4 opportunities for patient-centricity in drug development

1. Challenge: where is the patient in early-stage drug development?

The existing model on which drugs are developed consists of several components. First laboratory and animal research. After that, in Phase 1, safety and dosage research, involving healthy participants (or patients for biologics drugs). Only after years of research, in Phase 2, the patient is finally involved in the research process. But we don’t develop drugs for animals or healthy volunteers – we develop them for patients expressing the intended target. How can we involve them from the start?

Opportunity: bring the patient into the picture earlier

“Phase 0 microdosing studies, as implemented by TRACER, bring the patient into the research years earlier with a very low, non-therapeutic dose. This allows drug developers to obtain insights in real-life situations. Multiple compounds can be investigated quickly and efficiently in patients with the disease, even with different variants of a disease. For example, by including patients with multiple solid tumor types, a drug developer can increase knowledge about the disease mechanisms and investigate the new drug’s mechanism of action against selective diseases in different patients.”

– Ari Aminetzah

2. Challenge: how to reach patients across the globe?

To reach every patient globally, a new drug should ideally be available in every country. This involves market approval in different regions and markets, which may lead to different challenges in the development process. In global drug development, chances are that there is not one standard of care to compare a new drug against in Phase 3. In each country, different drugs may be offered as standard treatment for the same disease.

Opportunity: choose the right location(s) for your Phase 3 research

“The most important milestone in drug development is market authorisation and, therefore, the completion of a Phase 3 clinical program. Phase 3 is all about choosing the Standard Of Care (SOC) with which to compare the study drug and scaling it to a large patient population. The choice of SOC in Phase 3 depends on the country where the research is being conducted. To market your drug in another country where a different standard of care applies, additional research (bridging trials) may be required. Because this involves substantial additional costs, two things may happen. First, a drug developer looks at where they want to do the research, this depends on the market potential. And second, it may happen that a drug will become available later or even never in some countries.”

– Connie van Marrewijk

3. Challenge: how to bridge the gap between research and practice?

Clinical research takes place in a controlled and clean setting. This can contrast with the final use where factors like comorbidity and local practice may influence the actual outcome for a specific patient. This makes it possible that a drug in a real-life situation does not exhibit what it showed in clinical trials.

Opportunity: generate real-world data

“Another opportunity for patient-centricity may be to collect real-world data earlier in the drug development process. Examples are the U.S. Accelerated Approval Program and the EMA Conditional Market Authorisation. Both can be based on a surrogate endpoint and allow earlier approval of medicines that treat serious conditions and fill an unmet medical need. Of course, safety and effectiveness are proven first, but confirmatory research takes place while patients can already receive the drug. This system, now selectively available only for certain types of medicines, fits well with a growing desire of the pharmaceutical industry. Namely, earlier market access and gradually gathering real-world evidence.”

– Connie van Marrewijk

4. Challenge: how can we reduce the time to market?

Time to market is most important for pharmaceutical companies, after all, the faster a drug reaches the market, the sooner a return on investment can be made. This is also in the interest of the patient because of faster availability. A shorter research pathway means lower costs across the board and a faster time to market. This may result in a positive effect on affordability and accessibility. So, a fast time to market is a win-win for both the patient and the pharmaceutical company. To bring a new drug to market successfully – and thus reach patients – the market needs to be prepared for the arrival of the new drug. A process that already starts during development.

Opportunity: involve healthcare institutions early on

“Is the pharmaceutical organisation and the healthcare system ready when the new drug will enter the market? What is required to reach the patient when the drug becomes available? Setting this up can be challenging, especially if the pharmaceutical company does not yet have existing connections and infrastructure. Bringing a drug to market seems like a linear process, but the practice is highly complex and requires many close collaborations. Preparations already start during research by looking at the patient pathway. By involving hospitals and physicians in clinical research, you learn about local practice and how this may influence the drug’s effectiveness. When Scientific Experts (SEs) are involved in research, they will gain experience by using the treatment. This may have a beneficial effect on the market adoption and integration into treatment guidelines.”

– Patrick Filius