Maximizing European Market Access: Guidance for Young Biopharmaceutical Companies

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Achieving efficient and profitable market access for next-generation pharmaceutical products is extremely challenging. The number of drug launches is rising every year, taking competition levels higher with them. And because these novel products tend to be more tightly targeted to smaller patient populations than the “blockbuster” drugs of old, their pricing/reimbursement terms need to be tailored to match. This is especially the case with highly complex biologic drugs, which typically are expensive to research and develop. Below I offer a five-point plan to help small and start-up companies cut through the associated market complexities.

In 2021, the number of innovative biopharmaceutical product launches was significantly higher than the number launched in 2020. In 2022, experts predict that the volume should rise again as part of a sharp upward trajectory between now and 2026 (1). Many of these exciting new products are developed by small players, which inevitably come up against established names in trying to secure market access and optimize the terms of pricing and reimbursement.

Startups and modest-sized biopharmaceutical teams face a number of very real difficulties — both practically and commercially — in taking their products to market unaided by partnerships or marketing agreements. Compared with traditional small-molecule drugs, for instance, today’s tightly targeted biologics offer their developers smaller target markets. Consider too that such products require deeply involved product and process development, ultimately making them more expensive to make. The resulting
costs must be justified by benefits provided to patients if the drug sponsors want to generate profitable returns. Thus, biologic innovators and marketauthorization
holders of novel treatments need to proceed with a strong strategy and carefully thoughtout approach to optimize market access and make it possible to compete effectively in target countries.

Representing the second largest market globally for new drugs after North America, Europe today is only the third largest registrar of new drugs (behind North America and Asia) (2, 3). For sponsors located elsewhere, that makes the European community a commercially attractive market. The market, however, is highly diverse and fragmented: Each country has its own needs for new treatments and specific regulatory nuances. Those requirements continue to evolve over time. With the United Kingdom is now outside of the European Union, Brexit has added further complexity. Now the United Kingdom is an important market in its own right — and follows its own priorities and rules.

A Five-Point Plan
Young biotechnology biopharmaceutical companies can cut through those market  complexities to get their new products to market — particularly in Europe — both swiftly and cost efficiently. It requires a strategic approach that maximizes efficiencies, beginning with an initial target market and a core dossier of important information.

Develop a fit-for purpose market-access strategy. As blockbuster drug development gives way to more targeted and personalized medicines based on complex proteins, living cells, and genetic materials, the parameters involved in assessing, vetting, and commercializing such products inevitably will bear little resemblance to those used in reviewing traditional pharmaceuticals.

Specific expertise is necessary for carrying out a finely tuned pharmacoeconomic impact study on the community associated with a given medical condition and for communicating the benefits of innovative new therapies and treatments to regulatory authorities, payers/insurers, physicians, patients, and potential financial investors. When small-company managers seek help with devising and executing an optimal market access strategy, it is important that such support be tailored to the particular organization and its area of focus. That will help to maximize success and ultimately to secure the best pricing and reimbursement terms. It requires an intimate understanding of which countries will be best to target first for a particular treatment — and what it takes to get a dossier to the top of a fast-growing pile within a particular agency or commission so that it will be reviewed as a priority.

Maximize efficiency with a combined strategy for registration and market access. Applying for market authorization of a new biopharmaceutical product inevitably involves collating many valuable and detailed documents and data. Such processes and content should feed seamlessly into market-access strategy development. Aligning both streams of activity and working on them in parallel rather than sequentially will help to focus thinking, strengthen the overall narrative surrounding a new medication, accelerate its progress to market, and prevent duplication of efforts.

Start with a core dossier that serves most market needs. Although European market access ultimately will be achieved country by country, a successful approach will blend central, regional know-how and preparation with local applications. I advise presenting a core set of benefits and collecting a core dossier of information with about 80% of common messaging and content that will be the same for all markets. This can be developed in parallel with preparations of chemistry, manufacturing, and controls (CMC), as well as product-testing materials for market-access applications. That will ensure close alignment and provide optimal potential for resource and content sharing.

Select an initial target market based on maximum opportunity. With those most critical building blocks in place, commercial strategy teams can start to build out a market-access dossier for the primary target market. That would be the country you’ve identified as having the best potential for optimal pricing/reimbursement of a given therapeutic product or vaccine.

A knowledgeable partner should be able to help company decision-makers determine the best choice based on a number of factors:

• market size — in particular, the target population of patients with a particular condition and demand for the new treatment.
• local authority attitudes toward innovative treatments, including specific policies or “fast-track” processes designed to accelerate market access and/or compensate drug developers more generously for their innovative work.
• current and potential competition — what’s already on the market, available room for a new entrant, and so on.
• existing company activity or relationships (such as affiliates and partners) in a given market.
• possibility of manufacturing the product locally (whether through outsourcing, partnership, or an owned facility).

Complete a market dossier for that target country to use as a template in the future. With the core 80% of your market-access dossier developed already, you can proceed with final finessing for the primary market. That will require drawing on local knowledge of that country and of the particular priorities and preferences of local authorities’ key decision makers. Potential service partners with local in-country expertise as well as central regional knowledge could be well placed to help you find the “sweet spot” between general appeal and applicability and targeted messaging for each specific market.

Once this first dossier has been completed and approved for the preferred/primary country, then it can serve as a template for other markets going forward. That will minimize the amount of rework for each successive market. Here too, local experts should be able to help adapt the final 20% of input that is market specific, boosting your chances of success in accessing each country on the most beneficial terms.

The 80/20 Split
When creating market-access dossiers for Europe, a company must ensure that its clinical development plan is aligned to meet both with the sponsor’s own commercial goals and the particular needs of each market. For efficiency, the ideal is to aim for an 80% common core, with the balance being content that has been customized for local requirements.The majority of the dossier is common to all countries and should address.

• clinical and economic impacts.

• a plan to achieve market access at an optimal price.

• the new drug’s ability to improve patient outcomes and reduce burden on healthcare systems.

• integration of patient-reported outcomes into healthcare decisions.

Beyond those basic heterogeneous considerations with Europe-wide application, the remaining 20% of the dossier should provide details linked to individual policies and processes that are specific to each particular target market.

Further Considerations
Local market knowledge and connections are essential to removing potential blocking points, smoothing the way for progress, and ensuring that dossiers have a strong narrative that clearly “sells” the unique benefits of a proposed new treatment in terms that matter most to a given population and its overseeing authority. Even so, the path to market access may not be straightforward — in fact, it seldom is, particularly when it depends on the outcome of ongoing clinical studies or if a product and its associated dossiers are especially complex. Navigating all the potential options requires specialist expertise, whether to advocate the best clinical trial objectives or to propose and help find alternative funding mechanisms and equivalents. Possibilities include risk/benefit-sharing (e.g., with authorities, hospitals, or health centers), to bring a new drug to market. Managers of young companies should seek out expert advice on the complete spectrum of options.

When surveying markets for a prospective consulting or full-service partner, young companies need to consider to what extent each provider can simplify the process of market access for protein biopharmaceuticals, vaccines, and advanced-therapy medicinal products (ATMPs), whether those treatments are completely new or already have enjoyed
success in other territories. The right strategy should be tailored to the needs of the biopharmaceutical industry — particularly in spanning and connecting regulatory, registration, and marketaccess support (while bridging strategy, governance, finance, human resources, and more). These early decisions can be critical in obviating regional market complexity and in streamlining delivery of vital new treatments to the patients who need them.

What’s in the Dossier?
A market-access dossier serves as a platform for discussion and helps biomanufacturers make a convincing argument for the value of a product to the market. Information should be well structured and usable by field staff working in health policy. It also provides essential information for internal staff training about a drug. For example:• comprehensive background on the disease and indication.

• description of current regulatory guidelines and health authorities of the intended market.

• statements from pharmaceutical evaluators.

• descriptions of pharmaceutics and pharmacology.

• summary of clinical-trial results.

• (meta)analyses of systematic reviews and available care for the disease.

• details of drug costs and analyses of cost-effectiveness and budgetary impacts.

—adapted from Ecker + Ecker GmbH (Hamburg, Germany)
https://www.ecker-ecker.de/english/marketaccess-dossier.html

References
1 Mikulic M. Leading Biotech and Pharma Product Launches in 2021 and Revenue Forecasts for 2026. Statista 3 February 2021; https://www.statista.com/statistics/407953/revenue-forecast-of-toppharma-and-biotech-launches-worldwide.

2 The Pharmaceutical Industry in Figures: Key Data 2021. European Federation of Pharmaceutical Industries and Associations: Brussels, Belgium, 2021; https://www.efpia.eu/media/602709/thepharmaceutical-industry-in-figures-2021.pdf.

3 Du S. Pharmacy of the World: China’s Quest to be the No. 1 Drugmaker. Nikkei Asia December 2021; https://asia.nikkei.com/Spotlight/Coronavirus/COVID-vaccines/Pharmacy-of-the-world-China-s-quest-to-bethe-No.-1-drugmaker.

Jean-Luc Taborin is vice president and head of ProductLife Consulting in Paris, France; jtaborin@productlife-group.com; https://www.productlifegroup.com. He has been a consultant at Ernst & Young and worked for Sanofi, Eurofins, Deloitte, and Roland Berger.