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.

The Role of Stakeholder Education in Rare Diseases

Despite the growing pipeline of orphan drugs, ~95% of rare diseases do not yet have an FDA-approved treatment. This increasing momentum in rare disease R&D has further highlighted the ever-present need for stakeholder education and engagement, which is critical to begin well before drug commercialization. Patients, key opinion leader (KOL) physicians, and other physician specialists supporting treatment are primary players in the rare disease space that influence treatment selection and access to novel therapies. Rare disease stakeholder education plays a critical role at every stage in drug development and commercialization and should be top of mind for rare disease companies regardless of whether they are designing clinical trials or preparing for product launch.

Caption: KOLs play an important role in the patient journey. From diagnosis to treatment access, KOLs support rare disease patients in making important decisions about managing their condition and obtaining the best possible care.

How to Implement Rare Disease Stakeholder Education

Rare disease companies need to engage with different stakeholders to address various pain points along the patient journey and maximize the opportunity for their orphan drug. KOL communities will be especially valuable throughout the drug development process. As a part of tight-knit medical communities, KOLs collectively influence treatment standards and typically have a deep understanding of how access to care and treatment varies across global markets. KOLs will be play important roles in:

• Diagnosis: Timely diagnosis remains a major challenge for rare diseases; on average, patients wait close to 5 years and visit over 7 physicians before an accurate diagnosis is made. Delayed diagnosis can further complicate the patient pathway, often increasing the number of interventions required after the disease has progressed. Dedicated education efforts targeting physicians and patients, as well as improved testing access, can ensure accurate and earlier diagnoses.  Companies should look at this as an opportunity to educate stakeholders and develop genetic tests to increase diagnosis rates and maximize patient adoption. While there are currently no genetic tests available for many rare conditions, competition to develop effective diagnostic tests will increase after a disease modifying treatment is introduced to the market. KOLs can also support development of innovative screening techniques, including using artificial intelligence or predictive analytics with electronic health record data to identify patients at high risk of an undiagnosed rare disease. 
• Drug development: KOLs play an integral role in drug development, ranging from supporting animal model design, clinical trial design, and designing clinical endpoints (e.g., novel disease severity / quality of life scales). As disease experts, KOLs understand needs, market access challenges, and adoption drivers and should be considered an asset to pre-commercialization efforts for rare disease companies. KOL in-depth expertise and knowledge should be leveraged for refining target product profiles (in particular via advisory boards), understanding treatment barriers across markets, estimating the size of local patient populations, selecting sites and recruiting patients for clinical trials, and supporting market access and reimbursement decisions for regulatory bodies and payers. Companies will greatly benefit from early and global KOL engagement throughout the drug development process – whether as consultants or scientific investigators.
• Launch planning: KOLs are very familiar with the gaps in the existing patient pathway, and, post drug launch, can provide insights on ideal future-state diagnosis and referral patient pathways. Rare disease companies should continue to collaborate with KOLs to streamline the access pathway and demonstrate their long-term commitment to a given patient population. Post-launch trials also offer an opportunity for KOLs to disseminate information and develop stakeholder education deliverables about achieved health outcomes and / or pivotal trial results with physicians and patient advocacy groups (PAGs). Over time, KOLs become strong advocates for drugs, especially those that they have supported through development, and can be important allies as companies move towards commercialization. 

Caption: Companies looking to launch a new drug should focus on how they can work with KOLs and other stakeholders to best understand local market opportunities, including disease prevalence/incidence, frequency of diagnosis, and likelihood of treatment adoption.

Centers of Excellence (COEs) are another critical component of rare disease stakeholder education. COEs, in conjunction with KOLs and their reference networks, offer rare disease patients specialized care and expertise. COEs combine clinical research, knowledge, and treatment services to become a regional or global “hub” for undiagnosed or recently diagnosed patients. The multidisciplinary approach they take is not only valuable to patients, but also biopharma companies as they enter commercialization stages. COEs are a reliable source of support for companies, referring patients from their centers and supporting commercial activities later down the road. A strong relationship with COEs and their KOLs can mean success for rare disease companies looking to work closely with patient populations and demonstrate long term commitment.

Caption: Centers of Excellence are a critical component of the rare disease patient pathway. Patients seek diagnosis support, treatment advice, and care coordination from experts at COEs across the globe.

KOLs and COEs are key stakeholders in the rare disease space and should be top of mind for companies looking to enter the space. As competition within the market increases, how companies work with KOLs to leverage their expertise and best understand the treatment landscape will become a key differentiator. KOLs, though critical to the market, are not the only stakeholders that companies should keep an eye on. In the next part of these series, we will share why patient advocacy groups are an important ally for both patients and rare disease companies, and how they can successfully work together to introduce new treatments to the market.