Patient Advocacy Groups (PAGs)

1 in 2 rare diseases do not have a dedicated foundation or research support group. In the US, that number translates to close to 15 million patients navigating a complex journey without a dedicated team supporting them and ultimately seeking out a life-changing treatment. As the rare disease market continues to grow and evolve, so will the focus on patient engagement and advocacy, led in part by patient advocate leaders and community patient advocacy groups (PAGs).

PAGs engage with three primary rare disease stakeholders

Caption: Patient Advocacy Groups work with stakeholders across the rare disease patient journey and can be a valuable partner to biotech companies.

Patient advocacy groups (PAGs) are at the crux of a highly complex healthcare landscape. They play a central role in connecting several stakeholders and work as liaisons between patients, physicians, biotech companies, and community leaders. Companies frequently team up with PAGs to engage patient populations and offer support – such as treatment access and care – to both patients and caregivers. Patient Advocacy Groups also work with community leaders and far-reaching KOL networks to raise disease awareness and influence legislation and / or local policy. Other valuable activities, such as patient identification, clinical trial recruitment, and R&D collaboration, also support the indispensable role of PAGs in the rare disease market.

PAGs come in different shapes and sizes

Caption: Rare disease patient advocacy groups come in all shapes and sizes, supporting patients of many rare diseases at various levels – national or global.

The patient-focused nature of the rare disease space makes working with patient advocacy groups a necessity, not an option. While many companies may know the value that comes from PAG collaboration, they may not know how to go about having these conversations and develop a successful strategy. Companies should first thoroughly understand the various roles PAGs play in crafting an effective strategy and engage with these communities. Companies can take the knowledge they gain from these conversations to raise awareness, improve quality of life, and positively transform the treatment landscape for rare diseases. PAGs can support rare disease companies…

• …as Patient Advocates

It’s in the name: Patient Advocacy Groups, or PAGs, are there first and foremost to be advocates for patients. PAGs are very close to the patient journey and have a unique perspective on it, including unmet needs and ongoing challenges. These organizations support patients and caregivers in seeking out treatment centers, navigating complicated access pathways, and obtaining financial assistance. PAGs are direct advocates, often lobbying in the public domain and influencing regulators to improve patient access. Biotech companies should invest in long term collaborations with PAGs to create an open dialogue about how to successfully engage with patients, such as marketing tactics or educational tools. These conversations may unearth existing challenges or unmet needs for patients that companies can capitalize on and use to differentiate themselves. Insight into patient unmet needs also provides perspective on what types of services and support patients require, and new product or support offerings can be directly informed by these conversations and custom-designed to meet patient needs. Feedback on existing support services and disease challenges can help companies also refine their customer-facing strategies. 

• …as Educational Partners

With many rare diseases lacking educational resources, PAGs are crucial to minimize gaps in disease awareness. Besides providing patient and care education, they also work closely with COEs and KOLs to disseminate information about ongoing trials and new treatments, while encouraging patients to explore their options. The ongoing campaigns and awareness efforts often culminate in policy appeals and can even shift the treatment paradigm in the long term. Companies should consider this an opportunity to educate not only patients, but physicians as well, who are navigating a complicated diagnosis landscape with many unknowns. Diagnosis remains a large gap and could bring a big payoff to companies if they are able to develop new tools or tests to increase the diagnosis rate. Companies should strategically align themselves with PAGs to demonstrate a commitment to educating and supporting the patient population – a commitment that will reap impressive benefits later down the road.

• …as Research Partners

PAG proximity to the patient journey makes them a valuable resource in not only selecting treatments, but in developing them. Like KOLs, PAGs support clinical trials by identifying patient populations and referring patients for recruitment. Furthermore, as the voice of the patient, PAGs are pivotal in pushing novel or first-in-class therapies through the pipeline. Their firsthand experiences with patients can inform clinical trial design, from determining best-fit patient profiles to establishing trial endpoints. As mentioned earlier in this series, an early-stage collaboration can set companies up for success as they think through treatment protocols and, eventually, product commercialization. Questions around clinical trial endpoints, treatment administration, and existing treatment protocols can be very valuable when preparing for drug development and design. Working directly with patients and physicians gives companies a first-hand understanding of their customer profile and can better inform the product and its strategy.

What questions should rare disease players be asking PAGs?

Caption: How should companies guide conversations with PAGS? Companies can look for gaps in the current landscape and identify opportunities to add value to stakeholders across the care continuum.

Patient advocacy groups will always be front and center in the rare disease space, focusing on their patients and advocating for new and effective treatments. Engaging in conversations around the treatment landscape, disease challenges, and unmet needs can present companies with an opportunity to establish a trusted presence in the market. Companies should be excited to partner with these groups and do so early; loyalty within the rare disease landscape benefits all stakeholders – not just the patients.

AN ORPHAN DRUGS JOURNEY: COMMERCIALIZING RARE DISEASE DRUGS

In this series, Kaiser will explore how rare disease drug companies can develop commercial growth strategies to succeed against the challenges of a high cost and highly specialized market.

2018 saw 91 orphan drug approvals by the FDA – nearly 3 times greater than in 2013 and the highest number of approvals yet – along with over 400 designations for the candidates in development. 2019 is expected to bring even more orphan drug approvals as more companies begin to realize R&D investments in rare diseases. Driven by high pricing potential, significant unmet needs, and favorable regulatory policy, orphan drug sales are expected to balloon to reach $262B in the next 5 years. The market continues to grow more competitive and new technologies are emerging, such as Spark Therapeutics’ Luxturna, which marks the first FDA approval in a new class of one-time gene therapy treatments targeting inherited diseases. In this environment, companies will need to shift their focus to how they can play strategically and optimize the value of their orphan therapies.

Caption: Orphan drug approvals are expected to reach a similar level as 2018, with new tissue agnostic cancer therapies poised for approval and increasing market opportunities for emerging gene therapies, especially those enabled by advances in CRIPSR gene-editing. Source: FDA Orphan Drugs Data

Caption: Orphan drug sales are expected to grow at an ~11 % CAGR over the next five (5) years and continue to gain share of the overall drug market as sales growth outpaces non-orphan drugs, despite expected introduction of newer generics and biosimilars for non-rare conditions. Source: EvaluatePharma

Launching an orphan drug is very different from launching a standard, non-rare disease drug. For companies to successfully commercialize rare disease drugs, they need to understand the critical differences between traditional and non-traditional (orphan) commercial models and rely on a new playbook to capture and maintain market share.

Caption: Rare disease companies face different challenges in effectively deploying their commercial budget, namely in developing the market by identifying patients, targeting leading treatment centers of excellence, and establishing KOL partnerships. 

What makes commercializing rare disease drugs different? Rare disease drug companies need to consider launch from a patient-focused mindset rather than a traditionally physician-focused one. While traditional drugs depend on physician adoption and assume widely educated and diagnosed patient populations, the opposite is true for orphan drugs. Rare disease companies will have to manage their commercial budgets very differently, focusing on patient needs and setting the stage for:

• Targeted sales force development: In the rare disease drug space, quantity does not mean quality. Sales teams need to address patient populations specifically, ensuring they are reaching the right customers with the right messaging. Small, agile, and cross-functional commercial teams will contrast with the larger, specialized sales teams used for traditional big-name therapies.
• Stakeholder education: Identifying the right physicians, patients, and care settings is a challenge unique to the orphan drug market. Companies should expect to develop a market where limited educational resources, physician training, and diagnostics tools regularly inhibit accurate diagnoses of patients with rare conditions. Rare disease companies must be ready to develop their markets through targeted educational efforts and be armed with a thorough understanding of the addressable space. Many rare disease companies have established one-on-one nurse case management services to provide “personalized product support” to patients with treatment information, coverage options and authorizations, financial assistance, and care navigation. Examples of these services include Alexion’s OneSource Treatment Support and Shire’s OnePath program.
• Patient advocacy: Physicians and patient advocacy groups (PAGs) are often an essential part of care management for many patients. By sharing educational resources, helping to address financial issues, and creating awareness for patients to enroll in clinical trials, PAGs present themselves as a necessary resource not only to patients but to orphan drug companies as well.  Drug companies should strategically align themselves with these types of groups to locate and engage patients throughout the drug approval process.
• Effective KOL partnerships: In orphan diseases, it is common for a small group of specialized physician researchers and Key Opinion Leaders (KOLs) to manage and determine treatment for a significant portion of an orphan disease population through reference centers of excellence. Companies should consider involving KOLs with late stage clinical studies to engage patients early and potentially jumpstart early access initiatives, such as compassionate use. KOLs are also valuable assets during drug development; a collaborative approach, for example via advisory boards, fosters trust and confidence in the product all the way through launch.
• Pricing and market access: For US companies, outcomes-based pricing and value-based contracting will become a primary focus for rare disease companies moving forward. Last year, Alnylam announced a value-based selling strategy for Patisiran, an increasingly popular approach to make the high cost of these drugs more palatable. In contrast, companies launching in Europe will face challenges as the market prepares for continued high cost drug launches and a higher bar is set for outcomes in rare diseases with one or more marketed therapies.                                                 

Rare disease drug companies must be ready to actively engage in market development and support targeted efforts within this fragmented arena. In the next part of this series, we will understand why stakeholder education plays a critical role in market development for rare disease drug companies and how they can successfully capitalize on the opportunity despite the unique challenges it presents.