The New Paradigm in Drug Development: Why Platform Technology is the Solution Patients and Payers Need

We live in a dynamic time of drug development and innovation with many beneficiaries, the most important being the patient. These developments include everything from exciting gene therapies for rare diseases to mRNA technology that enabled COVID vaccine development in record time. Yet we also face significant challenges.

There is a global call for fairer pricing and wider access, especially for innovative new treatments. How can we, as health leaders, respond to this call? How can we work with our stakeholders to ensure that the system can effectively support new innovations so that the most important stakeholder, the patient, benefits?

Collaborations and partnerships

In answering the call, we need to focus our attention where it has the greatest impact, which is long before commercialization and long before we even have a viable drug candidate.

We are experiencing a significant paradigm shift: it is not so much about ensuring that one company’s medicines reach as many patients as possible, but rather about working together to create a viable system that supports innovation. and patients and which is expensive – indeed. That’s the point.

The price is just the beginning

Prioritizing the drug price conversation is a no-brainer. In the current paradigm, drug pricing does not enter the conversation until development enters the clinical phase. However, the bigger issue is how drugs are developed.

Most often, each new drug has its own development process, which requires significant investment. This investment in each molecule must be recouped in the market for drug development to be a sustainable activity. This is the main challenge around pricing.

There is a better solution.

Yes, there are issues at play that relate to larger economic forces, including the fact that the United States, essentially, subsidizes the cost of drug development for the rest of the world. For example, in Europe they operate on a single-payer system that pays a certain amount; in the United States, there is a multi-payer system that pays another. The argument could be made that the entire global payer system would have to change for reduced prices to be seen here in the United States. However, that’s not the only factor at play – not by a long shot.

Manage investment risk

The landscape of drug development and innovation is fraught with challenges. A company aspires to have a certain number of drugs in its pipeline, but to do so while mitigating the risks involved, it must invest in many more candidates in the research and development phases given the traditionally low probabilities of success, which equates to billions of dollars of capital.

A company can easily spend 15 years developing a drug only to see it fail, flushing a billion dollars down the toilet. Yet another one of their drugs ends up being a life-saving treatment, and the profits (hopefully) offset all other losses and are reinvested in new innovations.

Clearly, the model of investing huge sums of money in a drug only to try to get it back later is problematic at best and unsustainable at worst. There is a more viable solution from a drug development perspective. The aim is to create a model that would reduce the cost of each molecule by 90% and increase the probability of success tenfold. So what is this model?

New Model: Platform Technology

It’s a model that already exists and its power lies in platform technologies. Most of the investment goes into refining the technology, so that each compound it delivers is not a new, independent effort, but is extensively characterized. Thus, the cost of subsequent programs is relatively low because the company deeply understands the characteristics of each arriving molecule (i.e. pharmacology, tolerability) so that it is scalable and affordable.

Nucleic acids as target molecules have an advantage in moving us towards this affordable future. Using nature’s own digital information coding scheme, we are able to design drugs programmed to precisely engage only the target of interest before manufacturing the compounds. It also allows the designer to avoid committing to other sequences. This reduces the possibility of side effects and further decreases the cost of finding a candidate for development, and increases the likelihood of success.

We see this with some of the earliest genetic drugs such as mRNA therapy and gene editing, where scalable platforms are used (with even more advantages such as using the same delivery nanoparticle and the same chemistry for any new compound), and that’s pretty much mixing nucleobases to target different pathogenic genes (As, Cs, Gs, and Ts) to create the therapy of interest.

Early investment is essential

In the face of innovative but increasingly expensive drugs that payers will ultimately balk at, having a platform that can secure scalable genetic drugs is not just a sustainable business model, it’s the solution patients need. . Add to that a growing landscape of collaboration and partnership to tackle the thousands of common and rare diseases that are beyond the reach of a single company, and embracing partnerships that can help a company in specific disease areas is the wave of the future. This is the new paradigm, and it is worth investing in it as soon as possible so that patients can benefit from it at a lower cost.

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