Forming early partnerships to understand the patient pathway

Written by Marie Keetley, Global Business Development Operations Lead on Thursday 8th July 2021

In a world of increasing complexity, the primary goal of any healthcare system is remarkably simple: to ensure patients have access to the best care and the best treatments for their condition. However, in a healthcare environment characterized by high demand and finite resources, meeting that objective is not straightforward, particularly when it comes to accessing breakthrough medicines. With affordability now one of the biggest factors in healthcare decision-making, today’s pharmaceutical companies are under tremendous pressure to demonstrate the value of their innovations to a broad range of stakeholders, each with different definitions of value. To do so, they must capture the right evidence; however, as therapeutic understanding becomes more nuanced and stakeholder needs more diverse, this isn’t easy. It’s even harder in rare diseases, for which patient populations and clinical programs are naturally small. So, how can pharma companies build a robust evidence base to convince regulators, physicians, payers, and patients that a rare treatment merits reimbursement and adoption? It’s all about preparation, collaboration, and innovation.

The challenges

There are four main access challenges in rare diseases:

1. Small patient population

Populations of patients with rare diseases are small and dispersed, restricting the potential for large, randomized clinical trials. In some cases, pharma companies must seek a license for a product in countries where they have not been able to trial it in patients. This can present serious longer-term challenges, with reimbursement bodies often basing their decisions on data from large, phase 3 studies.

2. Limited clinical knowledge and understanding of patient pathways

In rare disease communities, there’s often limited clinical knowledge about disease burden and existing standards of care or patient pathways, which increases the importance of stakeholder education and market-shaping activities. Likewise, the expert community in rare diseases is often very small; in some countries, particularly those where trials have not taken place, pharma companies struggle to identify clinical experts to champion innovation.

3. High price

The challenges of clinical development mean it can be expensive to bring a rare disease therapy to market. This is often reflected in high prices as companies strive for a commercial return. The subject of drug pricing has long been debated, with pharma companies working hard to shift the emphasis from “cost” to “value.” In an increasingly price-sensitive environment, there are unique challenges when developing evidence to justify the price of new treatments for rare diseases.

4. Limited evidence

Traditional methods of gathering evidence are seldom available for rare diseases. Active comparators are often uncommon or non-existent, while safety and long-term monitoring data can be insufficient. Similarly, selecting the right surrogate endpoint can be problematic. These factors make it hard to develop the evidence to convince payers, HCPs, and patients that a drug is worth using.

So, how can pharma companies increase their prospects of getting rare cancer innovations to patients safely and quickly? The answer is to propose and execute their plans early.

Engage early

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.

Agile thinking

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.


If you have any questions or comments about our Rare Thoughts & Outcomes theme or want to learn more about our commitment to Rare, please get in touch.


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