The drug development pipeline has evolved from primary care to specialty and now advances deeper into exquisitely targeted therapies for shrinking niches of addressable populations. In parallel, governments and private payers continue to struggle with appropriately rewarding innovation while managing healthcare budgets in the face of aging populations. Given these trends, our value, pricing and access practice is increasingly supporting clients in four critical areas:
- Innovating evidence development approaches
Much of today’s innovative drug development pipeline does not conform readily to traditional value assessment approaches. For example,
- The value of “one-time” cell and gene therapies is heavily dependent upon durability of effect/benefit, which is challenging to establish at launch
- Rare disease therapies generally have difficulties enrolling large trial populations necessary to power head-to-head assessment vs standard of care
- Targeted oncology therapeutics often are trialled in a dynamic environment where standard of care is a moving target and differs across global markets
Our clients are increasingly challenged to re-imagine and generate stakeholder buy-in to novel evidence development approaches, spanning the needs of both regulatory agencies and market access stakeholder. Innovations we support include identifying novel trial endpoints and trial designs, greater incorporation of quality of life and patient reported outcomes, and novel use of real-world data and technology into evidence programs. The investment in evidence development both starts earlier and continues longer (post-marketing) to support and retain access and favourable pricing.
- Leveraging the increasing availability and acceptance of real-world data (RWD) sources pre-and-post launch
A key objective of HTA bodies and payers when assessing new medicines and negotiating pricing and access is to determine not only the potential value of the medicine based upon trial results but the likely value in the real-world setting. RWD is being leveraged to both substantiate therapeutic benefit in real-world usage settings, as well as to develop the evidence base to support initial HTA and payer discussions. As it becomes increasingly difficult for pivotal clinical trials in oncology and rare diseases to conduct comparisons with an appropriate comparator – or, indeed, any comparator at all – RWD is used alongside powerful statistical methods to construct a synthetic control arm to allow a comparison with a relevant comparator. In addition, RWD is increasingly used to provide support for the burden of disease that is addressed, justify comparators for HTA and access discussions, provide data inputs for economic models, and establish the natural history of disease. RWD is also increasingly used, often alongside data from clinical trials, to develop predictive algorithms that help to improve diagnostic outcomes or identify patients more likely to succeed on particular treatments.
RWD programs can leverage existing data infrastructure, e.g., claims and EHR databases, with data and analytics vendors growing more sophisticated in stitching together data sets to power greater insights. Additionally, heavier investments are being made to generate new and better sources of RWD, e.g., patient registries in rare diseases, and sophisticated infectious disease monitoring programs to support burden of disease substantiation for vaccine recommendations.
- Developing novel contracting and tender approaches
Biopharma clients are pressed to meet the needs of a wide array of budget holders across markets with differing budgetary constraints and cost-control tools at their disposal. Budget holders also have differing negotiation leverage depending upon a variety of factors including the criticality of the unmet need, pressures from external stakeholders, and the competitive therapeutic environment. Navigating these complexities to balance the needs of stakeholders – appropriate patient access, budget-holder affordability, and biopharma competitive position and profitability – is extremely challenging, requiring both local strategies and substantial global coordination as price transparency and international reference pricing initiatives expand around the world. Contracting and tendering considerations include such approaches as simple discounts and rebates, value- and outcomes- based and other innovative agreement structures, performance-based approaches, portfolio approaches, payment over time, and in some cases may also include ancillary considerations such as localized manufacturing or value-added services.
- Effectively addressing patient out-of-pocket costs and gaps in access
Patient out-of-pocket costs have become a critical access hurdle, not just for patients in the US but across an increasing number of developed and developing markets. In addition, some payers are increasingly pushing back on biopharma approaches to out-of-pocket cost mitigation. Our clients are challenged to innovate their approaches to drive patient affordability while balancing gross-to-net considerations.
In addition, gaps in access (e.g., uninsured patients, or critical need patients in markets without reimbursement) are a heightened challenge for advanced and targeted therapeutics, where high therapy costs, one-time use (in some cases), and limited addressable populations are all factors that make it more difficult for biopharma companies to make alternative access and patient assistance programs broadly available.
Optimizing market access strategies
Looking ahead, we anticipate a continuation of these trends and ongoing pressure to innovate value substantiation and access stakeholder engagement strategies on pace with biopharma’s clinical innovation. We continue to invest in our Value, Pricing and Access teams’ expert capabilities, tools and scale to meet our clients evolving needs.
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Authors: Eric Auger, Partner and Practice Head, Putnam; Alex Busch, Ph.D., Partner, Putnam; Joanne Evason, Principal, Putnam, Michael Lees, Head of Strategy and COO, PHMR