October 11, 2022 PAO-10-022--RT-02
Keeping pace with the changing trends and fast proliferation of demands on businesses for environmental and social regulations is certainly the main challenge for most industries. The speed of implementation of tangible measures, scalable improvements, and clearly defined objectives will make the difference and act as key indicators of success. The life sciences industry is certainly used to enabling growth in a highly regulated environment, but balancing risks and goals in the context of sustainability without clear guidance from regulators can easily end up akin to driving without a dashboard. Considering the unique multidisciplinary capabilities of the players in this field, I see enormous potential to leverage synergies and set new benchmarks for a strong and healthy future.
A key indicator that Agilent looks at to predict the future of the biopharma/biotech industry is the industry drug development pipeline. We are seeing a robust pipeline expansion in the preclinical and phase I stages that will lead to the expansion in later stages and ultimately in the approved biologic drugs in the coming years. We also monitor the drug development pipeline distribution between small, medium, and large companies so that we can better serve our customers in these segments.
For Agilent, as a global company, it is also important to track the industry pipeline by country. For example, we’ve recently noticed a significant increase in the new biologic drug development programs in China. This highlights a pivotal shift in R&D funding from biosimilars to novel therapeutics in that country.
And finally, Agilent monitors the investments into emerging drug modalities, such as cell and gene therapies. These programs carry the promise of changing standards of care in several disease categories, primarily cancer, and in previously uncurable genetic disorders. Agilent is well positioned to serve customers developing these emerging therapeutics with a broad portfolio of LC-MS, cell analysis, and genomics tools, as well as our GMP manufacturing capabilities for CRISPR guides and synthetic oligonucleotide APIs.
Over the past few years, we’ve seen the ability of the biotech industry to remain agile and adapt to new challenges like the COVID-19 pandemic. The urgency demonstrated during this time showed the strength of the industry and our ability to act quickly. One of the key indicators that has been, currently is, and will remain top of mind in the future success of the industry is patient needs.
While the biotech industry has seen life-changing innovations, there is still a substantial unmet need to address indications and disease areas that have not been successful to date. The future of the industry must remain focused on the ability to meet patient needs with innovative medicines, which will continue to be relative to what the market needs. A market adjustment and an increase in M&A and partnering will help advance clinical developments and bring novel solutions to patients.
I continue to see a huge uptick in high-quality science identifying potential new drug targets, and I view this as a signal of a bright future for the industry as biotech and large pharma look to advance these to the clinic with joint efforts. Through partnerships, the biotech industry will continue to improve the standards of care and shake the whole therapeutic field to offer new solutions. The innovation should be a strong driver and the indicator of a bright future for potential breakthrough treatments coming to patients and to the market.
At Astrea Bioseparations, we are dedicated to solving the downstream processing and bio-purification issues around cell and gene therapy, where traditional purification techniques are inappropriately re-purposed. Targets, such as lentivirus, adeno-associated virus, exosomes, plasmid DNA, and mRNA, are migrating rapidly toward adoption by healthcare professionals, the market, and patients alike. However, healthcare regulators have struggled to stay abreast of such rapid progress. A key indicator that will define how we support this critical new wave of therapeutics will be how leading regulators define the scope for their entry into clinical trials and beyond. This embryonic regulatory roadmap will direct the quality characteristics of the products that we are developing to support next-generation therapeutics.
Additionally, as we write this, global news outlets tell us that we are on the verge of a global recession. Inflation is wreaking havoc on many nations’ ability to service healthcare obligations to their citizens. These kinds of economic indicators should influence the future of our industry. We have an obligation to develop purification solutions that reduce time and cost of drug development. At Astrea Bioseparations, we have novel nanofiber-based technologies that allow “process compression.” Our goal is to significantly reduce the time and cost of purification using this paradigm-changing technology.
The critical indicator of the future of the pharma industry, including the future of cell and gene therapy (CGT), where we supply services, is clinical data. If the field continues to produce excellent clinical data with its second- and third-generation therapies, then the field will flourish, capital will flow into the field in large quantities, and many thousands of patients will stand to benefit. Like all participants in this emerging field, we monitor clinical developments very closely and, of course, have the benefit of supplying services to over 50 CGT developers, so we are acutely aware of the status of CGT clinical development.
Some people like to say that “the future is the in the stars.” In our scientific field, the appropriate version is “the future is in the pipeline!” This industry can essentially foresee most of its development, manufacturing, and commercial needs for the next 10 years, since the progression of different modalities through the pharmaceutical pipeline, from discovery and preclinical development through to commercialization and pending patent cliffs, is transparent. This can be witnessed from the stage where innovators are working on emerging modalities that show exciting possibilities in preclinical development, as well as when new modalities progress through to phase I studies and ultimately to high-potential new treatments that make it through to late-phase development and near commercial launch.
With pipelines reaching an all-time high — whether focused on new molecules, compounds, cell and gene therapies, or other emerging modalities — the industry as a whole now faces a challenge as to where to invest capital wisely and efficiently to meet the diverse needs of future development, manufacturing, launch, commercial execution, and ongoing supply of a record number of new products across an unprecedented number of modalities.
In addition to investment, successfully bringing this vast array of treatments to patients around the world will require the careful analysis of multiple indicators. These include development timings, success rates, changes between therapies’ initial and expanded indications, patient population sizes, and the necessary required production volumes across various dose forms, as well as many other factors. If we are to deliver successful treatments for patients, the industry needs to develop and evolve to more flexible business models, as well as leverage strategic partnerships across the value chain and find more efficient ways to allocate capital in time to be ready to serve our patients.
The cell therapy industry came of age in 2017 with the first U.S. FDA approval for a CAR-T cell therapy, and the industry is expected to only grow exponentially in the coming years. In 2022 alone, there have been multiple label expansions and one new therapy approval, and the FDA expects the rate of new therapies to increase further over the next decade. Looking ahead, Cellares is tracking regulatory approvals, new data, and technology advancements to forecast the future of the cell therapy industry. I’m paying close attention to clinical trial readouts and which therapeutics are delivering improved overall response rates, as well as expanding to other indications beyond oncology. Understanding the therapeutic pipeline helps to understand which new modalities are gaining traction. Another emerging trend over the last few years is the increase in allogeneic programs entering clinical trials. Other cell therapy modalities, including regulatory T cell (Treg), natural killer (NK) cell, and gamma delta T cell therapies, are also advancing closer to approval. Additionally, I’m tracking the M&A, IPO, and fundraising landscape within the cell therapy industry. By understanding where the money is going, Cellares can gain immense insight into industry and investor confidence and directionality. Lastly, one of the best ways to stay abreast of new innovations and cell therapy trends is to work closely with academic partners. By handpicking relevant groups, Cellares is learning what the latest and greatest technological advancements are, as well as what upcoming cell modalities are becoming more relevant as the engineering capabilities continue to develop.
As a leading global CRO and CDMO with a broad portfolio ranging from early discovery through to drug product manufacturing, Charles River has a unique line of sight across the biopharma industry. We use a variety of external indicators, data sources, and consultative relationships to validate and build our business strategy. These can be broken into two main categories:
Healthcare is an industry that is founded on innovation. Over the last 25 years, we have seen a percentage reduction in cancer deaths as a result of new medical innovations, such as immunotherapies. Similarly, we see existing, already approved drugs improving in their effectiveness over time. So, to predict any aspect of the future of this industry, we need to closely monitor the following 10 areas:
In looking at the future of the pharmaceutical industry, we examine the industry's overall contributions to the climate change crisis. With the SEC's new climate change disclosure rule set to go into place, it is more important than ever to look at greenhouse gas (GHG) emissions, including Scope 1, 2, and 3. It's imperative for companies to set baselines for their GHG emissions and begin reduction measures if they have not done so already. We're seeing a strong increase in science- and data-driven third-party programs like the Science Based Targets initiative (SBTi) from our clients. SBTi's goal is to honor the Paris agreement and keep global warming below 1.5 ºC. For the pharmaceutical industry to continue to thrive and positively impact people and the environment, we must measure progress, set science-based reduction targets, and develop a plan to execute.
The rate of change in the life science industry continues to accelerate even as the pace of funding moderated over the last year.
We are focused on the promise of curing intractable diseases, especially cancers, that can be achieved with gene-modified cell therapies. The challenge of robust, cost-effective manufacturing remains the primary barrier to broad patient access and benefit from these breakthroughs. To understand how quickly we’re progressing, we keenly follow the cost to deliver treatments, the number of patients who can access them, and how much we’re innovating.
Today, cell therapies are manufactured through bespoke processes with dose manufacturing times that drive costs upward of several hundreds of thousands of dollars per patient. It’s hard to see how these therapies can become widely available unless manufacturing costs drop.
Meanwhile, demand will rise by orders of magnitude from today’s levels. Investment in manufacturing capacity is a clear indicator of access, but even more critical is how companies try to address the growing skill gap to run production systems.
These fundamental drivers inform our work transforming the cell therapy manufacturing model. Automation can dramatically minimize the losses and variability of the patient-derived input to the manufacturing processes. By delivering superior cell product quality and quantity, we look to reduce the manufacturing time and cost for CAR-T and other cell therapies by up to 50% and drive down the infrastructure and staffing costs to make a dose. These support our goals of enabling the broad, global deployment of life-saving therapies to patients.
Our industry is paradoxically conservative and fast-moving, driven by advances in biology, computing, engineering, and medicine. Staying ahead requires a paradoxical approach to monitoring trends: choosing where to go wide and where to focus. Our teams’ diverse backgrounds and interests help enormously. We each think differently; we see different things.
We monitor macro trends predicting end-market demand: socioeconomics, demographics, population health, epidemiology, and government policies and incentives. What does disease burden demand, and what is the financial and policy climate encouraging? We follow the money: where customers and VCs are investing, M&A activity, and research funding. Who is making which bets?
Analyzing clinical trial and drug pipelines, new therapy research, marketed drug sales, expiries, new indications, and evolving research methods help us predict customers’ unmet needs. We listen to customers in multiple ways and track purchasing intent and behavior. Our bespoke biopharma resilience index helps understanding customers’ macro concerns, how they are addressing them, and how we can help.
Beyond indices and data, our team’s collective intelligence is crucial. We have thousands of industry and technology experts working alongside customers from research to biomanufacturing who regularly flag developments. With industry veterans that have grown and driven the industry over the last 45 years, we benefit from wisdom and insight that our customers value deeply.
I encourage people to explore adjacencies and be adventurous, too: read widely! And then, there is science fiction’s mixed record in futurology. Recent advances in engineering, biology, and computing, however, mean that we approach a time when the future will be what we imagine into being.
We work to identify leading indicators for the industry as a whole, along with the health of our business. To assess the health of life sciences overall, we look at the flow of capital into the various segments of the industry, including the funding of new companies, mergers and acquisitions, and the number of net new companies sponsoring research. Technology and business model innovation trends in other vertical segments outside of life sciences that help increase productivity also help us determine what the next big thing may be for our industry. We regularly track the trends in healthcare digitization and scientific breakthroughs that have potential applications for human health. We are extremely fortunate to be in a growing industry that has tremendous untapped potential to deliver scientific breakthrough faster and at lower costs in the coming decades.
We look at changes both in terms of clinician needs and the changing needs of our commercial clients. In the case of clinician needs, we evaluate the changes happening in their practices and patient sets. This includes indicators like overall employment figures for physicians and other clinicians, the rate of self-employed versus institutionally employed, adoption of technology like telehealth and other digital health solutions, practice scope changes, and emergent/growing diagnoses.
For our commercial clients, we look at their drug development pipelines that indicate which audiences they likely want to engage with going forward. We also look at advertising technology trends and how they may impact the expectations of our clients. And, ideally, we’re also helping our clients better imagine how they can effectively engage clinical audiences to be sure the right message is delivered at the right time.
A global system that assesses the cost to the environment and rewards sustainable solutions without compromising drug quality would motivate the industry to move in the right direction. Key indicators to analyze the status quo and make the right decisions for future developments can be found on several levels. On a global level, we see that the healthcare sector contributed to about 4.4% of the global total greenhouse gas emissions in 2014. The industry will need to change as a whole to make a sum global impact, so an important measure is the Scope 1, 2 and 3 emissions of companies (in kilotons of CO2 equivalent per year).
To support this, we need harmonized regulations and metrics that rate the sustainability and “greenness” of products and processes. Pharmaceutical products play a key role in achieving the UN Sustainable Development Goal of “good health and well-being” and therefore have a positive “handprint.” The design of pharmaceutical products that provide high functionality at low doses and a shift from curative drugs to low-dose preventive products are great developments. For example, mRNA vaccines provide a significant handprint at low doses while having comparatively low environmental footprints. For functionally equivalent drugs, the comparison of process mass intensity and carbon footprint per kilogram of manufactured product are essential indicators. In this context, one of the central elements of our sustainability strategy at Evonik Health Care is portfolio sustainability analysis (PSA). This leads to an annual Product-Application-Region-Combination (PARC) ranking for the whole product portfolio. Such analyses help us ask the right questions and identify areas for improvement in the product portfolio.
As the population continues to age, serious eye disorders that can cause blindness, including age-related macular degeneration (AMD), diabetic retinopathy, diabetic macular edema (DME), and retinal vein occlusion represent a growing area of unmet need.
While there are a number of available drugs considered to be effective in this space — requiring regular, ongoing intraocular injections — they are often uncomfortable, intimidating, and inconvenient to the point that treatment adherence can become a potentially blinding eyesight issue. And we know from ongoing studies of large databases of information that, for a majority of patients over time, their eyesight continues to worsen, not because of therapeutic efficacy but because of lack of treatment compliance. Who wants to get eye injections every month or every other month for the rest of their lives?
One of the key advancements that I see for the future of vision care is focused on the development of new solutions aimed at maintaining the vision gains that current treatments are providing patients over the long term. With an aging population, the prevalence of these serious eye disorders is growing at a rapid pace, and they can cause serious vision loss to patients, as well as a tremendous care burden to their caregivers and to our overall healthcare system. I see these challenges, while robust, as an opportunity to drive potentially eyesight-saving advancement and innovation in the space.
The next big wave of innovation in serious eye disorders is focused on increasing the longevity of action and durability of available treatments to ultimately relieve patients and their caregivers of the burdensome, frequent treatment regimens, all while maintaining their vision.
Two things will be key: companies will need to focus on real-world outcomes, and the era of targeted therapies is over. This is the last decade where we can focus on trials without mirroring those specific outcomes in real-world evidence. The political zeitgeist simply will not support that any longer. So therapeutic companies will need to look to ensure that the product lives up to its promise. We will need to focus on whole-body interventions like immunotherapy and whole-body approaches. There’s limited remaining efficacy in targeted therapies — we’ve squeezed most of the juice out of that.
In terms of fundraising and valuations, a couple of larger deals (Merck–Seagen, for example) and some meaty later-stage rounds, together with the success of the next round of COVID-19 vaccines, will shake the industry out of its malaise. We won’t go back to pandemic-era valuations or availability of capital, but that was undisciplined investing, and it is best that it’s just a blip on the radar versus a new normal.
While detailed data analysis is required to accurately predict exactly where the industry is going, we can still provide a picture of where the industry is moving. In order to gain an understanding of the industry, it is critical to evaluate the various stakeholders and where they are putting their efforts, including research institutes, universities, and biotech companies.
One of the most important indicators of the industry’s future is an in-depth understanding of the pharma pipeline — starting with research via preclinical studies and finishing with clinical phases and eventual approval of drug candidates.
At IFF, we spend a significant amount of time data mining the pipeline to predict how the industry is evolving and to anticipate changes. Once a drug has been successfully commercialized, we follow it closely through its product life cycle. Certain areas of focus, like patent extensions and additional approved indications, are equally important to track. From there, we will assess the generic filings and country-specific reimbursement policies to better determine the degree of acceptance and penetration that a drug will have in the market.
The future of the biotech industry is shaped by innovation shifts as researchers recognize value and build on each other's findings. Whether it is at conferences, through strategic collaborations, or from data identified through clinical trials, scientific progress serves as the foundation for the next wave of innovation. Also, discoveries in one field can impact other fields, influencing perspectives on all sides and evolving technologies into something new.
Therapeutic vaccines for cancer are one example of current shifting perspectives in our industry. Historically, this approach was overlooked due to previous limited success in patients related to the inability to elicit a sustainable, comprehensive, and targeted immune response against tumors.
The expedited response of the industry to the pandemic was made possible by prior drug improvements in delivery formulation to drive improved and more efficient immune response. This has brought a remarkable enthusiasm for platform-based vaccines.
In parallel, high-throughput techniques based on “-omics” (genomics, transcriptomics, proteomics, etc.) have increased the comprehension of tumor biology with the discovery of biomarkers that have the potential to become therapeutic targets. These discoveries have paved the way for therapeutic platform-based vaccines, and the immuno-oncology landscape is clearly moving forward with this new type of cancer vaccine approach. These immunotherapies are designed to educate durable and specific immune responses against cancer cells and have demonstrated clinical benefit in patients while showing an excellent safety profile.
We can consider this scientific progress as the forerunner of a potentially groundbreaking new era in solid or hematologic cancer treatment.
Emerging technologies that further our ability to garner deep biological insights are enabling the infectious disease industry to identify pathogens early and accurately. Artificial intelligence and genomic sequencing tools, popular within oncology, helped fuel the development of liquid biopsies for cancer detection. Now, the infectious disease space is adapting these technologies to improve the diagnosis of pathogens causing infections, which is a key advancement that enables quicker diagnosis and early treatment, helps us avoid the use of unnecessary antibiotics, and limits the spread of infections that could turn into pandemics. This is particularly important for immunocompromised patients, such as those undergoing cancer treatment, since infections can delay treatment and jeopardize their health.
Emerging research on the connection between microbes and disease states ranging from oncology to autoimmune disease will also play a key role in increasing the industry’s knowledge to develop novel therapeutics.
On a macro level in the pharmaceutical industry, the demand for new innovative drugs and platforms will continue. There is no reason to believe that human beings are not going to want to live longer, be healthier, and look better. All of these elements of the human experience are directly tied to the pharmaceutical industry, and it continues to drive the aforementioned growth.
Key indicators such as the number of new drugs being approved by the U.S. FDA are also a good metric to evaluate growth in the industry. Over the last 5–7 years, this expansion has remained consistent, with around 45–50 new drugs being approved.
Another key indicator is the increase in the demand for the services supporting drug development to commercialization. Larger pharmaceutical companies have continued to cut their manufacturing capacity over the last 10–15 years, resulting in the growth of CDMO services. While our formulation development services can support all dosage forms, a major indicator for us to watch is the approved dosage form. Consistently, greater than 70% of compounds approved happen to be in the solid/semisolid or non-sterile dosage area — a domain where we are well positioned to provide CDMO services.
These indicators all predict the trends of the industry, and, if any of them start to shift, we will re-evaluate our practices and the overall strategy of the company. Currently, as a CDMO supporting early R&D, clinical material, commercial manufacturing, and regulatory submission, we are uniquely situated to meet the specific needs required by the industry.
Looking beyond the stock market, Lightspeed assesses dealmaking rates and premiums, as well as the number and focus of venture funds raised, to keep its finger on the pulse of the healthcare industry.
There is also an increased need for aging care and quality-of-life services for senior living, especially because we have a significant aging population globally in the developed countries. In the United States alone, 15,000 people a day are turning 65.
We continue to be upbeat and optimistic about the future of life sciences and healthcare. Our belief is that this industry will continue to grow as healthcare expands the many touchpoints it has with the patient’s everyday life. This represents an exciting business opportunity to provide daily care on a global scale.
The crux of it comes down to this: create it, service it, and pay for it. While we focus on developing therapeutics to help people live longer and healthier lives, we also need to look at supplementary services to help them access benefits and utilize them appropriately and to provide the financial tools to make it affordable. The future of our industry is going to grow exponentially because of expanding healthcare needs and because of our aging population. Sustaining quality of life and providing an ever-increasing need for services will drive our industry forward.
Over the last two years, the pandemic forced a reset to the current way of doing business within the pharma/biopharma industry. The unprecedented speed of vaccine and drug discovery, testing, and approval for the treatment of COVID-19 indicates the industry's mindset and appetite for high-risk ventures for the fail-fast paradigm.
The convergence of technology, biology, health, and large-scale multidisciplinary collaboration accelerated the discovery and development of life-altering solutions and enabled democratized access. This trend will continue to evolve, with data generation, access, mining, and analytics becoming foundational across the industry for efficient high-risk discovery and development success.
Companies producing high-quality data (on platforms with seamless integration of biology (multi-omics), clinical data, and metadata) will be at the leading edge of the novel, paradigm-changing discoveries supporting product pipelines within this fail-fast ecosystem.
Metabolomics, the study of small molecules (both exogenous and endogenous to humans), will take center stage for discoveries of targets, biomarkers, and diagnostics. Most small molecules impacting human biology are highly conserved across species, thus enabling seamless, accurate, and rapid translation of biological insights between human and non-human models. Metabolomic signatures within clinical chemistry reference ranges (and integration with genomics, transcriptomics, and proteomics) provide direct data connectivity between molecular biology and an individual's clinical phenotype. Metabolomics will become an essential tool for understanding disease and discovering health solutions.
The biopharma industry exists to make better medicines for more people and thrives on innovation. As such, I closely track activity in new subfields: how many new companies are operating in a space? How many assets within a new modality are moving into clinical trials? How are regulators adapting? How mature are enabling tools and technologies?
On the basis of those metrics, we expect that precision medicines will likely eclipse non-personalized therapeutic approaches. For the past five years, at least one-third of all new medicines approved by the U.S. FDA have been personalized medicines — showing that the industry has embraced the sequencing revolution to better understand complex, heterogeneous diseases, such as cancer. And, based on our own experience with next-generation single-cell analytics, we expect that personalized therapies will become the dominant approach to medicine in the coming decade.
Cell and gene therapies (CGTs) in particular will help fuel that growth. Today, there are hundreds of companies running thousands of clinical trials in targeted populations for these often curative drugs, and the FDA is rapidly expanding to support an expected 10–20 approvals per year by 2025.
Like all new modalities, these therapies come with a set of challenges. Demand is high among CGT developers for clear guidance from regulators and for better tools and strategies to characterize their products. The growing adoption of new technologies and approaches in this space is a leading indicator of the need for innovation in this space to deliver the promise of precision medicine more broadly.
One key indicator to predict the future of the biopharma industry, as it relates to ESG and the environment, is carbon emissions. What we’ve asked ourselves at PCI, in order to understand what practices we can implement to reduce our carbon footprint, is: “How will the planet look in 10 years if we continue business as usual?” and “What do we need to start doing to help keep global warming below 1.5%?” By answering these questions, we’ve set benchmarks and targets, such as reaching zero carbon emissions by 2050.
Additionally, it’s important to partner with organizations like EcoVadis that evaluate our ESG efforts and assist us with reaching key benchmarks. These organizations help us understand if the goals we’re setting are realistic and what additional benchmarks must be set to achieve more sustainability goals over the next 20–30 years. PCI was recently awarded Bronze Medal status with EcoVadis for our sustainability practices, with a corporate score of 54, a 70% increase from our previous assessment. This puts us among the top 32% of all companies assessed by EcoVadis. This is a great sign of the progress we have already made, and we’re only at the beginning of our ESG and sustainability journey.
From a data perspective, the long-term and post-acute care (LTPAC) community has been a virtual blackhole for decades. Research that has been conducted in this space has historically been dictated by information and datasets that represent an incomplete snapshot of this population. With the only electronic health record (EHR) dataset able to track an individual on a multiyear course, we’re in a unique position to help life sciences companies drastically improve the way they study the LTPAC population. By utilizing unique insights into data, technology for disease management, and unification of the life sciences community and LTPAC providers, we can dramatically improve the quality of care in the future. By identifying genetic markers with specific drugs and therapies, we can create a more targeted, individualized approach to care. It is through this approach to care, fueled by anonymized data, that we will drive healthcare change for the greater population.
We believe that the key indicator of our industry’s future is R&D productivity. Today, it requires more than $2 billion of R&D investment on average to bring a new medicine to patients. This statistic is driven by the approximately 90% failure rate of clinical trials. By actively utilizing AI/ML technology and wet lab experiments in an iterative loop, we hope to dramatically improve those figures. As we and other companies continue to advance new science, technology, and thinking, we expect to see the industry bring a wider array of great new medicines to patients faster and at lower cost.
In times of uncertain market conditions and accelerated technological advancements, peering into the future of biopharma is a challenging exercise. However, two important factors stand out as shaping this industry, particularly in the oncology space.
First, the demand for innovative oncology treatments, as well as the rising incidence of cancer, will keep shaping the biopharma industry for decades to come. The global oncology market in the United States is forecasted to grow at a compound annual growth rate of 6.7% between now and 2030, due to a combination of an aging population, increased awareness about cancer screenings, and advances in diagnostic testing. The advent of immunotherapy held the promise of transforming the cancer treatment landscape to achieve long-term remission or a potential “cure.” However, recent clinical setbacks in finding combination partners to realize the full potential of immune checkpoint inhibitors have shown that novel treatment approaches are clearly needed.
Second, in oncology R&D, we are seeing a trend where pipelines are becoming heavily concentrated toward a limited number of programs and targets that are assumed to have a high probability of success based on investors’ sentiments. This risk-averse mentality has the potential to create fierce competition in the field and absorb a significant portion of resources, thereby leaving less for other programs, and could potentially overshadow promising research programs to the detriment of smaller yet suffering patient populations.
Over the past several years, the industry has seen pharmaceutical companies leaning more on third-party technology, outsourcing partners, and contract manufacturing and development organizations (CDMOs) to enable faster development and scale-up of much-needed therapies. For example, the innovation and demand for COVID-19 vaccines facilitated increased interest, development, and uptake of other biologic-based therapies. This has led to a profound shift, with a greater recognition of the need for strong partnerships and platform technologies that can overcome the challenges of manufacturing, delivering, and administering biologics.
Our business model at TFF Pharmaceuticals includes a network of partnerships with fellow biotech companies who are developing innovative biologic-based therapies, as well as partnerships with a number of different CDMOs. An example is Catalent, who can offer our Thin Film Freezing technology to their customers. The progress and changes made during the pandemic have made it apparent that strong, transparent partner relationships will be critical to the future of biopharma to help optimize development and delivery of biologics and enable broader accessibility of these therapies to patients around the world, especially given that biologic modalities require complex storage and shipping (e.g., through the cold chain). As demand for biologics increases, we believe that those trends will continue, and we expect increased reliance on strategic partnerships to both optimize manufacturing and scale-up of drugs and facilitate continued progress of formulation technologies.
On the science and technology side, we review primary and patent literature and maintain close connections with key opinion leaders; this literature research can help us anticipate future client needs and ensure that our own proprietary technologies and processes remain on the cutting edge. On the business side, we consider six principal indicators. First, we track the availability of funds for research and early development, evidenced in stock valuations and in the availability of private equity and venture capital funding. Second, we assess government and legal actions that might influence pricing and the acceptance of pricing models. Third, we monitor drug approvals and progress in clinical studies, segmenting programs according to likely regulatory pathway (we help clients progress each of 505(b)(1), 505(b)(2), 505(j), and both over-the-counter (OTC) and direct-to-consumer (DTC) programs). Fourth, we refer to pharmaceutical sales data and sales projections, the latter especially by companies with recent or pending topical product approvals. Fifth, we assess immediately preceding growth rates and sales trends. Finally, we assess evolving data on the incidences and prevalences of indications suitable for treatment by topical or transdermal administration. As a contract research and early development (“R&D”) organization (CREDO), the continued expansion of our business is predicated on addressing needs in the industry and on the competitive positioning of our offerings.
Considering that this industry is centered on the prevention and treatment of illness and disease, we pay close attention to factors that influence need and demand for pharmaceuticals. For starters, health trends, such as an aging population, are very telling. We look at the science behind reoccurring pandemics. We consider treatments and therapies that address common chronic diseases and conditions that currently have no cure, such as diabetes and types of cancer. We work to understand the development of genomics and biopharma and monitor the number of biopharma or biosimilar drug applications.
As a specialty pharmaceutical ingredients distributor, one of our key competencies is our ability to support our customers by anticipating what may be around the corner that could impact their business, proactively managing uncertainties and assisting in identifying growth opportunities. A continuous feed of intelligence and insights managed through various functions and leaders across our organization ensures that we stay up to date, agile, and ready to meet the needs of our customers. Our purchasing and operations team looks at economic indicators, such as domestic production and import and export volumes of raw materials. Meanwhile, our product management team closely monitors shifts or fluctuations in supply, demand, and pricing of critical chemicals and raw materials. Our marketing department engages with market and industry insight resources that inform our strategies to address consumer and demographic trends. At the same time, our technical, R&D, and regulatory experts are on top of the new policies, drug filings, and health-related news published by government organizations such as the World Health Organization and the U.S. Food and Drug Administration. As an organization, we work together to synthesize all these data to make informed decisions, act on opportunities, plan for challenges before they impact and, most importantly, better understand our customers' and supplier partners' current and future needs.
When looking toward the future, there are several indicators that I look at to get a sense of which direction the industry will be heading in the next 5–10 years and beyond. The first is understanding where the unmet need is, as well as what the emerging trends are in the types of assets that companies seem to be interested in developing, and, in conjunction, which areas investors are supporting with their capital. For example, within the gene therapy space, viral vector–based gene therapy platforms have had success with some limitations, so there is interest in developing non-viral alternatives. At Xalud, we’re developing a non-viral plasmid DNA platform that has the potential to change the paradigm in chronic inflammatory diseases by creating a gene therapy for the masses and not just for rare diseases. We have a pipeline with broad potential for our proprietary platform, including several programs in development, and we look forward to being part of this new wave in gene therapy.
In addition, as someone who’s worked in operations for a long time, I’m also always looking at how companies choose to manage their day-to-day work and attract talent, as well as how that may change over time. This new hybrid environment has continued to advance rapidly in the last two years, and it’s exciting to imagine how it’ll evolve in the next decade. At Xalud, we embrace the flexibility of this new way of working and are deeply invested in ensuring that our team stays connected, collaborative, and productive.
Nice Insight, established in 2010, is the research division of That’s Nice, A Science Agency, providing data and analysis from proprietary annual surveys, custom primary qualitative and quantitative research as well as extensive secondary research. Current annual surveys include The Nice Insight Contract Development & Manufacturing (CDMO/CMO), Survey The Nice Insight Contract Research - Preclinical and Clinical (CRO) Survey, The Nice Insight Pharmaceutical Equipment Survey, and The Nice Insight Pharmaceutical Excipients Survey.
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