients but also demonstrates to policymakers and payers the company’s commitment to outcomes—ensuring patients can safely access treatment and receive long-term follow-up.
     

Strategic Advantages Brought by PSPs

Beyond immediate metrics and return on investment, PSPs also provide pharmaceutical companies with broader strategic advantages in the rare disease field：

  • Product differentiation： In competitive therapeutic areas—including rare diseases with multiple treatment options—a strong PSP can make a product stand out. A well-designed PSP “demonstrates value beyond standard efficacy and safety measures.” In fact, payers consider PSP services an important differentiator when evaluating similar drugs. By truly addressing patient needs—such as education, financial assistance, and care coordination—PSPs transform a therapy from merely a medication into a comprehensive solution. This can positively influence physicians’ perceptions of their commitment to patient care, making them more inclined to choose the company’s product. For example, Takeda and Sanofi have distinguished their enzyme replacement therapies in rare genetic disease treatment by offering extensive patient support—including at-home injections and disease education—making their products more attractive to both providers and patients. Research and industry experts highlight patient-centric PSPs as a “win-win” scenario, improving outcomes while adding value to pharmaceutical products.
  • Accelerate market penetration： The launch phase of rare disease drugs can be improved by using hub services to streamline the onboarding process for new patients. PSPs offer faster patient enrollment verification, prior authorization support, and patient education, enabling quicker treatment initiation post-launch. This allows new therapies to establish a foothold in the market more rapidly. Furthermore, as mentioned earlier, when there is supporting infrastructure in place—where physicians know patients will receive assistance with complex self-injection, side effect management, and more—they feel more confident prescribing new rare disease medications.In Asia, where awareness of rare diseases may be lower and healthcare systems more fragmented, PSPs similarly help accelerate market penetration by bridging these gaps. Companies like Takeda have leveraged PSPs in Asia—for example, in Southeast Asia—by collaborating with NGOs, patient organizations, and professional consultants to increase diagnosis rates and ensure eligible patients receive treatment, thereby expanding the treatable market.
  • Stronger stakeholder relationships： Patient Support Programs can strengthen pharmaceutical companies’ relationships with key stakeholders—regulators, payers, and providers. Regulators recognize the value of programs that ensure safe use and monitoring; some programs involve risk evaluation and mitigation strategies (REMS), which often include patient education components that effectively serve as a form of PSP. Improved adherence data reduces payer uncertainty and reinforces the value proposition of the medication. When manufacturers can demonstrate through PSP data that patients receiving their therapy adhere longer and achieve better outcomes, it enhances their bargaining power in pricing and reimbursement negotiations. Providers (healthcare professionals) also benefit: PSPs handle supportive tasks such as insurance paperwork and patient training, alleviating burdens on medical staff and encouraging providers to become collaborative partners.

Regional Barriers and Efforts in PSP Development

United States： In the United States, PSPs—often referred to as “hub” services for specialty drugs—are an established component of rare disease product strategies. They aim to navigate the complex reimbursement landscape and ensure patients do not interrupt treatment due to financial or logistical barriers. However, utilization remains suboptimal—studies show low patient awareness and engagement, with only about 3–8% of eligible patients participating, primarily due to lack of awareness. Pharmaceutical companies are working to bridge this gap by promoting PSPs more actively through physicians, pharmacists, and digital channels.Compliance and regulatory scrutiny in the U.S. are stringent: companies must carefully design PSPs to avoid being considered inducements (for example, certain copay assistance programs are restricted under federal plans). Despite these challenges, patient support is increasingly recognized as an indispensable part of rare disease therapy launches. Companies like Pfizer have created dedicated rare disease support platforms—such as Pfizer’s “Together for Rare” and the VyndaLink program for tafamidis patients—that connect patients with needed services. Well-operated PSPs can improve treatment persistence.AbbVie’s Humira® case illustrates this well: by providing care support, education, and follow-up, the company achieved measurable adherence improvements over 1 to 3 years among PSP participants and significantly reduced hospitalizations compared to non-participants.

Asia： In Asia, the deployment of PSPs is accelerating, though maturity varies by country. In Japan and South Korea, many global pharmaceutical companies expand their patient support services (such as education and call centers) at product launch, similar to Western markets. In emerging Asian markets, PSPs remain a relatively new concept but are gaining attention, typically focusing on accessibility and affordability.For example, India now offers PSPs for oncology, rare genetic diseases, and other specialty areas. Given that about 70% of medications in India are paid out-of-pocket, PSPs mainly provide financial counseling, free medications or copay assistance, and patient education.Gaps remain: many programs are urban-centric and fragmented, with limited coverage in rural areas and language barriers. Awareness is also an issue in Asian markets; unlike in the U.S., where PSP information is publicly and officially accessible, PSP details in Asian countries are often difficult to obtain.The most open environment is in China, where patient support programs are being encouraged to improve rare disease drug accessibility—for example, through charitable foundations or tiered pricing schemes. Southeast Asian countries are discussing frameworks for compassionate use and PSP-like models.

In Taiwan, PatientsForce has localized PSP services for major international pharmaceutical companies, covering insurance navigation, financial assistance, patient education, nursing support, and medication adherence guidance. They also connect with home healthcare and home nursing systems. Based on investment return outcomes, treatment adherence among rare disease patients served by these programs has significantly improved, while also helping healthcare institutions alleviate staffing burdens—thereby enhancing patient satisfaction with medical care.

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