The journey of taking an innovative medicine out of research and into reality takes more than just understanding its science. Drug development is an undeniably complex process involving multiple institutions—non-profits and federal agencies that conduct clinical trials, regulatory authorities that approve new medicines, and distribution networks that ensure they reach patients who need them.  

Nearly 90% of drugs fail to make it this far. This underscores the importance of addressing the practical challenges of applying medical innovation, rather than focusing solely on the discovery of innovative medicines.

In global health, this disconnect between what research has shown to be effective and the reality of healthcare systems is known as the “know-do” gap. Bridging this gap requires consistent institutional support, sound health policies, and sustained investment in biomedical research and healthcare infrastructure. 

This was the theme of the 24th annual Seidman Lecture held at Harvard Medical School on March 12, delivered by Vasant “Vas” Narasimhan, chief executive officer of the multinational pharmaceutical giant Novartis. 

“In many cases, we can develop these medicines but they end up in the warehouse simply because the nuances of the distribution systems have not been solved,” Narasimhan said. 

Since taking the helm in 2018, Narasimhan has led Novartis’ advancements in cell and gene therapy. Drawing from his experiences in public health, and vaccine and drug development, he addressed health policy scholars across Harvard schools, as well as physicians from Harvard-affiliated and other Boston-area hospitals on the policy challenges facing the U.S. biopharma industry, such as short timelines for the renegotiation of drug prices and the rapid expansion of a federal drug affordability program. 

Impacts of health policy on innovation unfold over decades

Narasimhan began the lecture by emphasizing that the impact of health policy changes on biomedical innovation doesn’t happen overnight; it unfolds over decades. 

“That’s one of the hardest things I have to explain in the U.S. Congress,” he said. “When you make a change, you don’t see any impact in two to three years. The real question is what happens over decades in the investment horizon.” 

He illustrated this with the example of China’s biomedical ecosystem. When Novartis first established its office in Shanghai in 2011, you could see nothing but cleared farmland for miles, Narasimhan recalled. However, over the past decade, China has transformed itself into a biopharma leader, thanks to a series of policy reforms that have streamlined the country’s drug review, approval, registration, and market access processes. 

Among the key reforms Narasimhan highlighted is the creation of a public website that tracks all regulatory filings, ensuring a transparent drug approval process. The country has also introduced strict timelines for regulatory decisions, and expanded patient access to innovative drugs with the National Drug Reimbursement List, which makes a drug eligible for government-funded reimbursement across all provinces. 

As a result, China been able to attract significant foreign investment into its biopharma sector, making its drug market the fastest-growing market in the world. What was once cleared farmland is now a biotech hub that could soon rival the U.S. biomedical ecosystem. 

“The U.S. is retracting from the NIH and you see China pouring in. Even from a geopolitical standpoint, it’s the wrong move because there’s no way you’re going to slow down China by limiting data flow,” Narasimhan said. “The only way is to compete based on science, and by investing in science so that our scientists can stay up and hopefully out compete the science that’s happening there in Shanghai.”

What advancements in one-time therapies mean for U.S. health policy 

With rapid advancements in cell and gene therapies, the U.S. health policy is at an inflection point. Narasimhan stressed that while these cutting-edge treatments, often called one-time therapies, are costly, they hold the potential to cure rare diseases. This, over the long-run, eliminates long-term healthcare costs associated with the ongoing care and treatment of patients with rare conditions. 

One such drug is Zolgensma, a Novartis gene therapy for children under two diagnosed with spinal muscular atrophy, a fatal neuromuscular disorder. It’s currently priced at $2.1 million per dose. 

“But in the end, what was really interesting to see is [how] step by step every state Medicaid program—when they understood the economics—ultimately took it on,” Narasimhan said. 

But Zolgensma also highlights a dilemma in U.S. health policy: how to finance highly effective but costly one-time treatments?

The U.S. healthcare system is structured around drugs that are taken over months and years, allowing their cost to be distributed over time. But one-time therapies create large upfront expenses that strain healthcare budgets. As more of these treatments enter the market, health policy experts must find sustainable solutions that ensure access of these effective therapies to patients. 

“The answer can’t be that most of these medicines are being developed for young children, and the answer can’t be that we don’t get the children these therapies,” he said. 

 

The Annual Marshall J. Seidman Lecture Series was established in 2001 through an endowment to Harvard Medical School Department of Health Care Policy by Marshall J. Seidman, with the aim of fostering university-wide discussions on improving health policy at the state and federal levels.

###