We are living 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 allowed the development of the COVID vaccine in record time. Yet we are facing significant challenges as well.
There is a global call for more equitable pricing and broader access—especially for the newer innovative treatments. How can we as healthcare leaders answer that 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—will benefit?
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Collaboration and partnerships
In answering the call, we must focus our attention on where it has the most impact, which in this case is long before commercialization and long before we even have a viable drug candidate.
We are experiencing an important paradigm shift: It’s not so much about ensuring that drugs from any one company will get to as many patients as possible, but instead, it’s about working together to create a viable system that supports innovation and patients and does so cost-effectively. That’s the goal.
Pricing is only the beginning
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Prioritizing the conversation around drug pricing is a given. In the current paradigm, drug pricing doesn’t 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 a great deal of investment. This investment into each molecule needs to be recouped in the marketplace in order for drug development to be a sustainable activity. This is the primary challenge around pricing.
There is a better solution.
Yes, there are issues at play that speak to larger economic forces, which include the fact that the U.S., essentially, subsidizes the cost of drug development for the rest of the world. For instance, in Europe, they operate under a one-payer system that pays a certain amount; in the U.S., 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 U.S. However, that is not the only factor at play—not by a long shot.
Navigating investment risk
The landscape of drug development and innovation is fraught with risk. A company aspires to have a number of drugs in their pipeline, but to do that while mitigating the risk involved, they have to invest in far 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 have it fail, thereby flushing a billion dollars down the toilet. Yet another one of their drugs ends up being a life-saving treatment, and the profits mitigate (hopefully) any other losses and are put back into further innovation.
Clearly, the model of investing huge amounts of money into one drug to try to recoup it later is problematic at best and unsustainable at worst. There is a more viable solution from a drug development perspective. It lies in creating a model that would cut 90% of the cost out of every molecule and increase the probability of success tenfold. So, what is that model?
New model: Platform technology
This is a model that already exists and its power lies in platform technologies. The bulk of investment goes into perfecting the technology such that each compound that it delivers is not a new and independent effort, but it’s largely characterized. Thus, the cost of subsequent programs is relatively small because the company deeply understands the characteristics of each molecule coming down the pike (i.e., pharmacology, tolerability) so that it is scalable and affordable.
Nucleic acids as target molecules have an advantage in getting us to this affordable future. By using nature’s own digital information encoding scheme, we are able to design medicines that are programmed to precisely engage only the target of interest before we make the compounds. This also allows the designer to avoid engaging with other sequences. That reduces the possibility of side effects and further decreases the cost of finding a development candidate—and increases the probability of success.
We see this with some of the early genetic medicines such as mRNA therapy and gene editing, where scalable platforms are being used (with even more advantages such as using the same delivery nanoparticle and the same chemistry for any new compound), and it’s just about shuffling the nucleobases to target different disease-causing genes (the As, Cs, Gs and Ts) to create the therapy of interest.
Early investment is essential
In the face of innovative yet increasingly expensive drugs that payers are ultimately going to balk at, having a platform that can guarantee scalable genetic medicines is not only a sustainable business model, it is the solution patients need. Add to that a growing landscape of collaboration and partnership to confront the thousands of common and rare diseases that are beyond any one company’s reach, 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’s worth investing in sooner rather than later so that patients can affordably reap the benefits.
Credit: appledesign, Getty Images
Dr. Dietrich A. Stephan is the CEO, Chairman, and Founder of NeuBase Therapeutics, which is developing a new class of precision genetic medicines to address rare and common diseases at the base level. At NeuBase, he brings his deep expertise in genetic diseases, personalized medicine, and first-in-class therapeutic development to redefine medicine for millions of patients with limited treatment options. Dr. Stephan identified the molecular basis of dozens of rare and common diseases published extensively in journals and has helped launch 14 biotechnology companies to advance novel molecular diagnostics and first-in-class therapeutics to market. These companies include Navigenics, Pendulum, and Peptilogics. Dietrich remains inspired by the army of scientists and biotechnologists who battle daily for the lives of those suffering and dying needlessly from disease.
