Life sciences are integral to the European economy and its people. Over two years ago, this ecosystem had to rapidly come together, forging strong partnerships to deploy treatments at unprecedented scale.
This precedent should continue and be considered to aid people suffering from rare diseases. “Around 30 million people across Europe live with a rare disease1. On average, it takes six to seven years to obtain a correct diagnosis2 and over 40% of rare disease patients are initially misdiagnosed3 4,” says Ricardo Marek, President Europe & Canada at Takeda.
A strong policy framework incentivizing investments in the health care ecosystem and partnerships is critical. The region has strengthened its position through lessons learnt from the pandemic. But if Europe is to continue tackling the rising burden of diseases, it is critical that this momentum is maintained.
Marek adds: “To reinforce its global competitiveness, Europe needs to continually evolve its regulatory framework, adopting advances in technology, utilizing real-world evidence (RWE) and data analytics in a dynamic and iterative decision-making process.”
In conversation with POLITICO Studio, Ricardo offers valuable insights as to how this can be achieved — and the potential benefits that may ensue.
POLITICO Studio: What are some lessons learned and partnerships forged during the COVID-19 pandemic?
Ricardo Marek: The pandemic uncovered some of the components necessary to drive positive change. Multistakeholder partnerships were swiftly forged, with data and expertise shared to accelerate vaccine development and production5.
Takeda is proudly leading or participating in more than 100 research and development public-private partnerships across more than 75 countries6, dedicating the expertise of our scientists and our data, including H2O (Health Outcomes Observatory)7, ARDAT (Accelerating Research & Development for Advance Therapies)8 and Screen4Care9. These allow us and other stakeholders to go further to address challenges that could not be pursued alone and help accelerate progress toward delivering transformative medicines to patients.
PS: While rare diseases are, by definition, rare, the absolute numbers are still significant. What are some of the innovations Europe has achieved/is working on in this area?
RM: Around 30 million people across Europe live with a rare disease1. On average, it takes six to eight years to obtain a correct diagnosis, and over 40 percent of rare disease patients are initially misdiagnosed2.
It is critical for Europe to maintain a strong focus on innovation in medicines and health care infrastructure to improve diagnosis and deliver health care management and treatment options to these patients. Alongside raising awareness, Europe has developed a vibrant research and development community dedicated to improving the lives of rare-disease patients.
Some benefits have been reported for patients suffering from rare diseases. The European Commission has already authorized more than 200 orphan medicinal products (OMP), since the adoption of the EU Orphan Drug regulation in 200010. Moreover, the frameworks for addressing rare-disease patients’ needs in Europe have been significantly upgraded in the past 20 years, transforming care and innovation standards11.
Partnerships have been critical to the evolution of Europe’s rare disease ecosystem and remain essential for addressing unmet needs. Similarly, value-based partnerships unlock innovation and exert positive impact by delivering better outcomes for patients12.
PS: The OMP Framework has increased innovation in Europe, but disparities in access to medicines remain. How do you think people living with rare diseases can be supported better?
RM: Rare disease patients across Europe do not have equitable access to therapies13. Innovative payment models and early evidence dialogues may support broader and faster access to medicines.
Multiple challenges remain during patient journeys, ranging from diagnosis delays to inequitable access to treatment. These include slow regulatory processes, late initiation of market access assessment, duplicative evidence requirements, reimbursement delays and local formulary decisions. As the root causes are multifactorial, they can only be solved by different stakeholders working together14.
At Takeda, we have developed strong partnerships with trade associations such as the European Federation of Pharmaceutical Industries and Associations (EFPIA), EuropaBio and European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) and support the call from patients by the European Organisation for Rare Diseases (EURORDIS), for a European action plan on rare diseases and to make rare diseases a health priority. Alongside an evolved incentive framework, partnerships can effectively address barriers across the OMP lifecycle.
PS: There is increasing demand for treatments for people who live with rare diseases that previously have not had access to approved therapies. Considering this, how does Takeda build and maintain access to treatments across the whole lifecycle, from the initial clinical trials, to delivering affordable medicines to end-users?
RM: Access starts with science. A good example is next-generation sequencing (NGS) which has enabled rapid sequencing of the base pairs in DNA or RNA samples. This has opened the way for new technologies — and hence for developing innovative therapies for rare diseases.
Despite significant improvements, unmet needs persist among rare disease patients. An obvious unmet need includes the 95 percent of rare diseases that today have no authorized treatment11. Prevalence estimates suggest that these 95 percent affect a small fraction of the rare disease patient population1.
If we can determine if a medicine is going to succeed early in its development process, we can avoid placing patients, caregivers and health care providers in unnecessary stress. This requires companies and regulatory bodies to partner around a common goal of advancing promising therapies and terminating those that are not.
Furthermore, we need to rethink how we improve patient access in Europe. This requires companies, disease experts, patients and payers to come together and find creative solutions that allow for equitable access. We need to look at setting up partnerships between stakeholders early in the development process and explore ways to leverage digital technologies and alternative data sources where randomized clinical trial data is not available for ethical or other reasons.
PS: What part does data play in this pipeline? And how is Takeda leveraging its data across the medicine lifecycle?
RM: Data and evidence are crucial to medicine development and innovation. Along the value chain, data shapes research and development and clinical trial design, measuring and demonstrating the efficacy, safety and benefits of a product in development.
Randomized controlled trials are often not feasible for rare diseases due to small patient populations and variable disease courses, thus challenging the ability to produce sufficient evidence. However, real-world data is increasingly being collected through various alternative sources, allowing R&D developers to better understand disease lifecycles over time, explore unmet medical needs and study treatment outcomes.
One way in which Takeda is unlocking such potential is through a partnership with RWE4Decisions — a payer-led, multistakeholder learning network exploring RWE15. It highlights the need for robust RWE and accelerated development pathways to foster highly innovative technologies. Through this partnership, Takeda has contributed to the European Health Data Space initiative16 aimed at creating a harmonized healthcare data space which delivers for patients6.
Commitments such as this from major stakeholders whether in the European Commission, industry, academia, or health care systems will help to foster innovation in health care in Europe and beyond to deliver better outcomes for all, through standardization and patient-reported outcomes17.
Takeda is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people, and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology. We also make targeted R&D investments in plasma-derived therapies and vaccines. https://www.takeda.com/
C-APROM/EUC/CORP/0001 /October 2022
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2 Geng LN, Sum-Ping O, Geng YJ (2019) Phases of the Diagnostic Journey: A Framework. Int Arch Intern Med 3:013. doi:10.23937/2643-4466/1710013
3 Nothaft, W., Goldsmith, C., & Le Cam, Y. (2020). It takes far too long for a rare disease to be diagnosed. Here’s how that can change. Global Health: World Economic Forum. Retrieved 14 October 2022, from https://www.weforum.org/agenda/2020/02/it-takes-far-too-long-for-a-rare-disease-to-be-diagnosed-heres-how-that-can-change/.
4 Vandeborne, L., van Overbeeke, E., Dooms, M., De Beleyr, B., & Huys, I. (2019). Information needs of physicians regarding the diagnosis of rare diseases: a questionnaire-based study in Belgium. Orphanet journal of rare diseases, 14(1), 99. https://doi.org/10.1186/s13023-019-1075-8
5 Vuong, QH., Le, TT., La, VP. et al. Covid-19 vaccines production and societal immunization under the serendipity-mindsponge-3D knowledge management theory and conceptual framework. Humanit Soc Sci Commun 9, 22 (2022). https://doi.org/10.1057/s41599-022-01034-6
6 Takeda, “2022 Annual Integrated Report” (Tokyo, 2022), https://www.takeda.com/49fefa/siteassets/system/corporate-responsibility/reporting-on-sustainability/annual-integrated-report/Takeda_2022_annual_integrated_report.pdf.
7 “Health Outcomes Observatory,” accessed October 21, 2022, https://health-outcomes-observatory.eu/.
8 “Ardat – Accelerating R&D for Advanced Therapies,” accessed October 21, 2022, http://ardat.org/.
9 “IMI Innovative Medicines Initiative | Screen4Care | Shortening the Path to Rare Disease Diagnosis by Using Newborn Genetic Screening and Digital Technologies,” IMI Innovative Medicines Initiative, accessed October 21, 2022, http://www.imi.europa.eu/projects-results/project-factsheets/screen4care.
10 Orphan medicinal products. European Commission website. (2022). Retrieved 30 September 2022, from https://health.ec.europa.eu/medicinal-products/orphan-medicinal-products_en.
11 European Commission, Directorate-General for Research and Innovation, (2022). Collaboration : a key to unlock the challenges of rare diseases research, Publications Office of the European Union. https://data.europa.eu/doi/10.2777/831246
12 Peele, Pamela; Keyser, Donna; Lovelace, John; Moss, Deborah. Advancing Value-Based Population Health Management Through Payer–Provider Partnerships: Improving Outcomes for Children with Complex Conditions. Journal for Healthcare Quality: March/April 2018 – Volume 40 – Issue 2 – p e26-e32. doi: 10.1097/JHQ.0000000000000101
13 IQVIA Report. EFPIA Patient WAIT Indicator 2021 survey. April 2022. Available from: https://www.efpia.eu/ media/636821/efpia-patients-wait-indicator-final.pdf
14 EFPIA, “The Root Cause of Unavailability and Delay to Innovative Medicines: Reducing the Time before Patients Have Access to Innovative Medicines,” April 2022, https://www.efpia.eu/media/636822/root-cause-unavailability-delays-cra-report-april-2022-final.pdf.
15 Facey, K., Rannanheimo, P., Batchelor, L., Borchardt, M., & De Cock, J. (2020). Real-world evidence to support Payer/HTA decisions about highly innovative technologies in the EU—actions for stakeholders. International Journal of Technology Assessment in Health Care,36(4), 459-468. doi:10.1017/S026646232000063X
16 European Health Data Space (no date) Public Health. European Commission. Available at: https://health.ec.europa.eu/ehealth-digital-health-and-care/european-health-data-space_en (Accessed: October 20, 2022).
17 Stamm, T., Bott, N., Thwaites, R., Mosor, E., Andrews, M. R., Borgdorff, J., & Styliadou, M. (2021). Building a value-based care infrastructure in Europe: the health outcomes observatory. NEJM Catalyst Innovations in Care Delivery, 2(3).