Maintaining America’s dominance in medical research starts with smart policy

What defines America’s leadership in biopharmaceutical innovation today?

SJU: America leads the world in biopharmaceutical innovation because we’ve built an ecosystem that turns cutting-edge science into real treatments and vaccines for patients.

That vision has been deliberate from the beginning. Our founders understood that securing the fruits of invention was essential to a thriving nation. It wasn’t an afterthought; it was written directly into our Constitution, empowering Congress to promote science by securing inventors’ exclusive rights to their discoveries for a limited time in service of the public good.

Over the last 40 years, the U.S. has continued to make deliberate policy choices to ensure our ecosystem supports innovation. Landmark, bipartisan laws like the Bayh‑Dole Act, which enabled publicly funded discoveries to be commercialized; the Hatch‑Waxman Act, which created the balance between innovative medicines and generic competition; and the Prescription Drug User Fee Act (PDUFA), which gave the FDA the resources to establish the global gold standard for human drug review, helped build an environment where scientific breakthroughs can move rapidly from the lab to patients.

Because of these decisions, the U.S. remains the best place in the world to research, develop and manufacture lifesaving medicines. American patients benefit most: they gain access to 87 percent of new medicines, often within just a few months, while patients in other wealthy countries wait an average of three years, if they receive them at all. And this ecosystem does more than improve health: It supports millions of high-wage jobs, strengthens our national and economic security by reducing dependence on foreign supply chains and ensures the next generation of cures is discovered here at home.

America leads because we chose to lead. Protecting and strengthening these policies is essential to remaining the global engine of biomedical innovation.

What are the core strengths of the U.S. innovation ecosystem?

SJU: The U.S. has spent decades building an ecosystem that fosters collaboration and innovation. Our research institutions attract the best talent and drive discoveries that fuel the innovation pipeline — anchored by globally recognized research hubs such as Cambridge, Massachusetts and North Carolina’s Research Triangle Park, where academia, startups and established companies work side by side. That scientific strength is paired with public policy that encourages bold, high-risk private-sector investment: strong intellectual property protections and a predictable, science-based regulatory system that gives researchers and investors the confidence to pursue breakthroughs that take years to bring to life.

This system also strengthens our economy, our health security and the lives of millions of patients. It’s a system that other countries increasingly seek to replicate, but one that remains possible only because of the deliberate policy choices we’ve made to champion science, reward innovation and maintain an environment where the next generation of medical advances can be developed right here at home.

How do you view the competitive landscape today?

SJU: U.S. leadership in biopharmaceutical innovation isn’t something we can take for granted. China is executing a strategic plan to dominate the life sciences. They already conduct a third of the world’s clinical trials — up from just 5 percent a decade ago — and are on track to surpass the United States by the end of the decade. Furthermore, Chinese institutions have rapidly risen in global research output rankings — now occupying seven of the top 10 positions — outperforming leading U.S. research universities, including Harvard.

At the same time, some U.S. policy proposals risk weakening the very ecosystem that made us the global leader. We’re at a real inflection point. Instead of slowing China’s progress — or limiting productive global scientific collaboration — we should strengthen the environment for innovation. That means protecting the policies that have long supported breakthrough science and avoiding approaches that would pull back investment in the next generation of discoveries.

The old narrative of China stealing IP and developing “me too” medicines has given way to a new reality where China is now a leader in innovation. If we don’t double down on what works here in the U.S., we risk losing ground in the race to develop the treatments and cures that patients are counting on.

How is the biopharmaceutical industry working to ensure Americans aren’t dependent on foreign sources for medicines? 

SJU: Our industry is making major long-term investments to ensure Americans can rely on a resilient domestic supply of innovative medicines.

Our companies have announced more than $500 billion in new U.S. manufacturing and infrastructure investments over the next decade, expanding production capacity, modernizing facilities and strengthening the supply chain across the country. That commitment is already taking shape through new groundbreakings and large-scale manufacturing expansions in places like Virginia, North Carolina and California. These commitments support over 5 million high-wage American jobs, anchoring an industry that fuels economic growth in every state.

They also build on an already significant presence: The U.S. has more than 1,500 biopharmaceutical manufacturing facilities across 48 states, and that footprint has grown by more than 50 percent since 2018 — giving the U.S. one of the most robust, high-quality production networks in the world.

These commitments improve reliability and keep high-value jobs in American communities, all while ensuring patients can access the medicines they need when they need them.

How do policy decisions and global shifts affect American patients?

SJU: Policy decisions and rising global competition have real consequences for American patients. Government drug price setting policies, for example, weaken the incentives needed for long‑term, high‑risk investment in new medicines. When those incentives erode, companies pull back, and fewer breakthroughs reach patients.

At the same time, price controls are being sold as a quick fix for affordability and access to medicines, but evidence suggests the opposite occurs. Patients rarely see the benefits of these policies because they do nothing to rein in insurers and PBMs that decide what medicines are covered, what patients pay, and what hoops patients must jump through to access their doctor-prescribed medicines. This dynamic sets up a vicious cycle: policymakers cut prices to give relief to voters, but the savings go to middlemen instead of patients and pressure builds for harsher controls that don’t fix the actual problem.

Meanwhile, patients experience the downside of price controls. Take a patient like Linda Pfeifer, who manages type 2 diabetes and vision loss from diabetic retinopathy. The treatments Linda relies on today only exist because companies had incentives to invest in breakthrough research. Linda worries that drug price setting policies will make it harder for companies to justify similar investments in the future. For patients like Linda, biopharmaceutical innovation isn’t theoretical; it’s the real possibility of finding new, potentially curative, treatments for her chronic disease.

Global shifts matter just as much as policy decisions. If biopharmaceutical leadership shifts to another country, future breakthroughs will be discovered, developed and manufactured elsewhere, affecting everything from approval timelines to medicine availability and even raising national security risks if the U.S. becomes dependent on geopolitical competitors for breakthrough medicines.

Policy choices determine whether American patients remain first in line for new cures, or whether those cures are delayed or even reach American patients at all.

Which policy decisions matter most to sustaining innovation and lowering costs for patients?

SJU: There are three things we need to do to sustain innovation while lowering costs for patients.

First, we must strengthen the innovation ecosystem. This means fixing the Inflation Reduction Act’s (IRA) pill penalty, rejecting price-setting proposals like Most Favored Nation (MFN), and supporting strong intellectual property (IP) protections. These ensure that companies can keep investing in high-risk science that leads to the development of new cures and treatments.

Second, we must focus on the real drivers of high drug prices. For every dollar spent on brand medicine, 50 cents go to an entity that didn’t make it. The reality is that spending on medicines is padding the profits of middlemen like pharmacy benefit managers (PBMs) and 340B hospitals, often at the expense of patients. Recent PBM reforms in the February Consolidated Appropriations Act were a positive step, but as policymakers continue to seek ways to address rising health care costs and drug spending, they must explore additional reforms to address the harmful consequences of growing consolidation among PBMs and insurers who are increasingly denying care to patients.

Finally, we must modernize the system to drive innovation by improving FDA efficiency, streamlining clinical trials and adopting AI and other new technologies to strengthen U.S. manufacturing and resilience. These steps reduce delays, expand economic opportunities and ensure patients get timely access to safe, effective medicines.

Direct‑purchase programs (DPP) are gaining momentum across the health‑care landscape. What factors are fueling their rise and how might they change how Americans pay for prescriptions?

SJU: A major recent development in our industry has been the growing support for pathways that allow patients and businesses to purchase medicines directly from manufacturers, known as direct purchase programs (DPP). This approach reduces barriers imposed by PBMs, which control which medicines patients can get, when and where they can access them and how much they pay at the pharmacy counter. 

Today, more than 80 million Americans in high deductible plans typically pay the full price of a medicine. At the same time, PBMs often receive significant discounts and rebates from manufacturers — savings that rarely reach patients at the pharmacy counter.

DPP — resources developed by manufacturers on an individual basis — can change that dynamic, delivering real price transparency and expanding patients’ access to medicines. For employers, it offers greater predictability and the potential for lower overall drug spend, reducing dependence on opaque PBM compensation structures.

We’re already seeing more DPP platforms emerge, including PhRMA’s own site, AmericasMedicines.com, a new search tool to help Americans find manufacturer-direct purchase programs and patient assistance programs in one place.

Providing patients with a clear, affordable path to their medications could fundamentally change how Americans pay for prescriptions. To ensure patients truly benefit from these programs, insurers should also do their part by counting medicines purchased through DPPs toward patients’ deductibles and out-of-pocket maximums.

Which scientific or technological advances give you the most optimism about the future?

SJU: We’re entering a remarkable era for biomedical science. AI isn’t just hype. It is reshaping how we discover medicines and get them to patients — from accelerating clinical trial timelines to uncovering new uses for existing medicines. For example, AI helped find that a drug originally developed for morning sickness could help treat multiple myeloma. If managed right, this revolution will enable scientists to cure more diseases, lower costs and create a more patient-centered health care system built for the future.

Cell and gene therapies are also transforming the way we prevent and fight disease. By addressing disease at the cellular or genetic level — often with a single treatment — they offer the possibility of long-term, even curative results. Take beta thalassemia, a rare blood disorder affecting about 2,000 Americans. New gene therapies have the potential to correct the underlying genetic issue of this disorder and deliver lasting, possibly curative outcomes.

Metabolic breakthroughs are also reshaping how we tackle chronic diseases. Just a few years ago, “GLP1” wasn’t part of the public conversation; today, these medicines are changing how we manage some of the most burdensome conditions in health care.

If we align policy with science, the next decade could eclipse the last, delivering more cures, transforming more diseases and improving millions of lives.

What message would you like to make sure that America’s leaders remember about these crucial policy questions?

SJU: I hope leaders take away two things. First, America’s innovation ecosystem delivers real benefits for American patients, families and communities — from access to breakthrough treatments to stronger health security and economic growth here at home. Second, that system is facing growing global competition.

To protect those benefits, policymakers need to address consolidation and misaligned incentives in the health care system, particularly among insurers and middlemen that too often limit access, distort competition and prevent patients from fully benefiting from medical innovation. The right policy choices can strengthen competition, improve access and ensure the U.S. continues to lead the world in developing the next generation of cures.