Establishing criteria for unmet medical needs: let’s not leave people living with chronic diseases behind

In the world of medicine development, innovation is closely associated with unmet needs. When we as a pharmaceutical company advance innovative science into development, we take a holistic perspective on the unmet need – for instance, new biology, patient and caregiver preferences, a gap in current treatment options, better safety profile and value to society.

One of the requests from some EU member states in the ongoing revision of the General Pharmaceutical Legislation is to establish clear criteria for unmet medical needs. While it may not seem an unreasonable request, the practical task is nearly impossible: establishing criteria that will give an equal, non-biased consideration for all disease areas. Any arbitrary prioritization between diseases inevitably means that some patients – and likely some therapy areas – will be neglected.

One therapy area that may risk seeing a decline in the future development of new treatments is neuroscience. It is an area with life threatening, seriously debilitating, and chronic diseases of the brain and the nervous system that have a significant impact on public health and society. Despite the majority of political parties in the European Parliament and the European Commission’s President, Ursula von der Leyen, identifying mental health and degenerative diseases as a priority for this coming mandate, the proposed unmet medical need definition may disincentivize the continued progress to innovate in this therapeutic area. This seems counterproductive and illogical.

Unmet medical needs at the core of the decision-making for the development of new therapies

Targeting the development of treatments to address an unmet medical need is imperative for everyone – patients, caregivers, society and developers alike. No research or development activity begins without identifying whether there is a need. For instance, some patients have conditions for which no treatments are available, such as multiple system atrophy, which is a rare neurological disease. Meanwhile, other patients may demonstrate zero or only a partial response to existing treatments. There are also people living with Parkinson’s disease, whose quality of life decreases with lower physical strength for basic daily tasks. Migraine patients who may not be able to support themselves and their families due to the impact on their ability to work or frequent sick days. Societies that provide expensive in-patient care instead of facilitating patients with psychiatric conditions to live in their own home with adequate treatments. All these situations exhibit an unmet medical need.

With the narrow definition of unmet medical needs being considered, there is a risk that the development of new medicines for populations that do not respond to first-line treatment is disincentivized. Instead, continued efforts are needed from all stakeholders – political, society and industry – to ensure an inclusive framework that does not disfavor any diseases by design and genuinely fosters the development of new innovative treatments.

Translating science into transformative treatment outcomes

The Lundbeck R&D approach is built upon three pillars: a deep understanding of the biology, the science and the patients who fall into the areas where we believe our drug development innovations could impact their unmet needs.

• Let the biology speak – the importance of strong scientific basis cannot be emphasized enough: focusing on areas with untapped potential in neuroscience

Incentives for conducting and investing in R&D in the EU could factor in as one element of development decisions, but the scientific rationale remains key. The biological rationale centers around whether there is something in the human body (‘a target’) that can be affected to create a positive outcome; these could, for example, be receptors in the brain. Understanding these targets requires profound research activities and is critical in order to develop new medicines. Without a strong scientific knowledge base for the disease and its causes, undertaking innovation activities is challenging.

• 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.

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.

It is important that the future legislative framework serves innovator companies of all sizes, including academic spin-offs, start-ups, biotechs and small and medium-size enterprises, to foster future innovation and ensure the development of new and innovative treatments to address the unmet medical needs of patients.

1. The Significance of Meaning: Why Do Over 90% of Behavioral Neuroscience Results Fail to Translate to Humans, and What Can We Do to Fix It? – PMC (nih.gov)

Bio:

Lundbeck is a global mid-sized EU-headquartered innovative pharmaceutical company focusing on neuroscience, covering the full value chain from molecule to patient. Its 5,600 employees serve 8 million people every day.

Executive vice president Johan Luthman leads the Lundbeck R&D organization and holds more than 30 years of experience within the pharma industry. This experience includes executive positions in large pharmaceutical companies both headquartered in the EU and other parts of the world and as CEO for a small biotech start-up as well. He has a strong understanding of R&D policies that truly incentivizes medicine development decisions.