At Roche we discover and develop medicines and diagnostics to improve and save the lives of people around the world. We develop medicines driven first and foremost by unmet medical need with the patient at the centre of everything we do.

From this starting point, we proceed based on solid science e.g. a biological hypothesis, a deep understanding of the disease biology, or the ability to modulate a disease-causing target, etc.

There then needs to be the possibility to develop a medicine that can be understood in terms of what the human body does to the administered medicine (i.e. pharmacokinetics) and conversely what does the medicine do to the body (i.e. pharmacodynamics).  We also need a means of medicine delivery that is convenient to the patient. And that the medicine can also be produced at scale. 

Next we must look at the medicine’s suitability for development, for example is there an identifiable patient population (diagnosed), or the ability to de-risk the project by testing either the intended primary endpoint or a surrogate marker before embarking on a major pivotal trial investment. Can we recruit patients to a clinical trial, and achieve endpoints that are acceptable to health authorities in assessing benefit risk, and to payers in assessing the value of the therapy, within a reasonable time period?

At the same time, we need a thorough understanding of the competitive environment and any alternative treatment options. And whether or not our medicine could be ideally first or best in disease.  Finally, there needs to be a reasonable likelihood of a return on our investment to allow funding of a sustainable research and development engine.

Now let’s dig deeper on unmet medical need. This is a broad term with many facets, such as the opportunity to, for example:

•  alleviate or eliminate symptoms e.g. pain or bleeding in inflammatory bowel disease
• provide a less burdensome treatment e.g. fewer or less severe side effects and better tolerability or improved ease of administration, e.g. oral vs infusion, less frequent or home vs hospital-based
• modify the course of the disease and slow, stop or revert its progression by addressing its root cause e.g. for haemophilia
• prevent relapses e.g. in oncology
• cure diseases e.g. infections
• prevent the development of disease e.g. vaccinations
• restore function e.g. re-establish vision in blind or vision impaired people

The European Commission’s legal proposal on the revised General Pharmaceutical Legislation is currently moving forward with defining “unmet medical need” for the purpose of prioritising regulatory pathways, drug development incentives, and access. As the discourse advances, it is critical not to lose sight of the fact that:

1. It is complex if not impossible to distinguish and prioritise between unmet medical need that is deserving vs undeserving. On this, patients with different diseases and different healthcare stakeholders will have diverse perspectives.
2. Human biology is complex and frequently not yet fully understood, and so, during research and early development profound changes may occur ranging from changing the target disease, to expanding to further diseases, to stopping the project altogether. Scientific discovery is an iterative process.
3. Further approvals of new medicines in a given indication increase competition within the class and importantly provide additional treatment options, e.g. meeting the needs of patient subpopulations not addressed by the first in disease. Such investments need also to make a return. Examples are:

• GLP-1 Receptor agonists where further generation of molecules may yield more benefit, fewer side effects, or a more convenient administration.
• In Spinal Muscular Atrophy (SMA) where there are now three distinct mechanisms and modalities available to patients, giving options.  
• R&D in Alzheimer Disease is a high-risk investment and it is expected that different modes of action, and potentially combination therapies, will eventually slow or halt the debilitating cognitive decline. It is unlikely that the first disease modifying medicine to be approved will meet the needs of all patients or, ultimately, emerge as best in class.

Multiple dimensions of unmet medical need are deserving, are meaningful for individual patients, and frequently also for health systems and society as well. This applies not just for life threatening or severely disabling diseases. Pharmaceutical R&D takes many years if not decades and materially significant, at risk, investments.  We therefore celebrate authorised new options for patients with any conditions not currently adequately diagnosed, prevented, or treated.

For our company’s R&D investment decisions, it needs that there be a broad, encompassing definition of Unmet Medical Need, starting with the patient perspective, and that it includes the perspectives of further critical stakeholders: innovators, regulators, physicians, and payers. We envision a definition which is not discriminating or favouring patients suffering from some diseases over others.