London

Sunjeet Sawhney

Market Access as an integrated function comprising several elements (e.g., HEOR, Pricing, Government Affairs, Patient Advocacy, RWE) has come a long way in the last decade, and the need for increasing capabilities is undisputed within the biopharmaceutical industry. The external environment, especially post-COVID, has introduced many new health policy reforms in various EU and international markets with an increased focus on cost containment measures. The payer landscape is evolving with cross-country collaboration networks, payer buying groups, pricing pressures (particularly on oncology, combination treatments, advanced therapy medicinal products, and orphan drugs), as well as Health Technology Assessment (HTA) requirements (cost-effectiveness analysis and joint clinical assessment at the EU level).

The current pharmaceutical policy reforms introduced in several international markets, combined with increasing market access hurdles, comprise a perfect storm for the pharmaceutical industry and have caused several companies to make U-turns on European markets altogether. A prominent example was the closure of Bluebird Bio’s Europe-based headquarters because they could not align a price with the German Association of Sick funds on their sickle cell gene therapy. This sent a “shock wave” to biotech companies located in Switzerland and across Europe, as increasing scrutiny and pricing sensitivity of European payers had spoken directly to biotechs when considering international expansion into Europe.

Many recently introduced cost containment measures and policy changes are often short-sighted and unfit for purpose (for example, not incentivizing medical innovation). There is, however, no need to be discouraged by the changing payer landscape. The key to success in Europe and international markets is appropriate clinical evidence to support HTA requirements. Market access organizations have developed, and capabilities are expanding; however, integral access thinking from top to bottom is not yet optimally executed in most companies.

Where are the opportunities?

Assessing new healthcare products in Europe boils down to determining whether the new product (B) offers superior benefits over the existing one (A) and by how much. Crucially, this evaluation hinges on whether these benefits are clinically meaningful to patients in terms of both size and significance. European payers also scrutinize uncertainties surrounding the product's safety profile and its cost-effectiveness. Despite clear theoretical expectations for successful HTA evaluations, practical challenges often arise. It includes single-arm trials, lack of randomization, absence of relevant real-life comparators, and insufficient hard endpoints early in development planning. These limitations frequently lead to frustrations, rejections, and protracted pricing and reimbursement negotiations.

Status Quo of Access Function and Organizational Models

Most companies successfully operate the “Triad” model, consisting of a closely formed team of Access, Medical, and Commercial at regional and country levels. Although, at the C-suite level, this often does not exist. According to a recent article from BCG, “most global MA heads are positioned at the CEO-2 level (only).” Some are appointed as Chief Access Officers, reporting directly to the company CEO, which signifies the expanded role and importance of market access in decision-making. The latter is still very rare, and most access people are still structured under commercial or medical leadership. This structural dynamic is one of the key reasons for failure. Senior access people not being at the table or only represented by the CMO and CCO and similar commercial functions frequently leads to misunderstandings and biased decisions in deal-making.

Ed Schoonveld emphasized this in a recent article: “In several surveys with US and EU payers, serious flaws in biopharma’s evidence packages are highlighted. Payers state that primary endpoints, trial comparators (or lack of comparators altogether), and length of trials are often deemed inappropriate to support value claims, thus resulting in low prices and/or severe restrictions on use. Addressing payers’ evidence demands to demonstrate real patient value may increase cost and risk of clinical failure, but simply ignoring it without a trade-off analysis is not likely the right answer either.”

Likewise, it is inconsistent to operate a triad model at the regional and country level but not at the C-level. If you have a Chief Medical Officer and a Chief Commercial Officer, why not have a Chief Access Officer? In many pharmaceutical companies and biotechs, R&D leaders are typically the ultimate decision-makers in drug development and trial design, emphasizing speed to regulatory success over market attractiveness. Speed to market is important for commercial reasons as well but not if it leads to a race to nowhere.

So, what needs to change?

Future of Access Leadership in Biotech and Pharma

Biopharma's future success in an access-focused landscape demands a strategic shift from ad-hoc to strategic access thinking thoroughly. This necessitates early and holistic involvement of access considerations in business development, early development, clinical trials, and lifecycle management. As McKinsey points out in a recent article, “Evidence generation is becoming increasingly important and complex, with heterogeneous needs between countries—for example, cost-effectiveness is prioritized in some markets, while clinical benefit is prioritized in others. All of this could be addressed with a single global evidence plan.”

Such an activity requires a strong voice and a functional “triad” at the highest level of the enterprise. Ed Schoonveld highlights a recurring issue: companies often face unexpected access and pricing challenges due to insufficient preparation. Despite instances where limited evidence suffices, the importance of deliberate decisionmaking and rigorous analysis remains critical.

Organizational Development

To unlock the full potential of a market access-embedded company strategy in commercial-stage biotech, a Chief Access Officer alongside the commercial and medical functions allows for a comprehensive and equally weighted opportunity assessment and strategy development. Oftentimes, people on the second level report to a commercial role instead of directly to the CEO and might feel their voice is limited. This can lead to poor investment decisions during M&A, clinical development, and opportunity assessments if relevant payer intelligence and experiences are not sufficiently considered at the top decision-making table. Replicating what has existed and succeeded at regional and country levels is critical for future success.

Smaller biotechs in the rare disease space, oncology, and ATMPs (particularly those without a portfolio of products) depend heavily on the success of the pricing and reimbursement submissions to reach a break-even point and achieve sustainability. Mistakes in access planning could lead to dire upstream consequences if the data generation does not deliver sufficient leverage with payer audiences.

If we want to avoid the ever-decreasing ROI in biopharma R&D output, we need to commit to bringing medicines to patients with significant and distinctive value. Revenue forecasts should be realistic and reflective of the true value of the development program. Clinical trials need to be designed with endpoints that include an international payer perspective tailored for pricing and market access success. Market access leaders should be empowered and positioned at the right level of the organization's decision-making hierarchy to ensure their voices are heard and have an access facet to development and commercialization strategies.

The creation of the CAO is overdue and critical to building successful pharma and biotech organizations ready for survival and prosperity within the evolving global healthcare environment.