We live in a dynamic era of drug development and innovation with many beneficiaries, most importantly patients. These developments range from exciting gene therapies for rare diseases to mRNA technologies that have enabled the development of a COVID vaccine in record time. However, we also face significant challenges.
There is a global call for fairer pricing and broader access — especially for newer, innovative treatments. As healthcare leaders, how can we respond to this call? How can we work with stakeholders to ensure that systems can effectively support new innovations that benefit the most important stakeholders—patients?
Cooperation and Partnership
In answering the call, we have to focus on where the impact will be greatest, in this case long before commercialization, even before we even have a viable drug candidate.
We’re going through an important paradigm shift: not so much about making sure any one company’s medicines reach as many patients as possible, but about working together to create a working system that supports innovation and patients, and in doing so reduces costs -Effectively. That’s the goal.
Pricing is just the beginning
Prioritizing conversations around drug pricing is a matter of course. In the current paradigm, drug pricing does not enter the conversation until development reaches the clinical stage. The bigger question, however, is how the drugs are developed.
In most cases, each new drug has its own development process, which requires significant investment. For drug development to be a sustainable activity, the investment in each molecule needs to be recouped in the market. This is the main challenge around pricing.
There is a better solution.
Yes, some of the issues have to do with larger economic forces, including the U.S. basically subsidizing the cost of drug development in the rest of the world. For example, in Europe, they operate under a single-payer system that pays a certain amount; in the United States, there is a multi-payer system that pays another person. Suffice it to say, the entire global payment system would have to change to see price cuts in the US. However, that’s not the only factor at play—nor in the long run.
Manage investment risk
The prospect of drug development and innovation is fraught with risk. A company wants to have many drugs in their pipeline, but to do this while reducing the risks involved, they have to invest in more drug candidates in the R&D stage because the probability of success is traditionally low – which is quite in billions of dollars. dollar capital.
It’s easy for a company to spend 15 years developing a drug only to fail, washing out $1 billion. Yet another of their drugs ended up being a life-saving treatment, with profits mitigating (hopefully) any other losses and reinvesting in further innovation.
Clearly, the model of throwing huge sums of money into a drug to try to recover the cost later is problematic at best and unsustainable at worst. From a drug development perspective, there is a more feasible solution. It lies in creating a model that can reduce the cost per molecule by 90% and increase the probability of success tenfold. So, what is that model?
New Model: Platform Technology
It’s a model that already exists, and its power lies in platform technology. Much of the investment has gone into perfecting the technology so that each compound it provides is not a new independent endeavor, but is largely characterized. Therefore, the follow-up program is relatively inexpensive because the company has a deep understanding of the properties of each molecule (ie, pharmacology, tolerability), so it is scalable and affordable.
Nucleic acids as target molecules have the advantage of getting us into this affordable future. By using nature’s own scheme of encoding digital information, we are able to design drugs that are programmed to precisely target only the targets of interest before we make compounds. This also allows the designer to avoid contact with other sequences. This reduces the likelihood of side effects and further reduces the cost of finding development candidates — and increases the likelihood of success.
We’re seeing this with some early genetic medicines, such as mRNA therapy and gene editing, which are using scalable platforms (with more advantages, such as using the same delivery nanoparticle and the same chemical composition for any new compound) ), which simply shuffles nucleobases to target different disease-causing genes (As, Cs, Gs, and Ts) to create therapeutics of interest.
Early investment is critical
In the face of innovative but increasingly expensive drugs that payers will eventually hesitate, having a platform that promises scalable genetic medicine is not only a sustainable business model, but a solution that patients need. Coupled with growing collaborations and partnerships to address thousands of common and rare diseases beyond the reach of any one company, partnerships that can help companies in specific disease areas are the wave of the future. This is a new paradigm that deserves an early investment so patients can reap the benefits in an affordable way.
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