Preparation is key; it’s important to understand current clinical practice and policy levers that could create opportunities for your intervention and be proactive in considering the evidence required to gain reimbursement and access. This means engaging early with stakeholders to understand what value means to them and the data needed to demonstrate it. Early engagement is as important internally as it is with customers and users. The different objectives of clinical trial groups and market access teams may hinder a pharma company’s ability to identify, co-ordinate, and collect data that will either help pricing and reimbursement or demonstrate value to clinicians and patients. With clinical trial programs in rare diseases naturally infrequent, it’s important to establish – from the very beginning – the endpoints that are likely to resonate with your key stakeholder groups. This will only come through proactive (and ongoing) engagement with external influencers and joined-up thinking across the organization. Direct engagement at this early stage with patients, or through patient advocacy groups (PAGs), provides an invaluable opportunity to incorporate the patient voice.
Agility is crucial. The healthcare environment is dynamic, so pharma companies must be able to react quickly to what they learn.
Traditional approaches to developing evidence are unlikely to be successful (or even possible) for rare diseases. Thus, it’s important to reimagine research, evidence, and pricing. Designing real-world studies is challenging, but with proactive engagement and creative thinking there’s much that can be done to capture data that enhances a value proposition. For example, it’s possible to develop a detailed understanding of current patient pathways and standards of care and use it to model what a new intervention will displace and how that will impact clinical practice.
Similarly, given the common absence of active comparators in rare disease studies, some pharma companies are exploring the possibility of using synthetic cohorts derived from real-world data as a comparator. By marrying a clinical trial with other existing datasets, literature evidence, or standard-of-care models, it’s possible to develop innovative but relevant real-world comparators that help you demonstrate differentiated value.
With extremely small patient populations in rare diseases, companies know that every patient in a study is gold dust; they can’t afford to lose anyone to follow up. There’s increasing investment in supporting patients through clinical trials – using a variety of technologies and engagement methods – to ensure that everyone recruited to a study stays on it. However, engagement doesn’t end at the clinical trial; pharma companies also need to consider long-term follow up throughout the patient pathway and how they sustain patient engagement in the real world. Innovation is critical. Successful pharma companies seek active involvement of patients as experts by experience, through building strong, long, and collaborative relationships with PAGs to ensure the patient voice is present and influential at every step of the patient pathway.
Finally, collaboration is key. Securing access to rare medicines is a team sport and pharma companies will not win if they play the game alone. Leaders recognize the importance of building enduring partnerships with all external stakeholders to understand the landscape and the real-world value drivers – enabling them to be alert and responsive to change as it happens. The most successful organizations partner with specialists in market access, real-world evidence, and patient engagement to develop patient-centered, outcomes-focused solutions that demonstrate value and drive access. The best partners have a deep understanding of the therapeutic environment, trusted relationships with stakeholders across the ecosystem, and expertise in designing bespoke research programs that capture the right evidence at the right time.
As advances in technology bring new hope in the treatment of rare diseases, health systems need innovation and support to ensure patients get access to the most effective medicines. It’s a simple goal that unites us all. The only approach is to think early and work together.