Innovative biopharma has the aspiration to bring treatments new to the world to market in order to improve patient health and well-being. Underlying challenges and risks are enormous, while success significantly contributes to public health, for example, the introduction of a powerful vaccination for pandemic infectious disease or new approaches to treat life threatening rare disease.

In biopharma R&D, it is not uncommon for development programs to look promising in the early stages, only to discover further down the line that the design is sub-optimal to generate the required evidence package for innovative treatments, leading to a need for major developmental amendments. By the time this is realized, considerable investments of time and money have already been made. Drug development is one way to fundamentally improve patients’ lives, but it is resource-intensive, time-consuming, inefficient and risky.

We are faced with clinical development programs that are getting longer and increasingly complex, and yet the probability of success is not improving at the same pace. The rate of success of typical candidate drugs making it from the research stage to availability at the pharmacy is less than 1%. This set-up does not fit well with the industry’s drive to deliver more affordable and impactful medicines. To increase the challenge further, market authorization these days does not automatically result in commercial success. Market access is growing as the second big hurdle for innovative biopharma, and can be considered to be the more significant hurdle in comparison to getting marketing authorization.

The issue is that the same drug development paradigm invented and used in the 1960s is still being applied in most biopharma companies today. Multiple stakeholders, including regulators and payers, agree that this current drug development paradigm is problematic and that the industry will benefit tremendously if the current clinical program design and protocol execution can evolve and be optimized. In order make this shift, we need to combine the early enrichment of clinical efficacy evidence, clinical effectiveness evidence and more robust safety monitoring.

If only we had the tools…

In today’s technologically advanced world we have the tools - as well as the data - to optimize the drug development process. Big data provides access to an unimaginable breadth of information. Predictive models can now be used to increasingly analyze any available pre-clinical, clinical, real-world data to reduce possible risks. There is also increasing interest and a growing number of capabilities in using relevant data that is observational in nature, such as real-world data and pharmaco-epidemiological data.

There are various methods, languages and purposes associated with each type of data-related technology, which makes it complicated to navigate. Nonetheless, the drug development paradigm is ripe for reform and stands to benefit from a more integrated and advanced approach that mimics as closely as possible the benefit-risk profile of a new treatment in the real-world setting. Fortunately, we stand at a tipping point where we can choose either to revolutionize how R&D is being done or leave it as it is.

What the future of R&D looks like

In the future, developing new and innovative drugs should be more rewarding – for biopharma, regulators, payers, and patients. Drug development will be more calibrated towards meaningful benefits for patients.

In this vision, successful pharmaceutical companies will be those that early on combine randomized controlled trials with emerging opportunities from using retrospective real world data like codified in EHR and generate real world evidence on outcomes during clinical development.

For now, the full value of this vision remains intangible, but it is not impossible to achieve. But how does the industry move forward to ensure it makes the best out of its tipping point position? Our white paper, Breaking the Constraints of the Current Development Paradigm, investigates this very question.