Fueling the future: the role of biotech in advancing innovative science and delivering new medicines

Advancements in the exciting field of gene therapy, as an example, are largely driven by the biotech industry, and thanks to the innovative new medicines being discovered and developed by biotech companies, patients with genetic disorders now have more and better treatment options.^2-4 Biotech companies are helping to uncover underlying causes of diseases, better understand how to slow disease progression, and maybe even find cures for certain conditions. But with high reward comes high risk.

The risk comes not least in funding the complex, unpredictable, expensive, and time-consuming business of biotech.  Research and development take time and require substantial investments – on average it takes more than a decade to bring a drug to market and costs close to USD 2 billion.^5,6 This is an expensive waiting game for many shareholders who would like to see a faster return on their investment.

The obvious risks and challenges for biotech companies are associated with applying cutting-edge science to develop transformative new medicines and funding such pioneering long-term efforts while being exposed to volatile financial markets. In addition, there are three key challenges many biotech companies face as they mature and advance pipeline projects towards regulatory approval and market launch – 1) transitioning from early-stage development to full-fledge commercial-stage biotech companies, 2) navigating disparate regulatory agencies, and 3) securing pricing and reimbursement for new products.

Transitioning from an R&D-driven organization to a full-fledged commercial-stage biotech company is a resource-heavy, complex, and yet necessary transformation to ensure that new medicines can be taken from bench to bedside. It requires balancing resources within an organization, simultaneously acquiring the right talent to support both R&D and Commercial activities and establishing new ways of working and expertise. Traditionally, biotech companies are known for their scientific research and clinical development expertise. Building out commercial expertise is a huge shift for a small organization.

Navigating various regulatory agencies can also be challenging. In some instances, regulatory agencies have not evolved to keep pace with innovation or lack input from patients early on in discussions. This can be a time-consuming and costly effort, particularly for small biotech companies that need to collaborate with regulatory agencies to meet data requirements and seek approval.

The good news is that the growing number of approvals – with an all-time high of FDA approvals observed in 2023⁷ – indicates that the recent and ongoing implementation of more flexible approaches by regulatory bodies – such as conditional approvals or accelerated approvals with post-marketing confirmatory studies,⁸ additional tax credits or fees exemption, and potential market exclusivity for a fixed number of years^9,10 – is paying off. This results in bringing innovation to patients and clinicians quicker, which can help small biotechs commercialize new treatments in parallel to advancing data and science.

It also is a more fluid, modernized approach that recognizes the advances being made through science even in the face of limited clinical experience that can be gained in post-marketing requirements.

Finally, the lift to commercialize a product starts with gaining market access. This requires negotiations with healthcare systems in each country to set pricing and seek reimbursement. In Europe, this is a multi-year and multi-country effort that can take anywhere from three to five years. It requires a high level of acumen and understanding of the pricing and reimbursement frameworks for each country and the ability to engage directly with governments and healthcare systems.

When I joined Hansa Biopharma in 2018 (then known as Hansa Medical) it was a small R&D-driven organization of about 30 individuals – each with a strong commitment to advancing impactful science. Over the last 5 years, we have transformed Hansa into a burgeoning commercial-stage biopharmaceutical company with a diverse workforce of more than 30 nationalities and the necessary expertise across market access, corporate affairs, medical affairs, marketing and analytics.

With conditional authorization in Europe obtained in 2020, we are navigating the multifaceted EU regulatory system to ensure patients can access our treatment while ensuring the price reflects the value of our product to the patients who need it most. While balancing the growth of the organization with the right resource allocation will remain a challenge, we have a highly experienced leadership team well-equipped to advance the science and the business.

The inherent risks and significant commitment in time and money associated with scientific advancement coupled with the time, resources and activities needed to successfully commercialize a product require believing in the long-term value creation potential of biotech. It requires savvy, long-term, specialist investors who understand both the business and the science of biotech. And ultimately, it requires believing in the innate value of scientific discovery and the development and delivery of new medicines to patients with serious unmet medical needs.

At Hansa Biopharma, our mission is to develop innovative, lifesaving and life altering therapies, bring these to the patients with rare diseases and conditions who need them, and generate value to society at large. We are delivering on our mission and laying the groundwork for having a meaningful, positive impact on many more people’s lives in the years to come.

Søren Tulstrup, President and CEO, Hansa Biopharma

References

1. Beyond borders: EY biotechnology report 2022.
2. Hanna E, et al. Gene therapies development: slow progress and promising prospect. J Mark Access Health Policy 2017;5:1265293.
3. Carvalho M, Sepodes B, Martins AP. BMJ Innov 2021;7:123–134.
4. De Luca M, Cossu G. Cost and availability of novel cell and gene therapies: Can we avoid a catastrophic second valley of death?: Can we avoid a catastrophic second valley of death? EMBO Rep. 2023 Feb 6;24(2):e56661.
5. Singh N, et al. (2023) Drug discovery and development: introduction to the general public and patient groups. Drug Discov. 3:1201419.
6. Sabatini, M.T., Chalmers, M. The Cost of Biotech Innovation: Exploring Research and Development Costs of Cell and Gene Therapies. Pharm Med 37, 365–375 (2023).
7. Senior, M. Fresh from the biotech pipeline: record-breaking FDA approvals. Nat Biotechnol 42, 355–361 (2024). https://doi.org/10.1038/s41587-024-02166-7
8. FDA’s accelerated approval pathway: A rare disease perspective. National Organization for Rare Disorders. Available at: https://rarediseases.org/wp-content/uploads/2022/10/NRD-2182-Policy-Report_Accelerated-Approval_FNL.pdf
9. Designating an Orphan Product: Drugs and Biological Products. Available at https://www.fda.gov/industry/medical-products-rare-diseases-and-conditions/designating-orphan-product-drugs-and-biological-products
10. European Commission. Orphan medicinal products. Available at: https://health.ec.europa.eu/medicinal-products/orphan-medicinal-products_en