With its focus on value, a traditional Global Value Dossier reinforces a common pitfall in clinical development and commercialisation. This is the idea that if a product works clinically, it will also be successful commercially. Let’s rebrand Global Value Dossiers (GVD) into Global Value & Access Dossiers (GVAD) and use them as a tool to start thinking about access strategically. GVADs are a lever for understanding access barriers deeply (through root cause analysis), early (when trials can still be influenced), and widely (by all functions in the organisation). As such, they allow for effectively anticipating and addressing access barriers, to improve patient access to innovation.
Global Value Dossiers (GVDs) are widely used within pharmaceutical companies to describe a new product’s value proposition to external stakeholders responsible for ensuring patient access.
They typically cover:
- the current unmet need of patients
- the mechanism of action of the new product
- the new product’s impact in terms of:
- clinical outcomes and other patient-relevant outcomes
- costs and cost offsets at the level of pharmaceutical budgets, healthcare systems, and wider society
Global Value Dossiers are missing the ‘Access’ part of the story
With its focus on a new product’s value, a GVD reinforces the excitement within a pharmaceutical company about a new product. The company has spent years and millions of dollars to bring the product to where it is today. The stakes are high, as ~ 90% of drug candidates fail during clinical development. Here is one of the few products that can improve patient outcomes and/or the way in which we deliver care, and that made it to the final stage of marketing authorisation. You can imagine the feeling.
However, with its focus on value, a GVD also reinforces a common pitfall in clinical development and commercialisation – the idea that if a product works clinically, it will also be successful commercially.
Value does not equal Patient Access
One or two decades ago, clinical value may have been enough to ensure patient access and commercial value. Today, ensuring patient access has become more complicated. Current GVDs neglect this reality, as well as the three key buckets of challenges related to bringing new innovations to patients:
- High cost of goods may require pricing beyond what payers are able and/or willing to pay
- Value assessment frameworks may need adaptation to capture the full value of a treatment
- Health systems are not always ready to start using a new treatment
The cost to manufacture an advanced therapeutic medicinal product (ATMP) completely differs from the cost of conventional small molecules and biologics. They can be as high as USD 500,000 to USD 1,000,000, excluding the costs associated with R&D and commercialisation. Consequently, payer willingness to pay can be at odds with sustainable pricing from a company perspective. Where today’s ATMPs may be achieving some reimbursement success in orphan indications, future ATMPs addressing larger indications are likely to become unaffordable.
Conventional value assessments are meant to seek certainty before reimbursement. Yet, many of today’s therapies inherently come with thin data packages and clinical uncertainty stemming from methodological challenges, related to:
- Ethical problems related to setting up a control group, due to the life-threatening or highly debilitating nature of the disease
- Very small trial populations due to the rarity of the disease
- Slow progression of the disease due to the efficacy of treatment and/or the nature of the disease
These challenges hamper manufacturers in meeting payer evidence requirements. They may not be able to show a comparison with a control group, demonstrate a significant difference between groups, or gather data on hard clinical outcomes within the timespan of the trial. Or, in the worst cases, all of the above.
Furthermore, not all value assessment frameworks capture the full value of new innovations, especially when the value goes beyond the health of an individual patient, such as the value of improving public health or addressing health workforce shortages.
Even when ATMPs obtain a positive reimbursement decision, substantial delays often still exist before they reach patients. These delays can be related to:
- Manufacturing
- Financing the high upfront costs
- Updating clinical guidelines
- Training healthcare professionals
- Identifying patients
- Setting up centres of excellence
- Updating care pathways
- Preparing for administration
These challenges often remain unanticipated, causing downward adjustments of revenue forecasts once therapies are in-market.
Access barriers need to be understood deeply, early, and widely
Deep understanding of the root causes of the anticipated access challenges allows for identification of what is needed to tackle these challenges: do you need redirection of the internal course of action and/or shaping of the external environment?
Early understanding of the access challenges is needed to allow for early strategy development, especially in when it comes to clinical trial design or working with external stakeholders to co-create tailormade solutions to ensure timely access for patients.
Wide understanding of the access challenges across the organization is needed to have all internal ducks in a row, fully aware of and focused on tackling the key access challenges in bringing the value of the new therapy to patients.
Let’s start leveraging the widely used GVD as a tool to start thinking about access in a more strategic manner. This rebranded Global Value & Access Dossier (GVAD) contains the same elements as a GVD:
- Why – The current unmet need of patients, clinicians, the health system, and society
- What – The mechanism of action of the new product and its impact in terms of:
- clinical outcomes and other patient-relevant outcomes
- costs and cost offsets at the level of pharmaceutical budgets, healthcare systems, and wider society
But it goes beyond that:
- How – The road to patients, the anticipated access barriers, and their internal or external root causes
This will make the GVAD a cross-functional steppingstone for developing early patient access strategies that address internal and external access barriers. As such, it will increase the chances of clinical success translating into patient access and commercial success – i.e., a sustainable model to drive innovation.
Want to know more?
At Inbeeo, we provide tailored solutions for today’s toughest market access challenges. We work with clients in the early stages of the development process to define the right value strategy and the right price to ensure patient access to complex innovations. We’re happy to discuss in more detail how best to identify, understand, and address key access challenges early in the development process.
