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When considering factors like disease biology and patient needs in drug discovery and development, the goal is to deliver effective and scalable treatments globally by balancing established approaches like small molecules or protein therapeutics with more complex modalities such as RNA, cell, and gene therapies.
The pharmaceutical community is relentlessly racing to accelerate the discovery, development, and delivery of novel therapeutics against the most difficult-to-treat diseases. We are in the midst of both a tremendous evolution in understanding the biological basis of disease and a new era in the science of therapeutics to turn this knowledge into transformative treatments.
A staggering amount of new modalities have emerged alongside traditional ones, creating an unprecedented inflection point for the industry. To determine how to best compete, leaders in the drug discovery and development ecosystem must be deliberate in their strategic selection of modalities to address complex disease biology and meet patient needs.
The stakes for people with medical needs and the degree of uncertainty in drug discovery are incredibly high, so how does a company set itself up for success in R&D and manufacturing?
As scientists invent therapeutic breakthroughs across biological pathways of disease, the choice of modality (small molecule, protein, cell and gene therapies, RNA and others) is critical. The goals are to deliver a therapeutic with the best possible patient outcome and experience, and to accelerate access for all through scalable, cost-effective manufacturing solutions.
When thinking about modality selection in drug discovery and development, the first steps are assessing and understanding the disease biology we want to modulate, the current available therapeutics and the optimal target product profile to maximize patient impact as early as possible. These factors all inform modality selection, and I believe selection of the right modality at the beginning of a new project is key to the ultimate success of a drug. There is no “one-size-fits-all” approach, but rather several factors that need to be balanced.
Ultimately, we are looking to deliver the most effective, practical, and simplest therapeutic options for patients, something that can be delivered at a global scale. Often, this means using well established approaches — small molecules or protein therapeutics — that generally work by modulating protein function directly at the protein level to restore normal cell functions and fight disease.
In some cases, we can’t sucessfully intervene at the protein level, and that’s when we can turn to the more complex modalities, such as RNA, cell, and gene therapies. These often present more development and commercialization challenges but bring unique biological or clinical benefits, often for patients in some of the hardest-to-treat disease areas.
At times, the first iteration of an approved therapeutic, often via the modality that delivers the desired biological outcome to patients, may not have the most optimal profile or be the most convenient. As an industry, it is important we embrace technological advances and keep moving forward. As the understanding of biology and patient needs grow, therapeutic options should be optimized — for example, by replacing therapies administered via intravenous injection with less invasive options.
Of course, charting the next frontier of drug discovery and development is no small feat. As members of the pharmaceutical community, we should be relentlessly seeking opportunities to target new potential disease areas and improve current therapies. That means we cannot always do it alone. It requires a collaborative position and a humble mindset within the global innovation ecosystem to succeed.
The largest opportunities to access innovation and benefit patients often are enabled by early-stage partnerships that combine big pharma engines with external innovators to create unique, high-value products. It is our aspirationto be the best at translating therapeutic concepts, regardless of source, stage or modality, into differentiated products and deliver commercial manufacturing processes, optimized for cost and ease of supply, to maximize the business and patient access.
One of the most essential elements in enabling this — and one of the aspects I love the most about my job — is to adopt what I call a “source-agnostic” approach to product invention. We combine the strength of our internal capabilities with the world’s top minds, from academic institutions to small and large biopharma companies, seeking the best partners and working together toward success.
Our goals are to address disease most effectively and at scale, constantly improving therapies. This ultimately requires us to leverage the complete modality toolset as we iterate from first-in-class interventions to best-in-class, transformative treatments for patients with broadest possible global access.
Richard Tillyer, PhD, Global Head, Discovery, Product Development & Supply, Johnson & Johnson Innovative Medicine