A: I think it’s the incredible pace of incremental and breakthrough innovation. Incremental innovation, for example, would be converting a product from reconstituted to ready-to-use (RTU) or reducing dosing frequency (e.g., 3 a day to once a day or daily injection to weekly injection). In other words, innovations that increase patient adherence or improve ease of use or safety for a caregiver or patient. Often, incremental innovation contributes to better outcomes, improved patient adherence, and reduced medication errors.
The breakthrough innovations are the new chemical entities (NCEs) or new biological entities (NBEs), cell and gene therapy, or those that offer a disease cure versus a symptomatic treatment. For example, consider the evolution of hepatitis C drugs from treatment to cure. When I started in the pharma business, which is not that long ago (1997), the treatment for hep C was interferon alfa, with significant side effects and limited efficacy. Today, the antivirals offer a cure. It’s a great example of breakthrough innovation made possible by advances in high-throughput assays, creating and screening massive combinatorial libraries and quickly analyzing massive databases to find the one or two molecules that actually hit the target with minimum side effect. Breakthrough innovation leads to improvements in the quality of patients’ lives and ultimately leads to cures.
What makes the U.S. market unique is a wonderfully dynamic and complex interplay between physicians, patients, providers, distributors, marketers, and developers. It’s a vibrant and maddeningly complex ecosystem that we have in the U.S. that is a real differentiator. There are inputs and feedback loops into what is needed to serve patients from all those parts of the ecosystem. On one hand, it can make for an unnecessarily long gestation period for any innovation to come to fruition. On the other hand, when we get it right, the innovative solutions are excellent, and the outcomes are great for patients. We need to set aside protectionism and individualism more often to better serve the greater good, alleviating our own human suffering. We need more collaboration.
A: Innovation, reliability, and transparency are some of the biggest differentiators when it comes to the U.S. pharma/biopharma industry. The number and percentage of new chemical or biological entities developed in the United States continues to grow both in terms of total numbers and percentage breakdown relative to other regions. Additionally, core areas of the United States continue to be hubs for development of new technologies.
Reliability and transparency are crucial in an industry focused on safely and efficiently saving lives and improving health.
As a key supplier into the industry, these are aspects that Grace has long embodied and are integral to our core model and the value we deliver to our clients.
A: The United States has scale, which is a competitive edge in a highly globalized industry like pharma or biopharma. From drug development and production to revenue generation, the U.S. is leading overall — representing nearly half of the global market. Five out of the top 10 pharma companies in the world are based in the U.S., spending more than $60 billion combined per year on research and development (R&D). And due to demographic mix and chronic indications, pharmaceutical spending is only increasing, with the U.S. expected to spend up to $650 billion in medicine by 2023.
The robust U.S. pharma or biopharma ecosystem is also a key differentiator. The U.S. has multiple geographical areas where noteworthy stakeholders interact closely, to innovate the pharma solutions of tomorrow — such as Boston, Research Triangle Park in North Carolina, and Cambridge, Massachusetts. Within those clusters of innovation, you can find the best and brightest minds — from top universities, venture capitalists and governmental institutions — who are willing to fund long-term research programs. On top of that, you’ll find privately held companies seeking to commercialize their products and large pharma companies ready to bring those innovations to market. Supporting companies, such as leading excipient or technology providers, also participate by providing enabling technologies that accelerate the process of bringing new drugs to market. For other countries across the globe, the U.S.’ advanced pharma ecosystem would be incredibly difficult to replicate.
A: A common challenge that the pharma/biopharma industry faces globally is managing high-risk R&D programs. These programs are lengthy as well as costly. These factors lend the pharma/biopharma industry to be the most R&D-intensive sector of the world economy. At approximately 15% of net revenues reinvested into R&D, pharma/biopharma companies are twice as R&D-intensive as the next two high-intensity sectors: technology/hardware and technology/software.
What differentiates U.S.-based pharma/biopharma companies is their historically higher contribution to the worldwide R&D effort, which currently stands at approximately 45–50%. As a result, the number of new chemical and new biological entities (NCE, NBE) introduced to the market by U.S.-based companies almost matches the number of all other geographies combined.
This U.S. dominance in pharma/biopharma R&D will persist for some time. Still, it will likely get less prominent as the industry globalization drivers continue to rebalance investments toward other regions with solid pharma/biopharma economic and R&D activities, such as Europe and China.
A: The two main differentiators of the U.S. pharma/biopharma industry are that the funding availability is much higher and there is a single drug supply across the entire United States, making trial design and packaging easier. The additional funding allows for more research and clinical development, leading to more lifesaving therapies for patients. In 2019, the pharmaceutical industry devoted $83 billion to research and development, and in 2020, clinical research funding by the National Institutes for Health reached $17.6 billion.
Additionally, trial design and packaging is more simplified in the U.S. compared with other countries, such as those in the European Union. Products must be adjusted to accommodate the various languages spoken across the EU, as well as ensure the specific design and packaging requirements of each country are followed and met.
Manny Soman, Chief Executive Officer, Verista
A: The United States is heavily investing in and chasing new discoveries in cell and gene therapy, especially in the post-pandemic era, which is creating tremendous opportunities for emerging biopharma in major hubs like Massachusetts and California. There is clear indication that the industry will double or even triple with products launching in record time. During the pandemic, we’ve seen mRNA drugs launch in 10 months, so we know how to get more drugs to market in a shorter time. With that said, the industry is highly regulated, which slows things down. Furthermore, the extensive data generated throughout the product life cycle, from R&D to manufacturing, needs to go through rigorous safety and efficacy scrutiny that stresses the system.
The advantages that Verista brings to our clients to enable them to achieve these goals includes our risk-based approach to validation, ensuring that critical business and regulatory functions are adequately tested to show that systems are fit for intended use and data is accurate and not compromised. We also leverage our partnerships with companies like Veeva and Kneat to help our clients with digital transformation, which enables them to get systems implemented faster to streamline business processes and bring data from the benchtop and manufacturing shop floor to the decision makers in less time.
Verista is the only compliance services company that can cover the entire drug development continuum. We have invested heavily to be able to help our clients maintain compliance from early drug development and R&D through commercialization, manufacturing, and post-approval support.
A: The U.S. market helps drive a lot of the innovation in the pharma industry, as the free-market pricing policies allow for risk-taking. We see this across the spectrum, as both innovative and generic players shape their product development strategy with the U.S. being the central component. The number of new drugs that are being brought to the market by small companies is a testimony to an environment that allows innovators to capitalize on their strengths while partnering with contract players across geographies. This is especially significant as we see a number of orphan diseases for which companies are able to provide solutions. This environment has allowed for advances in technology and increase in targeted therapies. Another aspect that is a key element is the role played by patient advocacy organizations in ensuring treatment reaches patients, as is seen in the recent case of the approval of Aducanumab for Alzheimer’s disease.
A: The U.S. pharma industry is one of the most pioneering groups in the world. There’s a real focus on collaboration and innovation as the foundations of its success, and we’re incredibly lucky to have some of the world’s best academic institutions driving a lot of that collaboration. For example, Dr. Richard Smith at Pacific Northwest National Laboratory has worked on most if not all ion mobility technology developments and is renowned across the mass spec industry for his achievements. His lab developed the structures for lossless ion manipulation (SLIM) technology that MOBILion’s first commercial high-resolution ion mobility (HRIM) instrument is driven by.
MOBILion Systems Inc. is based in the Northeast biotech corridor, so we have been lucky enough to see firsthand some of the outstanding work that happens through industry partnerships. Without collaboration, none of U.S. can work effectively –– we rely on our partners to tell U.S. how to develop instruments that meet their exact needs, and they rely on U.S. for the technology that can provide vast improvements to the data they’re able to generate from daily research.
Strong alliances across all aspects of the industry have enabled so much improvement not only to the development, manufacture, and marketing of new medicines, but ultimately to changing people’s lives for the better by diagnosing, treating, and curing diseases more quickly and effectively.
A: Our industry distinguishes itself through its flexibility to meet patient needs. The continually evolving global healthcare landscape leads to new requirements for safe and effective drug products. Supporting clients across 35+ countries, Alcami continues to adapt and advance our technologies like nitrosamines testing, extractables and leachables, elemental impurities, and rapid sterility to achieve evolving regulatory requirements in order to support our clients in producing high quality, safe, and effective drug products.
Beyond that, the industry must stay agile by keeping an eye on the future while having a pulse on the ground. This means investing in the right instrumentation, scientific teams, and forward-looking capabilities while ensuring that efficiency and customer service aren’t compromised. Companies like Alcami must continually strive to advance internal regulatory and subject matter expertise to reduce project obstacles.
A: There are marked differences as well as notable similarities between the United States and rest of world.
While the U.S. represents the largest pharma industry in the world, it is followed closely by China and India, both of which, like the U.S. industry, are seeing growth and attracting investors. One major difference is that the U.S. maintains the strictest regulatory framework, although China and India are catching up fast.
The United States is home to some of the world’s biggest pharma companies, in contrast to India, for example, where smaller contract manufacturing organizations (CMOs) dominate and focus mainly on manufacturing generics. The symbiotic relationship is clear: R&D is high on the U.S. agenda, and manufacturing can often be provided by Indian CMOs, further driving the imperative for unified regulatory frameworks, which will make the pharma industry truly global.
The U.S. pharma industry is characterized by the availability of capital and the market appetite to support a high-risk/high-reward R&D strategy in complex therapeutic areas. It spends far more on R&D than any other country and, therefore, has the highest healthcare costs.
Unifying factors across the globe include the length of the drug development cycle, the length of time data needs to be kept, and the rigor of the regulatory process — all of which have left a legacy of resistance to adopting new technology.
This resistance is crumbling and comes at an exciting time for the U.S. life science community. Maturing technology, including the IoT, artificial intelligence (AI), computational power, and sequencing, coupled with the spirit of innovation that is now — finally — making inroads into pharma is revolutionizing the sector. The science community welcomes new disruption and is starting to take a high-tech approach to solving old problems, like connecting the diversity of analytical equipment in the lab to feed new machine learning strategies that unlock data-driven insights into developing new therapies.
A: The U.S. pharmaceutical and biopharmaceutical market is truly a unique driving force within the industry; despite consuming only about 10% of the world’s medicines by volume, the total healthcare spend in final dosage forms equates to roughly 50% of pharmaceutical total market by value. The impact of this means that most drug targets for approval are focused with the U.S. as one of if not the primary market. Moreover, the total spend on pharma from the United States has a direct impact on the pharmaceutical R&D pipelines, whose comparatively large financial allocation to R&D is based largely on the profits derived in the U.S. market. This also gives a comparatively large responsibility to the U.S. FDA on the approval of these medicines and often sets the standard for approval within the globe. This was quite evident with the recent COVID-19 pandemic, where medicines where researched, developed, scaled-up and distributed first within the U.S. markets and the Western EU. The U.S. market is also ripe for innovation, as nowhere else in the world are small and medium sizes companies able to take an idea through to commercialization through the systems of funding available to entrepreneurs. To address this corner of the market, we at BASF, a leading supplier of innovative pharmaceutical and biopharma ingredients, established strong technical competencies, laboratories, and manufacturing sites within the United States to match the innovation power. We have also established expert colleagues geographically positioned in the leading markets, like Boston and California, in addition to our headquarters in New Jersey, the leading region for pharmaceutical innovation. The U.S. remains highly important for us, to ensure collaboration and co-development of leading medicines with innovative, biopharma, and generic pharmaceutical companies.
A: Understanding complex biological phenomena has required concurrent advances in experimental methods and computing power. As Freeman Dyson observed, “new directions in science are launched by new tools much more often than by new concepts.” Advances in new tools are generating novel data modalities and higher-resolution data. These data, along with rapid advances in algorithms and large-scale computing, are driving the creation of ever more comprehensive models of disease, disease progression, and health at both the individual level and the population level.
While the U.S. invests more aggressively in innovation across all these fronts, collecting and connecting data is hindered by the greater fragmentation of healthcare data in the U.S. than in countries with national healthcare systems, as well as by cumbersome electronic heath record (EHR) systems that do not yet incorporate patient omics data. Even so, pharma/biopharma companies that are leveraging new integrated analytics platforms are able to combine multi-modal, multi-sourced data, expediting the discovery and delivery of new, more effective, and safer therapies to patients.
A: COVID-19 has demonstrated that we need a healthcare system that is better prepared for future public health emergencies for all patients. The substantial challenges faced by the U.S. and its local public health authorities in testing, treating, and vaccinating Americans during the pandemic highlight key infrastructure gaps in the healthcare system that need to be addressed.
COVID-19 has demonstrated the critical importance of globally diverse biopharmaceutical manufacturing supply chains to respond to crises and has highlighted the potential benefits of bringing back the manufacturing base to the U.S. and the Western world.
By continuing to foster collaboration, steps to address gaps in the science, technology, engineering, its workforce, and policies to incentivize investment in R&D and manufacturing infrastructure, the U.S. pharma industry could plug key gaps and be ready for the future.
Despite the influence of several emerging countries, the United States has captured a dominant share of the pharmaceutical market worldwide. The country is home to some of the biggest pharma companies worldwide, and American consumers have access to one of the most advanced pharmaceutical systems in the world, albeit at a cost. According to Statista, the pharmaceutical sales revenues in the United States have accounted for nearly half of the global total in recent years. Five of the leading pharma companies worldwide were from the U.S. in 2019-2020. (https://www.statista.com/topics/1719/pharmaceutical-industry/)
U.S.-based pharma companies spend around 20% of their revenues on R&D, which is a sizeable investment. The R&D expenditure of the U.S. pharmaceutical industry was more than U.S $10 billion in 2018.). It is very likely that this figure will significantly increase in the years to come due to the costs associated with developing safe and effective COVID-19 treatments and vaccines.
Nancy Lurker, Chief Executive Officer, EyePoint Pharmaceuticals
A: Simply put, no other market or country develops and commercializes nearly the same volume and scope of innovative drugs as the U.S. We have an incredible innovation engine in the U.S., and it allows us to save more lives. The COVID-19 vaccines are a prime and timely example of this.
Jeff Elton, Ph.D., Chief Executive Officer, ConcertAI
A: The story less told is that the U.S market has the strongest foundation for advanced AI and other insight tools of any in the world. It has 100% consistent digital clinical infrastructure in place as a result of the full implementation of the HITECH Act, giving us more access to clinical data in electronic form than any other country. This is why the real-world data (RWD) market is larger and growing faster here. If you have data derived according to consistent standards, you have the basis for AI and machine learning solutions.
We saw the benefits of this market with COVID-19 research initiatives –– the mobilization and speed of clinical research was breathtaking. We were able to accelerate our understanding of primary modes of transition; catalyze an almost entirely digital and decentralized trial infrastructure ‘incubating’ for years; and make determinations of benefit and risk for narrow populations in record time.
RWD is being used to plan almost every trial. It is supporting the growth of the digital armamentarium of evidence-generation approaches — extending and augmenting the legacy approaches of randomized controlled trials — by bringing more evidence together, faster, for provider, biomedical innovator, payer, and regulatory decisions. While the F.D.A. may have issued preliminary perspective on RWD in 2018, the real advances in use and utility have been in the last 18 months. Even with the debates of late around accelerated approval processes, it is clear that standard of care analyses and complementary views on the effectiveness and safety of current therapies will become part of almost every regulatory submission and integrated into every decision.
Mike Hopkins, Vice President, Business Development, U.S., Sterling Pharma Solutions
A: The key differentiator for the U.S. pharma/biopharma industry is the diversity and current availability of funding and investment. Innovator drug development companies can readily access funding through venture and equity investment, while the IPO market is also very favorable in the United States.
With this increased access to funding, coupled with the rise in orphan drug designations, smaller companies are able to support clinical development of programs further than previously possible — even to commercial launch — when, typically, their focus would have been to out-license or divest a product soon after successfully completing phase II trials.
This has led to an increase in innovation and the development of new products, with the majority of small molecules in the clinical pipeline being developed by small and virtual companies, some with only limited numbers of internal programs. This combination of factors has resulted in a healthy emerging pharma sector within the United States, and for contract development and manufacturing organizations (CDMOs), the small nature of many of these companies has been a key growth driver.
Nate Mccutcheon, Executive Vice President and Chief Operating Officer, Shionogi
A: Globally, our industry has an obligation to contribute to society in a way different from other industries by advancing scientific innovation to help patients live longer, healthier lives. However, how this innovation is encouraged greatly depends on the environmental factors in place to reward this critical work.
Innovation today is significantly supported by private investment, and, to receive any investment, there is a requirement to show a stronger opportunity for return than other industries. Striking the right balance of obligation and incentive is difficult for many U.S. pharma/biopharma companies. As an example, we have seen multiple companies committed to advancing science to help address antimicrobial resistance fail in their mission, despite the critical need for advances in this disease area.
To date, and with some exceptions, the U.S. government has not extensively regulated the price of prescription medicines, instead relying primarily on market forces to determine prices. This has created a complicated and unique reimbursement system, but a stronger incentive than other markets worldwide to develop new innovative drugs, while also increasing incentives for the use of generic products.
While the U.S. system may not be perfect, it is still one of the world’s largest markets, helping to ensure a sustainable model that encourages breakthrough innovation on a global scale, the core of what drives our industry.
Maaike Everts, Ph.D., Director Strategic Marketing Parenteral Drug Delivery Solutions, Evonik
A: One important factor that differentiates the pharma/biopharma industry in the U.S. from others around the world is the drivers for investment. In the U.S., early-entry pharma and early technologies — such as the mRNA technologies we are seeing now — are encouraged and rewarded. The availability of venture funds and a culture of capitalizing on novel ideas from academia makes the U.S. an attractive environment for early adopters of new technologies. In contrast, the EU often lags in terms of investment and funding, even though they catch up in the long term. An advantage of the EU, on the other hand, is its more expansive capabilities for handling biologicals. With our global presence in locations across the U.S. and the EU, Evonik is well positioned to benefit from the best of both markets.
Marie E. Lamont, President and Chief Operating Officer, Inteliquet
A: The U.S. market is defined by size in terms of both population and investment in innovation, and also by complexity.
The size and diversity of the U.S. population, the number of diverse healthcare organizations competing to provide better care, and a system that rewards entrepreneurship and taking a chance on finding better solutions has continued to make the U.S. either the home of innovators or a destination for them. Our higher education system in particular rewards the practical application and testing of new ideas in a free and competitive market. That translates the brilliance of scientists into access for patients, and innovators are rewarded and celebrated. Consider the number of technology and science accelerators as an example of this.
The complexity of the U.S. market can also be the most frustrating in the world. There are layers of process between manufacturers, the payers of treatment, care, or therapeutics, and the patient. These layers of complexity often make it difficult to understand how funds flow and the true cost of healthcare. Yet while frustrating, this very complexity engenders creativity, technology revolutions, and unconventional solutions, which dramatically change how care and treatment are delivered. Social determinants of health are an important focus for the WHO and countries around the world, but the U.S. market using intriguing technology and data is a leader in using population health to drive care options and health offerings to it citizens.
Yan Zhang, Ph.D., Chief Executive Officer, Mission Bio
A: We have so many accelerators here — great science, tons of experience, creative corporate leadership, and a strong regulatory structure. But there’s a clear bottleneck in getting the benefits of our industry to patients, and that’s the U.S. insurance system.
There are well-established issues around fair access to healthcare, cost structure, and the tension between lowering costs while improving quality care. All of these things will be further exacerbated as we develop the next generation of diagnostics and advanced therapeutics.
Powered by the growth of genomics, we are on the verge of turning medicine into what we’d always dreamed it could be: preventive instead of reactive, personalized instead of prescriptive, curative instead of lifetimes of treatments.
But if a new therapy costs $1 million, insurance companies may not pick up the tab, even if it is curative, less expensive long-term than years of ongoing therapy, and prevents further, costly complications down the road. The insurance market is structured around employers as payers, and, since employees don’t stay with one company or insurer for a lifetime, the long-term benefits may be less attractive than keeping down short-term costs.
The pool of uninsured and underinsured also are at higher risk for inferior treatment of their chronic illnesses. That’s unfortunate on its own but is compounded by the fact it shrinks the potential market for new therapies that disproportionately impact this group, which slows down bringing the benefits of genomics and most advanced therapeutics to those who can benefit the most.
Fabian Gerlinghaus, Co-founder and Chief Executive Officer, Cellares
A: Uniquely, the U.S. biopharma industry benefits from robust long-term support for novel technologies. The two key pieces here are public funders, which continue to back early research, and investors who are willing to place big bets on novel ideas. This is exemplified in the cell and gene therapy space on the one hand by billions of dollars in long-term support from NIH and California’s CIRM, and on the other by the record-level venture investments and IPOs seen last year alone.
As a result, the cell and gene therapy space has added so much momentum that demand well outpaces supply for approved therapies. Cell therapies in particular have extended manufacturing timelines, with challenging complexities due to their level of patient personalization. Addressing the manufacturing bottlenecks will be key to unlocking the next generation of therapies, which have the promise to treat more patients more quickly.
This unique ability of the U.S. market to find and support innovative solutions promises solutions for these manufacturing hurdles, and is already on track to do so. We are gratified that investors have backed Cellares, for one, recognizing the possibilities for our automated Cell Shuttle technology as an exciting tool for meeting this burgeoning demand.
Glenn Mattes, President, Chief Executive Officer, and Director, TFF Pharmaceuticals, Inc.
A: What differentiates the U.S pharma/biopharma industry is its size and the diverse approaches to addressing different target indications. The U.S. is the world's largest single pharmaceutical market, generating more than $490 billion of revenue.
R&D is focused on precision medicine, offering more targeted therapies by discovering specific disease biomarkers, tumor antigens, and molecular targets that maximize efficacy and reduce side effects. At TFF Pharma, we are also focusing on targeted delivery by administering drugs directly to the site of infection, which could lower dosage and potentially improve efficacy and adverse side effects.
We have also seen that partnerships and collaborations can really be a perfect marriage for getting products to market. Collaboration is part of our DNA, so we partner with a broad array of pharmaceutical companies, academic institutions, and government partners, merging resources and expertise to advance research efforts and improve patient outcomes.
Ian Walters M.D., Chief Executive Officer and Director, Portage Biotech
A: In my view, it all starts from the ground up. In the U.S., the biopharma industry greatly favors early-stage company formation. The appetite for risk is also quite different. The funding ecosystem for early-stage startups is more advanced than in other countries, which allows for more innovation and greater acceleration of clinical development for biopharma products. Additionally, the U.S. capital markets are more rewarding of novel technologies, which further enables both development and innovation. This means that more technologies discovered in the U.S. have various avenues to move toward the market and also means that novel and innovative products that may carry more risk still have the funding and development opportunities that they need to be successful.
At Portage Biotech, we have capitalized on the advantages of the U.S. pharma and biopharma industry while also bringing technologies into our pipeline from places that don’t have the same opportunities as in the U.S. market. These historically under-appreciated technologies — including our lead invariant natural killer T cell (iNKT agonists), developed via technology licensed from Oxford University — allow us to stand out from the typical U.S. market, but our footprint in the U.S. ensures we have the funding and resources to aid in their successful development.
Marco Taglietti, M.D., Chief Executive Officer, SCYNEXIS
A: The U.S. pharma/biopharma industry has been and remains the most dynamic and productive in the world, thanks to a combination of resources, scientific talents, financing opportunities, marketing incentives, and ingenuity. It’s this extraordinary and unique combination that has allowed the U.S. biopharma industry to advance the field of medicine at an unprecedented pace. The pandemic has underscored what can be achieved when pharmaceutical companies collaborate with each other and academic research institutions to develop safe and effective therapies and vaccines. New promising discovery paths open when companies explore novel approaches, such as in silico medicine, mRNA technology, or individualized medicine, to list just a few examples.
However, we should not take for granted that this marvelous ecosystem will last forever unless we work to preserve its strengths and vitality. Policy changes, even if well-intentioned, may have unexpected consequences that could disrupt the ability to attract new talent, achieve adequate return on investment or find adequate financing resources, which ultimately could limit the industry’s capacity to find new treatments to help improve the health and wellbeing of us all.
Prakash Pandian, Chief Information Officer, Curia (formerly AMRI)
A: The changing dynamics of healthcare — whether from a policy standpoint or patients taking more control of their own health — stand out as a differentiator when comparing the biopharma industry in the U.S. to other countries. Things can move fast here. The coronavirus pandemic is a good example of that. It created urgency unlike any other and as an industry, we had to research, develop, manufacture, and deliver large amounts of vaccines and drugs to the market quickly. In addition, the pandemic forced us, especially here in the U.S. as innovators, to focus on advanced technologies — both biological and digital. We are actively engaging in AI and machine learning and dealing with cybersecurity. There is competition for talent, which is in high demand. The U.S. is a leader in innovation, but other countries are nipping at our heels. It’s no time for U.S. to be complacent.
Talat Imran, Chief Executive Officer, Rani Therapeutics
A: The U.S. has an amazing appetite for innovation. There is a high concentration of talented researchers and scientists at universities and corporations here, and they come from around the world. I know this firsthand, as my father, Mir Imran, an accomplished medical innovator and entrepreneur, came from India to the United States because he believed the U.S. was the best place to learn, create innovations, and build companies. With so many great minds collaborating, innovation flourishes in the U.S.; it’s no surprise that roughly two-thirds of the biggest pharma companies are based here.
Omid Farokhzad, M.D., Chairman and Chief Executive Officer, Seer Bio
A: The United States has a biopharma market that encourages innovation and risk-taking, letting new technologies take root and grow — often before they can do so elsewhere in the world. Part of that is due to the huge amount of capital available, which enables companies to dream big and plan long-term, and certainly the open and transparent regulatory environment means innovation is enabled. U.S. biopharma companies have often set the pace for the incorporation of novel technologies and approaches to arrive at discoveries otherwise not possible. Today, this spirit of innovation and risk-taking is more alive than ever before and we are all beneficiaries of it.
Dr. Kurt Nielsen joined Pii in 2019 as President and CEO. He is a seasoned pharmaceutical executive with over 20 years of diverse experience, most recently as the President of Lupin Somerset, responsible for all their generic and branded products. Prior to Lupin, he held the post of Vice President, U.S. Development, Portfolio and Launch Management at Sandoz Inc., where he was accountable for the U.S. development of generic, OTC, and specialty brand products. Dr. Nielsen has also held positions at Catalent, where he was Senior Vice President of R&D and Chief Technology Officer, and URL Pharma where he was the Executive Vice President, Pharmaceuticals.
Many of the change agents I have seen in 2019 are derived from changes in regulatory law, commercial downscaling, and impact from patent expiry strategies. The largest external regulatory change came from the issuance of the long-awaited EMEA Annex I, clarifying which technologies are required and acceptable, when and why.
The change in operational focus, from clinical scale-up to commercial scale-down, is enabling use of smaller, modular, flexible fillers with self-contained isolators. In parallel with the approval of biosimilars and biobetters, there is strong industry focus on individualized micro-batches, for CAR-T solutions and gene therapy products. The use of process automation and robotics have increased in all fill-finish unit operations. Widespread implementation of ready-to-use/ready-to-sterilize components and single-use (SUT) in upstream and downstream (SUS) through final fill designs have changed how facilities are planned, reducing plant size and changing warehouse space to accommodate densely packaged plastics goods.
Filling modalities have also been changing; bags that can be mated to lock-luer fittings with pre-sterilized needles and blow-fill-seal/form-fill-seal are re-emerging as processes that offer potential unit cost reduction. Traditional vial and syringe container designs are also changing as suppliers improve standardize offerings while having options including clear plastics.
The most exciting technological or scientific advancement that has influenced our business strategy in 2019 is our novel epigenetic regulator program. Unlike gene therapies, which target and modify DNA directly by inserting specific genes into patient’s cells, epigenetic regulators control or modify gene expression through processes that do not alter the sequence of DNA directly. Our lead asset DUR-928 is a small endogenous molecule that plays an important role in regulating cellular functions such as lipid homeostasis, inflammation and cell survival, crucial pathways involved in many acute and chronic diseases. DUR-928 has shown positive results in a phase IIa trial for the treatment of alcoholic hepatitis, a devastating acute condition with high mortality rates and limited therapeutic options. We are also advancing programs in other indications that could benefit from DUR-928, such as non-alcoholic steatohepatitis (NASH) or psoriasis. We believe that epigenetic regulation is a powerful and untapped treatment approach for many challenging diseases.