Disease burdens, clinical studies, and investments – is there proportionality?

The reasons for the disparity between the extent of clinical drug research and the disease burden likely vary for different diseases. However, there are probably some common factors, including the ability and willingness to pay or lack thereof, and thus the expected return associated with investment. In these cases, it makes sense to establish commercial incentives for research and development in under-prioritized disease areas.

Date: November 2, 2023

Author: Jonas Hink

Published: Dagens Pharma

There is often a debate about whether Pharma’s business model is sustainable, or whether new incentives and payment models are needed to create a more sustainable market in areas where it is not attractive to invest in the development of new treatment options. There are several examples where there is a mismatch between societal needs and industrial interest.

From a societal perspective, ideally, most clinical research should be conducted in diseases that represent the greatest disease burden. Similarly, resources for research into new treatments should be distributed in relation to the underlying causes of the overall disease burden.

When it comes to investment in the development of new drugs, there are many other factors that influence which disease areas are prioritized, but overall, it revolves around risk and return.

 

Requires the ability and willingness to pay

There are many types of risks to consider when investing in the research and development of new molecules and drug candidates. Validated targets, positive pre-clinical results of high relevance, well-defined regulatory processes, established manufacturing methods, predictable side effect profiles, and the feasibility of clinical trials are some of the factors that increase the likelihood of a drug candidate reaching marketing approval.

However, marketing approval is not sufficient for a new drug to become an economic success. It also requires the ability and willingness to pay, even though patents and market exclusivity can protect the drug from competition for a certain period.

According to IHME (The Institute for Health Metrics and Evaluation), one-third of the total disease burden in high-income countries (measured in Disability-Adjusted Life Years or DALYs, in 2019) is caused by cancer and cardiovascular diseases. These two disease categories correspond to about 40% of ongoing industry-sponsored phase 3 clinical trials registered on ClinicalTrials.gov. In contrast, mental and musculoskeletal diseases account for only about 10% of industry-sponsored phase 3 clinical trials, despite these two disease categories constituting nearly 20% of the overall disease burden. Looking more closely at mental and musculoskeletal diseases, it is especially lower back pain and depressive disorders that make up the majority of the disease burden, and there is a mismatch between the extent of clinical drug research and the disease burden.

Common features of underprioritized areas

From a global perspective, there are other disorders and conditions that constitute a significant disease burden, but where only a relatively limited number of clinical drug studies are conducted, for example, in neonatal diseases and diarrheal diseases.

The reasons for the disparity between the extent of clinical drug research and the disease burden likely vary for different diseases. For example, it is possible that there are other and perhaps more relevant types of treatment for lower back pain than drugs, while ethical and regulatory considerations may influence the extent of drug research in neonatal diseases.

However, there are probably some common factors, including the ability and willingness to pay or lack thereof, and thus the expected return associated with investment. In these cases, it makes sense to establish commercial incentives for research and development in under-prioritized disease areas in order to create sustainable business models and/or a more sustainable market for Pharma.