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Posted by Heather Cumens on February 26, 2024
rare disease marketing strategy

Building a go-to-market strategy for a rare disease demands meticulous planning and a deep understanding of the distinctive marketing challenges associated with such conditions. From limited patient populations to specialized physicians and complex diagnostic and treatment pathways, the intricacies of rare diseases necessitate a tailored approach to marketing. In this article, we delve into the essential elements and strategic pillars for developing a successful rare disease go-to-market strategy. From understanding the rare disease market and identifying target patients to strategic methods for positioning in rare diseases and orphan disease pricing strategies, we explore the multifaceted aspects of marketing rare disease treatments. Additionally, we shed light on the key performance indicators (KPIs) that are vital for evaluating the effectiveness of go-to-market strategies for rare disease treatments. By delving into these critical areas, we aim to provide valuable insights for stakeholders navigating the intricate landscape of rare disease marketing.

How to Build a Rare Disease Go-to-Market Strategy

The unique challenges associated with marketing rare disease treatments require careful planning and consideration to fully account for limited patient populations, specialized healthcare providers, and complex diagnostic and treatment pathways,

To build a rare disease go-to-market strategy, several key considerations and strategic pillars should be taken into account. Here are the essential elements for developing a successful rare disease go-to-market strategy:

Understanding the Rare Disease Market – Identify your Target Patient and Disease Community

Effective rare disease marketing requires an ethical, human-centric approach, with a special focus on patient and caregiver support and treatment adherence1.  Often a go-to-market strategy for rare diseases benefits from segmenting the audience based on factors such as disease severity, treatment preferences, geographic location, and level of disease awareness.

Market Research is vital to help understand the specific rare disease landscape, including prevalence, patient demographics, disease community, diagnosis, treatment options, and unmet needs. 

While the patient-centric approach helps marketers to better understand the end recipient of their rare disease treatment, several other stakeholders are vital to embrace and understand when marketing rare disease treatments.  Each rare disease market will have specific stakeholders that need to be identified.  These may include patient advocacy groups, healthcare professionals, researchers, and regulatory bodies, as well as the primary target audience that will include patients, caregivers, HCPs, and other stakeholders involved in the care continuum.  

Patient advocacy groups are a great resource for raising disease awareness, providing educational resources, and supporting patient empowerment.  Leverage their networks to reach and engage with patients and caregivers and provide resources.  Providing targeted education and training programs for healthcare professionals who specialize in the rare disease area is a great recruitment tool as well.

Academic institutions and research organizations provide opportunities for partnerships to advance disease understanding and treatment options, clinical trial collaborations, and advocacy initiatives.

Market Assessment: 

Before rolling up the sleeves to execute a go-to-market strategy, it’s essential to find solid numbers, identify patient volumes, and assess the unmet need for the specific rare disease3. Companies must understand the regulatory pathways for rare disease therapies and ensure compliance with relevant regulations and guidelines.

In addition, a reimbursement strategy to address payer challenges and facilitate patient access to treatment must be developed.  Relevant reimbursement pathways, including government healthcare programs, private insurance, and specialty pharmacy networks must be identified and targeted with real-world evidence that demonstrates the clinical and economic value of the new drug.  Health economic analyses, cost-effectiveness studies, and quality-of-life assessments can all help support reimbursement decisions.

Companies should initiate dialogue with payers and reimbursement authorities during development and routinely present compelling data on the disease burden, treatment efficacy, safety, and cost-effectiveness.

Strategic Methods for Positioning in Rare Diseases

It’s crucial to assess if the rare disease drug can be differentiated, generate evidence to back its claims, and have a clear value proposition for payers in reimbursed markets3.  Rare disease treatments should focus on addressing specific unmet needs within the patient population. This could include targeting symptoms that are not effectively managed by current therapies, providing disease-modifying effects, or offering improved safety profiles.

The clinical efficacy of the treatment should be demonstrated through robust clinical trial data. This includes showing improvements in key endpoints such as symptom reduction, disease progression, quality of life, and patient survival. Demonstrate the long-term benefits of the treatment, including sustained efficacy, disease stabilization, or delayed progression. This is particularly important for chronic rare diseases where maintaining stable health over time is a priority.

It is important to emphasize the safety profile of the treatment, particularly in comparison to existing therapies or standards of care. Rare disease patients often have few treatment options, so safety is a critical consideration for both patients and HCPs.

If the treatment offers a more convenient or less invasive mode of administration (e.g., oral vs. injectable), highlight this as a key advantage. Improving the ease of product use can enhance patient adherence and quality of life.

If the treatment is tailored to specific genetic mutations or patient subgroups, emphasize the personalized approach and potential for better outcomes compared to one-size-fits-all products.

Pricing Strategy in Rare Disease Treatments

Setting a pricing strategy for rare disease treatments requires careful consideration of various factors, including development costs, market dynamics, patient affordability, and value delivered by the treatment. 

Development Costs

The pricing of rare disease drugs is often based on the costs of research and development, the projected number of patients who will benefit from the drug, and a profit margin1.  Calculate the total development costs of bringing the treatment to market, including research and development expenses, clinical trial costs, regulatory fees, and manufacturing expenses. The cost of capital and desired return on investment for stakeholders must be calculated into the equation as well.

Market Dynamics

The pricing strategy takes into account the rarity of the patient population, the level of clinical benefit or improvement in patient outcomes, and the existence of benchmarks or references for prices of existing therapies2. Determining the unique value proposition of the treatment compared to existing therapies or standards of care is vital to understanding the market dynamics. Clinical efficacy, safety profile, convenience of administration, and impact on patient outcomes can all modify the pricing and marketing of the treatment.

If the treatment has received orphan drug designation or market exclusivity, consider the potential for pricing flexibility and extended market exclusivity periods. Orphan drugs often have higher prices compared to non-orphan drug counterparts, and payer spending on these treatments can vary significantly from year to year, given the small patient population affected. Take into account any regulatory incentives or financial incentives associated with orphan drug status.

Consider Patient Affordability

Recognize the financial burden of rare diseases on patients and their families, including out-of-pocket expenses, insurance coverage limitations, and access barriers.  Balance the need for affordable pricing with the requirement for sustainable revenue generation to support ongoing research and development efforts.

Understand the reimbursement landscape and payer policies for rare disease treatments.  Pricing and reimbursement for rare disease drugs are often negotiated individually with pharmaceutical companies in each country, where national health policies vary. Some countries have put in place government intervention to restrict public reimbursement and promote the cost-effective use of orphan drugs4.

Monitor and Adjust Pricing Strategy

Continuously monitor market dynamics, pricing trends, and patient access issues. Be prepared to adjust pricing strategy as needed based on changes in the competitive landscape, regulatory environment, or reimbursement policies.

By carefully considering these factors and aligning pricing strategy with the value delivered by the treatment, rare disease therapies can maximize patient access while ensuring financial sustainability for continued innovation and development.

Promotional Strategy for Building Brand Awareness for Optimum Patient Acquisition

The promotional strategy for building brand awareness and optimizing patient acquisition in a go-to-market strategy for rare diseases involves several key considerations in building a targeted and multifaceted marketing strategy.

Effective rare disease marketing requires a human-centric approach, with a special focus on patient and caregiver support and treatment adherence. It is essential to personalize the message and connect with customers in a way that is as unique as the disease they are facing.

Develop Educational Content

Create comprehensive educational materials about the rare disease, its symptoms, diagnosis, and treatment options.  Use educational events, webinars, and workshops to raise awareness.

  • Distribute content through various channels such as websites, social media, patient support groups, and healthcare professional networks.
  • Invite key opinion leaders, healthcare professionals, and patient advocates to speak at events and share their expertise.

Engage with Patient Advocacy Groups:

Collaborate with patient advocacy organizations specializing in the rare disease area.  Depending on how well-established a therapeutic area is may influence the type of approach. For well-established disease states with a broad patient base and established patient advocacy groups, less educational materials around the disease state might be warranted.  Marketing could focus on comparison and unique value propositions that differentiate from established competitors in the space.  

Less established patient communities for rare diseases may need more educational materials.  Patient advocacy groups may need more support in pushing their agendas to the forefront of public knowledge. In cases where no established or proven therapies exist, marketing may need to pivot towards emphasizing symptom improvement, patient success stories, and engagement with healthcare professionals.

For extremely rare diseases, both the patient base and support community may be very small.  Consequently, these therapeutic areas may require additional resources, educational support and materials for patients, and sustained research into new treatments. Marketing efforts may need to prioritize the dissemination of educational resources and the establishment of patient advocacy and support groups.

  • Establish patient support programs to provide assistance and resources to individuals affected by the rare disease.
  • Support awareness campaigns, educational events, and patient empowerment initiatives organized by advocacy groups.

Utilize Digital Marketing:

Leverage digital marketing channels to reach a targeted audience of patients, caregivers, and healthcare professionals. Share patient testimonials and success stories to humanize the impact of the rare disease and highlight the benefits of treatment.

  • Develop a search engine optimization (SEO) strategy to improve visibility in online searches related to rare diseases.
  • Run targeted online marketing campaigns on platforms such as Google Ads, social media platforms, and healthcare-related websites.  Engage in market research to determine which social media platforms patients use most.   

Target Healthcare Professionals:

  • Develop educational materials and resources for healthcare professionals who may encounter patients with the rare disease.
  • Provide continuing medical education (CME) opportunities, clinical updates, and resources to support healthcare provider knowledge and awareness.
  • Engage with specialty societies, medical associations, and professional networks to reach healthcare professionals.

Setting metrics and monitoring performance for Rare Disease Marketing Strategies

Key performance indicators (KPIs) are essential metrics that help evaluate the effectiveness of a go-to-market strategy for rare disease treatments. Here are some key performance indicators for data-driven marketing:

Patient Acquisition Rate: Measure the rate at which new patients are acquired over time. This metric indicates the effectiveness of promotional efforts and patient outreach initiatives.

Disease Awareness: Track changes in disease awareness levels among target audiences, including patients, caregivers, and healthcare professionals. This can be measured through surveys, website traffic, and social media engagement.

Healthcare Provider Adoption: Monitor the adoption rate of the treatment among healthcare providers, including physicians, specialists, and other relevant professionals. This metric reflects the acceptance and uptake of the treatment within the medical community.

Patient Engagement: Measure patient engagement with educational materials, support programs, and online communities. This can include metrics such as website visits, email open rates, participation in webinars, and attendance at patient events.

Treatment Adherence: Assess patient adherence to the treatment regimen over time. This can be tracked through medication refill rates, appointment attendance, and patient-reported outcomes.

Reimbursement Success: Monitor the success rate of reimbursement submissions and approvals for the treatment. This metric indicates the accessibility of the treatment to patients and the effectiveness of reimbursement strategies.

Market Share: Evaluate the market share captured by the treatment compared to competitors or alternative treatment options. This metric provides insights into the competitive landscape and the effectiveness of market penetration efforts.

Cost per Patient Acquired: Calculate the cost incurred for acquiring each new patient. This metric helps assess the efficiency of promotional and marketing initiatives relative to patient acquisition outcomes.

Long-Term Outcomes: Monitor long-term outcomes such as disease progression, survival rates, and quality of life improvements among treated patients. This provides insights into the real-world impact of the treatment beyond short-term metrics.

By tracking these key performance indicators, stakeholders can assess the success of their go-to-market strategy for rare disease treatments and make data-driven decisions to optimize future efforts.

The Challenges of an Effective Marketing Strategy for Rare Disease Drugs

Developing an effective marketing strategy for rare disease drugs presents several challenges rooted in the unique nature of these conditions. With small patient populations and limited awareness among patients and healthcare providers, reaching and engaging with the target audience poses a significant hurdle. Furthermore, high development costs and regulatory complexities add financial and logistical barriers to market entry. Access to care and reimbursement hurdles further complicate efforts to ensure patient access to treatment. Collaboration with patient advocacy organizations becomes crucial for raising awareness and providing support services. Additionally, ethical considerations mandate a sensitive approach that prioritizes patient well-being and dignity. Overcoming these challenges necessitates a comprehensive approach that involves collaboration among stakeholders to address awareness, access, and regulatory issues, ultimately improving outcomes for patients with rare diseases.

Understanding and Influencing HCPs – A Guide for Pharma, Medical Device, and Diagnostics Marketers

Physicians play a crucial role in the decision-making process when it comes to the use of new treatments, medical devices, and pharmaceuticals. They are responsible for selecting the most appropriate treatments for their patients, based on their clinical expertise and judgment, so it is essential for companies launching new and innovative therapies to understand how HCPs make decisions, where they go for information, and how to influence their behavior in a positive way.