Putting plans in place to overcome all these challenges may be daunting and result in internalisation of teams during critical planning phases. Consequently, development and evolution of a value story that resonates across core stakeholder groups, clearly articulating the clinical and cost rationale for treatment and readying the market for implementation, may not be given due consideration.
Not adequately demonstrating and communicating value could delay market access, restrict the target patient population and result in low demand. At OPEN Health, we understand the importance of building a strategy that is underpinned by the needs of stakeholders across the value chain, and fuse value demonstration and communication in order to build a cohesive communications platform that drives a unified message through all stakeholder groups.
Demonstrating value to patients
The passive patient is becoming a thing of the past; now, more than ever, patients are the navigator of their own destiny when it comes to decisions regarding their treatment and healthcare. This is especially true for patients with a rare disease who, along with their parents and carers, must become the expert in their condition to ensure that they receive the right care that is aligned to their individual needs. Patient advocacy groups are hugely influential in this area, and their activity has been a key driver of health technology appraisal decisions in the past.
The introduction of gene and cell therapies is seen as a lifeline to some – a chance for a better future. Those with a rare disease, as well as their parents and carers, are keen to understand the good, the bad and the ugly to support them in deciding whether pursuing ATMP treatment is the right approach for them. Life science companies need to engage with patient advocacy groups early in the development journey to identify meaningful patient-centric clinical trial endpoints, build the narrative around immediate and long-term treatment considerations, and develop an appropriate patient support programme that openly discusses the complexities of ATMP treatment and provides materials to support patients to overcome the likely challenges in their treatment journey.
Demonstrating value to payors
With significant price tags, life science companies are under pressure to develop innovative pricing strategies that share the risk of treatment inefficacy. This risk needs to be balanced against the potential long-term benefit of treatment with an ATMP. Many national reimbursement systems implement a one-size-fits-all approach to assessing value, the parameters of which may prevent accurate representation of the burden of disease and impact on quality of life. These factors should be acknowledged during value assessments where there is a high medical unmet need and high levels of healthcare utilisation, both of which may be reduced by effective therapy. Life science companies need to find ways to demonstrate and quantify the value of ATMP treatment to the healthcare system over the long-term, and engage with payors to find ways to incorporate these into the value dossier and co-create appropriate pricing and value-based outcome models. Evidence generation strategies should be considered and carefully crafted with the end goal of ensuring access to the maximum number of patients who would benefit most.
Demonstrating value to clinicians
The efficacy of ATMPs continues to be the core driver of value for clinicians. When ATMPs demonstrate incremental benefit over current standard of care, as opposed to overall cure, clinicians will balance efficacy with safety and, importantly, the complex treatment process commonly associated with ATMPs. This may restrict the use of ATMPs to specific patient types in the minds of clinicians, which accentuates the need for a great first-use experience. With small patient populations currently the target for ATMPs, there is a core communication challenge for organisations trying to implement targeted educational campaigns aimed at clinicians most likely to be involved in patient selection for treatment. With an average of 7.3 physicians seen over the diagnostic period, it is critical that life science organisations fully understand the diagnostic and referral pathway to develop relevant targeted communication strategies. These strategies should aim to increase awareness of the symptoms of specific rare diseases, support patient identification through implementation of appropriate initiatives and enhance understanding of the potential value of ATMP treatment. In addition, there needs to be a dedicated support package for clinicians within centres implementing ATMP treatment strategies, ensuring clear communication of the complex processes involved in ATMP preparations and administration, supporting an evolution of the service model required to deliver ATMP treatment and developing a process for ongoing monitoring and assessment of long-term efficacy.
OPEN Health: supporting companies commercialising an ATMP
It is well documented that bringing an ATMP asset to market is no easy task, and there is little advantage in making an ATMP available if the patients who can benefit the most are unable to access it. At OPEN Health, we bring together expertise from across evidence generation, market access, healthcare professional communications and patient engagement to support companies commercialising an ATMP in navigating the route to market and create meaningful value strategies that consider the needs of all core stakeholder groups in order to facilitate successful market entry and ongoing uptake.