- Let the molecule speak – developers taking risks in the efforts of making the biggest difference for patients
The next challenge to solve is the compound under development. Can a molecule – chemical or biological – be designed to interact with the target in the right way? It is important to understand if the molecule has similar effects in humans as seen in preclinical models, which is unknown before starting clinical development, spanning on average over a decade. In neuroscience, less than 10% of the medicines that enter clinical trials end up with a positive benefit-risk profile and are authorized by regulators.1 Clinical development phases are conducted in a sequential manner, with results from each phase informing scientific and financial decisions for progressing to the next. Therefore, predictability of incentives at the time of those decisions is critical to balancing the major financial risk with the opportunity to recover costs and invest in the next new molecule.
- Let the patient speak – integrating patient insights in everything we do
Patients are an integral part of the drug development and innovation ecosystem, owing to their lived experience and the ability to point to the unmet medical needs that matter the most. Therefore, defining unmet medical needs solely based on a quantitative methodology, such as mortality and morbidity reduction, is misleading and arbitrary.
Patients are an integral part of the drug development and innovation ecosystem, owing to their lived experience and the ability to point to the unmet medical needs that matter the most.
At Lundbeck, patient insights are systematically included in development efforts. We work with patient engagement as well as patient experience data as an integral part of our evidence generation. In addition, we involve patients to gain inspiration and obtain advice across the organization, which often leads to an understanding of important issues that are not always clear from existing data or key opinion leaders. Prescribing physicians talk to our medical organization about where they see unmet needs so that their insights and opinions can feed into our innovation activities and development decisions; patients as well as prescribers look to developers to provide the treatments of tomorrow.
The EU incentives for medicines development should avoid unintentionally consolidating research efforts in certain therapy areas
The EU politicians should do everything in their power to avoid further consolidation on specific disease areas. There is already a high risk of organic consolidation because innovation relies on novel scientific findings. For example, after the first CAR-T therapies were approved, there has been a surge of new CAR-T development programs, with more than 900 currently in development.
Choosing a statistical method to define unmet medical needs is likely to work better for some disease areas, such as cancer or cardiovascular, that are easily measured with biomarkers. Promoting consolidation around supposed “high-priority disease areas” would negatively impact the EU’s own competitiveness in a global context – as a region, we have a rich biotech and pharmaceutical ecosystem targeting multiple different disease areas. Rather, the unmet medical need definition should be science-based, include important needs defined by stakeholders, and encourage continued research not only for life-threatening or severely debilitating diseases, but also for chronically debilitating diseases and those that significantly impact people’s quality of life.