July 21, 2023 PAO-07-23-RT-02
A significant majority of our customers are from regulated industries producing drugs, manufacturing and reprocessing medical devices, cleaning healthcare instruments, performing laboratory analyses, and similar activities, all of which of course directly impact patients. The pandemic helped remind industries that cleaning must precede disinfection. While always a focus for Alconox Inc., an increased focus on patient-centric concerns reminds us all that what we manufacture, the quality and regulatory procedures we follow, and the product support we provide all end up affecting the safety of patients. As a vendor cleaning and impacting so many industries directly and indirectly interacting with patients, we have a responsibility to not just meet but exceed cGMP, internal QARA, and related regulations and expectations.
Several ways to interpret this question. Big picture, it has been influencing the way I work since the early 2000s, when there was a shift in funding organizations to pressure investigators to perform more “translational research.” Since then, there have been many advances in the field of personalized medicine, from pharmacogenomics to autologous cell therapies. Now, in my current position, my time is primarily spent supporting cell and gene therapy production, from R&D to manufacturing, as well as CMC, potency, and diagnostic assays. On the production side, with the increasing shift toward personalized medicine and autologous therapies, I find myself troubleshooting and designing processes that seek a balance between cost and process development time. Accommodating the inherent variability of donor and patient samples is driving the need to design workflows that are flexible, modular, and fast. The potency and diagnostic assays side of things is more interesting. Especially in the wake of the FDA Modernization Act 2.0, I am engaging with enormous creativity in cell-based assays, with end users seeking more complex methods, like 3D cell culture models, to better predict patient-specific responses to therapies while lowering costs and increasing throughput.
One-size-fit-all solutions in drug development are problematic as many of the current drugs are extremely toxic — significantly affecting patient quality of life (e.g., chemotherapy). Precision medicine seeks to develop personalized drugs, which optimize the individual patient outcomes by delivering more efficient and less toxic compounds.
However, some of the most lethal diseases, such as cancer, are extremely diverse. There are many mechanisms that can trigger cancer of a certain type, depending on the patient’s genetic background and health circumstances. At Envisagenics, we are focused on developing therapies based on RNA splicing errors versus genetic mutations, allowing us to vastly increase our target search space and stratify patient cohorts at much deeper levels to find medicines that are more likely to work for more targeted patient populations.
We study the spliceosome to develop personalized therapies. The spliceosome is the largest and most dynamic biological complex in cells, and as such, it is prone to failure. Spliceosomal failure causes splicing errors that result in the expression of neoantigens that we leverage for drug targets. By stratifying patients based on spliceosomal profiling, we can identify drug targets prevalent in patient subpopulations and then develop immunotherapies that are effective in 15–80% of all of patient populations.
Offshoring to low-cost countries, especially for generic medicines, has forced Western pharmaceutical manufacturers and service providers to seek higher value-added platforms to maintain competitiveness. One such area is patient compliance to ensure a patient is complying with the prescribed frequency for dosing. Patient compliance is a common problem for most of us who take various medicines. It is a bigger concern for pediatric and geriatric patients who may not keep track of their daily dosing regimen or others. These populations have made us move away from bulk packaging of bottles. We are now focusing on unit dose cups and other specialty packaging configurations. A specific number of tablets or capsules in a sachet may improve compliance for solids. Unit dose cups offer a better way to control and record what was administered to a patient for liquids. Patient-friendly packaging is one result of the patient-centric focus in our industry. The increased focus on patient-centric concerns encourages the development of AI technologies that prioritize patient well-being, autonomy, and engagement. By incorporating these principles, AI can be valuable in delivering more patient-centered and personalized healthcare experiences.
We’re asking some additional questions now. There has always been significant interest in understanding how our hydrolysates have an impact upstream but understanding the impact later in the process is something we’re keen to understand. We want to make sure we’re not adding risk to the patient or the other processes leading up to the patient. One item being discussed in a consortium we’re part of is the aggregation of endotoxin in the culture media and its impacts elsewhere in the process. Also, understanding any how any processing aids we use may impact downstream purification. If Nu-Tek is lucky enough to be included in a customer media formulation, we want to make sure we’re ticking all the boxes, so there’s no unnecessary risk to the patient. By asking the right questions and being transparent, we’ll be prepared to adjust to decrease or eliminate risks entirely.
While our industry has always been patient focused, we are seeing a greater trend to really drive toward this goal. This evolution is impacting how we develop products. There is a great emphasis on making treatments more tolerable for patients and designing them to better support patient compliance. This has resulted in a dramatic increase in the use of auto-injectors and prefilled syringes with needle safety devices. These forms make drug delivery easier and increase patient compliance by putting the technology in the patient’s hands and avoids the need to go to a clinic or doctor’s office. Meeting these growing needs, PCI has invested significantly in equipment/capacity to deliver these innovative auto-injector/pen solutions. Our team of packaging design experts work closely with our clients to develop and design patient centric packaging to deliver true value to our clients and meet their patient’s need.
Our goal is to improve patients’ lives through better health outcomes. That begins with really understanding the needs of the patient. Increasingly those patients are all around the world, some in highly developed countries and certainly some in less developed countries. We seek to understand the way products are intended to be administered, risks, compliance, and adherence barriers, such as potent side effects and comorbidities, relative literacy levels and patient comprehension, opportunities for disease state education, and the supply chain and delivery mechanisms, among other considerations. These opportunities allow us to really optimize the delivery system to the patient, not only in the drug delivery device but more broadly in a systematic approach to how drug is delivered, whether that is clinically or through self-administration.
Involving the voice of the patient in the protocol and the related decentralized clinical trial (DCT) strategy is key to improving the overall patient experience and increasing patient access. Patients will carry a varying degree of interest in participating in the research remotely; some patients, depending on their disease and past experience, will always opt to be in front of their medical team for every visit, so moving to an entirely virtual setting on a clinical study is not always the right move.
At PPD, we have developed a model that looks at the visit schedule, associated assessments and procedures, patient support needs, and their disease story to predict a patients’ willingness to participate in a trial. This calculation is used to determine if the protocol could be optimized to increase patient participation rates by reducing patient burden. Understanding where flexibility/optionality for patients will be most meaningful can make a difference in overall study compliance and retention.
Additionally, effectively managing the increased complexity brought in by adding more modern and decentralized strategies is key. Installing an application on a patient’s smartphone/device or adding a wearable to the study to enable remote data collection alone does nothing to improve engagement if the app or device is burdensome or glitchy. Successfully reducing patient burden with these modern study strategies depends on both ease of use and a robust operational oversight model. It’s important to find a partner who empowers continuous innovation and successful execution of novel site and patient strategies.
Working at a contract development and manufacturing organization servicing more than 110 global partners, our team’s utmost pride lies in our patient-centric approach, placing their well-being at the forefront of our operations. We are driven by our commitment to delivering innovative solutions and services, accelerating the speed to market and strengthening the reliability of our supply chain for new therapeutic products.
We firmly believe that transparency plays a pivotal role in minimizing potential safety risks for patients. By leveraging our advanced capabilities, we proactively eliminate any potential sources of human error, ensuring both accessibility and the highest level of safety for patients who rely on the products we manufacture.
Imagine your scientific breakthrough earns the FDA’s stamp of approval, but your advanced therapy is then delayed in being administered to patients while payers debate cost. Like it or not, the patients you strive to help are thinking about the price tag. That’s why innovators need to consider any and every way to lower their cost of goods sold (COGS). The earlier in the development process you do this, the better. For example, decisions involving single-use systems and consumables, resins, column packing, batch size and productivity, and process optimization can combine to drastically lower your COGS, ultimately benefiting patients.
A patient-centric approach to medicine will likely increase the need for development and manufacture of biologics based on less common protein variants than those on the market today. This in turn will increase the value of next-generation technologies for recombinant protein production, including Vectron Biosolutions' platform technologies that enable robust production of tricky proteins and that reduce the overall COGs of protein manufacture.
Patient centricity is at the heart of decentralized clinical trials (DCTs). Our industry saw a rise in DCTs, fueled by innovation in data collection modalities and the pandemic forcing decentralization. We’ve also seen it discussed that DCTs can be difficult to operationalize, and a concern that our industry tends to fall back on established methods and norms. Ultimately, DCTs attempt to reduce patient burden and increase patient access to trials. In general, DCTs solve more problems than they cause, from aiding in enrollment to increasing diversity in patient populations. However, DCTs also compound existing data problems, which are only getting more and more complex. Life sciences companies are focused on cycle time improvements, and, within this complex landscape, expectations to gain insights using advanced analytics are also increasing. This combined focus on innovation, cycle time reduction, and patients means that addressing fragmentation within the overall data life cycle has become critically important. Demand for connected data and insights means that life sciences is increasing their attention on advanced infrastructure and analytics for clinical data. As DCT data collection broadens to include more new devices and apps, not only do pharma and biotech need the ability to bring these sources together for faster access and decision-making, but their digital data infrastructure must also address patient centricity within the analytics and data consumption components of the clinical data life cycle. This means data availability for advanced applications like AI/ML and data connectivity to enable collaboration between all data stakeholders, from sponsors to sites to patients.
For all of us who touch the pharma space, patients are at the center of the work we do. Now more than ever, patients are looking to engage in their healthcare, increasing their expectations of the information providers share with them and utilizing various types of tracking devices to have better direct insight into and management of their own health. With this proliferation of data available from different sources, we’re seeing more opportunities in the pharma marketing space to analyze this data to better inform the type of information shared with providers to ultimately enhance the patient care delivered.
While patients enjoy the additional health insights offered through wearables and other devices, there are increasing patient concerns on the privacy of that data though. Therefore, a critical first step for pharma and healthcare companies with access to patient data is to anonymize it and then analyze the de-identified data. If done responsibly to protect identities, harnessing this data has the power to help HCPs inform treatments and for pharma to use in the drug development life cycle, and both of these things at the end of the day serve to improve patient outcomes in the future, which is a step towards the greater good.
At Faeth, our emphasis on patient centricity directly shapes our research and treatment development approaches. We’re fundamentally driven by the need to make our precision nutrition treatments for cancer patients not only more effective, but also accessible to all patients, regardless of their geographical or economic circumstances. Recognizing that traditional clinical trials can be taxing and inaccessible to many, we have partnered with decentralized clinical trial providers to enable remote access to our trials. By bringing the trial to the patient, we’re able to eliminate many burdens of participation, making it easier for diverse patient populations to contribute to and benefit from our research.
Our commitment to patient centricity extends to personalized meal plans, created with the help of our science-driven chefs and registered dieticians, and delivered to patient homes at no cost. We also offer a support app that patients can use to foster a community with others in the trial, and to receive assistance from a doctor or registered dietician during their treatment journey. This dedication to patient well-being ensures we do more than treat a disease — we provide comprehensive care that acknowledges and addresses the complex realities of our patients’ lives.
Personalized approaches to treating cancer have extended the lives of many patients over the past decade, with more promising advances on the horizon. This has led to a rapid increase in the number of precision therapies, including modified cell and gene therapies, available to cancer patients.
But the experience of patients remains difficult, in part because those undergoing treatment for cancer often have more questions than doctors can answer: What’s the best treatment for me and my specific cancer? How well did my last treatment work, and will my cancer be back?
Answering these questions increasingly requires higher-resolution diagnostic modalities, and it’s one of the forces driving Mission Bio. Our technology has become a crucial tool for understanding how cancer subtypes resist treatment and how a patient’s specific disease is responding.
Personalized therapies are only as good as the technology used to identify the right targets in the right patients. We’ve realized that with our Tapestri single-cell multi-omics platform, it’s possible to track disease burden and potential therapeutic targets with a single-cell resolution in the same assay. Helping patients find the right treatment is our preeminent goal.
We’re making progress as a field in addressing questions in a useful way for patients. Measurable residual disease (MRD), a way of testing for risk of disease recurrence, is already standard for certain cancers but gives no insight on the proper treatment. Our Tapestri platform can take the next step, querying treatment-relevant targets on a cell-by-cell basis to give patients concrete options for treatment.
ScaleReady’s core values are built upon our founding partners’ patient-centric focus. As we interface with all stakeholders in cell and gene therapy, we reserve particular attention for the patients and their families. They are fighting for their lives and a hope for something better. We pride ourselves on having an organization where each individual is dedicated to working with the urgency and tenacity they deserve.
We recognize that time is precious and often running out for the patients who most benefit from cell and gene therapies. The same is true for our client base, the cell therapy drug developers, both directly and indirectly: they are driven by the same urgency to reach patients quickly. But they are also faced with the economic reality faced by many biotechs, as almost all are pre-revenue, with a lot of work that needs to be done to reach the clinic — and money is always running out.
Their success is critical for patients, and drives us every day to support them. Despite the demonstrated curative power of cell therapies, our field is only averaging about one new approved T cell drug per year since the initial approvals in 2017. Especially given the recent wave of industry layoffs and inability to raise more money, we are determined to help our partners focus on simplifying and standardizing, so we can successfully streamline and scale manufacturing processes across the globe. This is the only way to ensure these incredible treatments reach all of the patients in need.
Catalent continues to deepen its understanding of the impact that drug product design decisions have on patient outcomes, including patient usability. This awareness is incorporated into the product development work we undertake with our customers, potentially by enhancing molecule design, formulations, and dose form suitability for a specific indication and patient population. We also proactively identify unmet patient needs in targeted indications that could be addressed through enhanced drug product design and bring those ideas to innovators developing related treatments. These insights can also be applied to the development of consumer-preferred forms for over-the-counter and consumer health products.
Our patient focus extends beyond development, as we continue to work diligently to reliably supply drugs, vaccines, gene and cell therapies, and consumer health products that are safe and effective for customers and patients around the world. As with all our operations throughout the organization, our “Patient First” core values guide us.
Customer centricity has long been the core of our focus at Thermo Fisher Scientific. However, the combination of environmental factors like digital innovation, accessibility, and an overall increase in patient engagement across systems has changed the way we think about clinical trials. The critical areas of our focus have been around trial accessibility, speed, flexibility, and quality — all spaces where baseline expectations are evolving and where patients could be more involved in their own treatment. And lastly, embracing that engagement and co-creating to drive meaningful innovation.
One example is decentralized approaches to clinical trials, imaginably improving the overall trial experience and increasing opportunities to access a wider range of patients. In this space, we offer our customers services that support a broad range of trial types supported at the patient's home. This could make participating in a clinical trial sponsored by our customers easier for patients. We also now have capabilities to support different clinical trial protocols that enable success without patients traveling to hospitals, potentially making it easier on patients, their families, and caregivers. Decentralized clinical trials could also streamline operations and make collection more efficient. At each step of a clinical trial, we are focused on reimagining the patient experience and are creating new services and ways of working to optimize the patient experience for our clients.
Another key shift when thinking about the patient experience we offer our clients is thinking about their expectations in today’s digital era. Speed, flexibility, and data visibility are a must. Access to data and information should be easy for patients. We are accelerating our digital strategy, including investing in digital transformation that connects our physical and digital supply chain to enable real-time visibility and decision-making — this is a must in today’s world. We’re also looking at how data can further optimize patient experience and optimize clinical trial outcomes with adherence measurement smart packaging solutions and more.
How do we keep up with clinical trial patient centricity and the ever-changing world? The answer is co-creation. When we identify a challenge, problem, or opportunity and have a concept –– we have trusted partnerships with several of our customers that we rely on for direct VoC to ensure our innovation is meaningful. This partnership helps ensure we are optimizing clinical trials with patient-centricity front and center.
Patient-centric care is a driver for decentralized clinical trials. Decentralized trials are those in which many of the patient visits or tests can be done at home or in local clinics, as opposed to a central hospital location. Rather than a patient coming to the trial, the trial goes to the patient. This model is more convenient for patients and gives sponsors a better understanding of the patient.
Decentralization also fosters greater diversity of inclusion by making trials more accessible to a broader population. When paired with community outreach in underrepresented areas, decentralized trials can be fully representative of the patient populations targeted by a therapy. This representation is critical, not only for fairness but also for the integrity of the treatment response data that informs a drug’s approval and labeling.
Trials that involve imaging are the most difficult to decentralize because nobody has a CT scanner in their living room. However, it is possible to equip community hospitals and local imaging centers with the skills and technology needed to participate in clinical trials. In this way, imaging-based trials can decentralize too.
Our game-changing technology is making this decentralization a reality. Our clinical trial imaging management platform makes it easy for organizations and imaging centers to become certified trial sites. This helps to decentralize imaging-based clinical trials and makes them more accessible to patients who can benefit. We’ve shown that with increased accessibility comes more efficient trial recruitment and increased representation, which gives patients, trial sponsors, and regulators alike confidence in the outcomes.
Patient preference has been a substantial force in shaping the biopharmaceutical market for years. One clear example of this is the preference for oral medicines, since the majority of biologic therapies have to be administered via subcutaneous injection or intravenously. These injections can be extremely difficult and painful to self-administer with burdensome dosing schedules, so it’s no surprise that a majority of patients would prefer an oral alternative.
That is what motivates us at Rani Therapeutics. We’ve conducted studies to understand just how powerful that preference really is. Even among patients whose injection regimens are as infrequent as twice a year, 76% would prefer to take a daily pill.
Putting patient preference first is also a demonstrated growth and revenue driver. When Johnson & Johnson introduced their subcutaneous formation for Darzalex, 85% of patients converted from IV administration, and sales increased by 52%. Similarly, with Rybelsus, an oral formulation of semaglutide, sales more than doubled in 2022, and Novo Nordisk boosted their sales expectations by over 10% for 2023.
This patient-centric mission is what has driven us from day one at Rani, because it’s not just a business decision — our goal is to offer patients a better way to get treated for their conditions. The RaniPill is designed to offer patients an oral alternative to injections by delivering biologics painlessly via a microinjection to the small intestine. This is a platform technology, meaning we have the unique opportunity to help millions of patients across a variety of therapeutic areas.
Rune’s goal is to improve the care of people with Parkinson’s today, so disease-modifying therapeutics can be developed in the future. This people-centric approach to Parkinson’s disease innovation is integral to who we are; we’re proud to be one of the few companies with a patient advisory board. Our first product, StrivePD, collects and analyzes a continuous stream of objective, quantifiable data from Parkinson’s patients via Apple Watch, so their clinician can make informed care decisions. Equipped with the knowledge of how symptoms change throughout the day, doctors can personalize care and tailor treatment regimens to each patient’s specific symptoms. Because no two people with Parkinson’s have similar symptoms, we’ve worked closely with the Parkinson’s community to ensure that StrivePD can accurately make sense of movement data. Through our ongoing interactions with the broader Parkinson’s community, Rune Labs is able to adapt the design and features of StrivePD to fit the needs patients identify in their own life, and as those needs change. As Parkinson’s disease progresses, symptoms and patient needs can vary significantly. By centering the input of people with Parkinson’s during the years-long course of development and as their symptoms change, Rune Labs has created a user-friendly interface, not just for clinicians but for the patients as well. This dedication is also reflected in our wrap-around clinical trial platform, StriveStudy. In addition to enabling remote patient monitoring, StriveStudy is intended to improve the clinical trial experience for people with Parkinson’s by removing the burden of self-reporting and exhaustive questionnaires.
At Sherlock, a focus on helping people is truly baked into our DNA. Our foundational technology was developed by Jim Collins and David Walt, synthetic biology luminaries from Harvard University’s Wyss Institute who have been leveraging CRISPR and other cutting-edge technologies to make diagnostics more accurate and affordable, easier to use, and useful in more environments and by more people.
For us, putting people at the center of what we do means understanding who our customer is and designing our diagnostics with them in mind. That drives Sherlock’s commitment to the “three A’s”: affordability, accuracy, and accessibility. The pandemic showed just how large the appetite is for pushing more testing into the home — not just COVID-19, but for a wide range of diseases — and we believe that achieving this will break down barriers to access that have driven inequities in healthcare for too long. The world needs more flexibility in where and how testing can occur to control the spread of disease, and Sherlock is at the forefront of meeting this need.
The patient is always the focal point of any work we do in medicine. At Karius, it’s in our culture — our co-founders were inspired to build our technology while trying to save the life of a child with an undiagnosable infection. Having the clarity of vision — that our goal is to improve patient care and save lives — sets the tone for everything we do.
We saw a huge unmet need to help patients who are immunocompromised. They are at high risk from getting infections, accounting for 3 million hospital admissions each year, and can be challenging to diagnose, resulting in high mortality rates. Many of these are people struggling with cancer, where infection is unfortunately the primary or secondary cause for half of all deaths.
For us, that meant developing a quick test that might get answers to more patients faster, without a week-long battery of trial-and-error testing from standard methods. This was the vision for the Karius Test, a single blood test that non-invasively detects over a thousand pathogens in two days.
In listening to patient concerns, it becomes clear that answers are key to their peace of mind — and if we are aiming to treat the patient instead of just the disease, our approach must include providing better information, with context, so that care teams can quickly and confidently tailor the most specific course of treatment possible for their patients.
Cellares’ mission is inherently patient-centric: accelerating access to lifesaving cell therapies. Cell therapies are personalized medicines created from a patient’s own cells, reprogrammed to boost their cancer-killing capabilities. But while the therapies themselves are highly effective, the manufacturing processes used today create manufacturing bottlenecks, which limit availability of these potentially curative treatments to patients. A 2022 study found that about 40% of multiple myeloma patients eligible for CAR-T waited a year to receive treatment, and more than one-fourth of patients died during that wait.
The root of the problem lies in the personalized nature of cell therapies. Each dose is made for an individual patient in a complex process with many manual manufacturing steps. To make 10,000 patient doses, this manufacturing process needs to be repeated 10,000 times, which results in the inability of pharma companies to meet patient demand, high manufacturing costs, and high process failure rates. Patients often aren’t able to wait months for treatment, and if a manufacturing error ruins a dose, they might not be healthy enough for another round of apheresis.
With our automated and high-throughput solution, the Cell Shuttle, cell therapy manufacturing becomes scalable, cost-effective, and reliable. With cell therapies being approved as earlier lines of treatment and thousands of additional therapies in clinical trials, it is clear that we urgently need a technology to scale manufacturing to treat tens of thousands more patients than is possible with current paradigms. With the Cell Shuttle, Cellares is accelerating access to lifesaving cell therapies.
Advanced therapies are a relatively young space but have already provided one-and-done functional cures for patients that had run out of options. When I think about how best to serve patients, I focus on several places where we can respond directly to their needs, both directly and indirectly.
Because our job is to make sure the next generation of cell and gene therapies reaches patients, we think about how to shorten their development cycle — it already takes a decade or more to bring a new drug to market, and medicines are getting ever more complex. We also look to reduce costs for development and manufacturing, which will be reflected downstream in both costs to patients and access to the best new therapies. It is also crucial to reduce toxicity and immunogenic responses and to improve the purity of both in vivo and ex vivo gene therapies, and these remain top priorities throughout the manufacturing process.
VintaBio was founded by pioneers in the space who developed the clinical-grade vectors for manufacturing the first FDA-approved gene and cell therapies. Everything we do is geared to making sure that great ideas don’t die on the vine, because a cure that never reaches patients is an unthinkable waste.
As an EPC and consulting firm, we are somewhat removed from the direct patient experience, but we find ways to keep patients at the heart of everything we do.
The speed at which this field is evolving is staggering, and it has broad implications for how life science innovators design, build, and operate a new generation of research and commercial facilities focused on personalized medicine. Rather than churning out repeatable formulations to meet the needs of a large population, these facilities will focus on the aseptic production of many very small, personalized batches — sometimes as small as a single dose. To make these facilities work both financially and in terms of GMP standards, project owners and their EPC partners must rethink their process design, equipment selection, cleaning, and decontamination protocols — everything.
Even the question of where to locate these facilities is complex; manufacturers need to consider their access to a transportation network capable of moving small, time-sensitive doses from the life science facility to the patient’s bedside. Add the logistics of finding a qualified workforce, managing waste, establishing adequate utilities, and many other site-specific details, and the challenge of identifying a suitable site becomes immense. All-new design solutions will play a key role in solving this challenge.
Another hurdle to patient centricity is the availability and affordability of therapies. An important impact that we can have on making therapies available to patients is delivering facilities that are more flexible, consume less energy, and replace repeat manual labor with automated solutions to reduce the risk of human error and lower the cost of goods.
In advanced therapies, especially autologous cell therapies, the patient comes to the center of the whole manufacturing effort.
For non-personalized products, if you reject a batch, or you exceed the temperature limits during transport, it is an issue: a financial loss and a potential shortage of the product. But advanced therapies are based on human cells, and today’s cell therapies are typically reserved for very sick patients who have failed all other treatments. If you fail to manufacture an autologous product, you take away a rare opportunity for a specific patient — they may have exactly one try for a treatment that could prolong or even save their life. Even though we do not know the patient personally, we feel we should do all we can to return the product to them.
With this in mind, we put extra effort into making all the processes seamless, from the blood collection through the infusion back into the patient. Even slight errors may result in failure to deliver a treatment back to someone in desperate need.
As a Qualified Person — the EU designation for the person responsible for final certification of a product — I feel even more urgency. Making sure that as many patients as possible can get autologous therapies requires close cooperation with the treating physician and the regulator to make it happen, especially in more complicated cases. And at all times, we need to put the patient's benefit over the company's KPIs.
I’ve had the privilege of working in healthcare for 30+ years, and it’s been great to see how medicine has shifted from being paternalistic to being more sensitive to the patient perspective.
One important part of this shift is recognizing the stigma that is often associated with diseases such as alcohol-associated hepatitis (AH). At DURECT, one conscious change that we’ve made is the terminology that we use to describe AH. We're working with the liver community to minimize the use of biased/pejorative language. Using the term “alcohol-associated hepatitis” instead of “alcoholic hepatitis” mirrors other changes such as the shift from “alcohol abuse” to “alcohol use disorder.” We’re committed to staying up to date with the latest professional society guidance in the liver space.
In addition, we have focused our work on AH because of its acute nature, high short-term mortality, and lack of approved therapies. A startling 30% of severe AH patients die of this potentially reversible disease within 90 days of hospitalization. We are conducting a phase IIb trial in patients with severe AH to evaluate the ability of our lead compound, larsucosterol, to reduce 90-day mortality and the need for rescue therapy by liver transplantation. Developing therapies that can improve these outcomes is critical in AH, so we are prioritizing outcomes that have the biggest impact on these patients.
I look forward to seeing patient-centric care continue to evolve as we all work together to improve the healthcare experience for patients.
Our industry has placed a renewed focus on patient-centric concerns and values in recent years. This shift in focus has transformed the way that we work, making us more dedicated than ever to improving the lives of those with the greatest needs.
At Chiesi, we are in a unique position as a family business and as a certified benefit corporation, which allows us to focus on the long-term support of rare disease patients and their communities. We believe there is a societal obligation to address rare diseases and are committed to ensuring that patients are getting the best possible care, which is why we are continually adapting our strategies to meet their needs. We work in close partnership with patients, caregivers, medical, educational, and governmental entities to ensure regulatory and environmental payer practices are beneficial to the patients we serve.
We recognize that patients with rare diseases require a holistic approach to care. For this reason, we have partnered with a range of international and regional rare disease advocacy groups to co-develop support programs that provide patients and caregivers with emotional support, educational resources, and access to specialized healthcare providers. By offering comprehensive care, we can ensure that our patients receive the support they need to manage their condition effectively.
We also collaborate with government institutions and all stakeholders to ensure expeditious access to therapy for patients. We are engaging in health economic studies and a continual evaluation of the societal burden of rare diseases. As a proponent of shared responsibility with national healthcare systems, the ability of patients to manage healthcare costs is vital. Through our Chiesi Total Care program, we work with healthcare systems to ensure that our treatments are accessible so patients can receive the care they need.
Additionally, we make it a focus to involve patients and their families in the drug development process from the earliest stages of research and development. Our patient advocacy team has established patient advisory boards across our pipeline to ensure inclusion of patient feedback in all stages and commercialization of therapies. We believe this is imperative. By collaborating with patients at the onset, we gain an even deeper understanding of the way they are affected by their rare disease and develop treatments that are tailored to their unique experiences. This approach allows us to prioritize patient outcomes and create medicines that are designed to meet their specific needs, rather than simply creating a drug in a “one size fits all” approach.
It is our mission to generate innovation and value by putting the patient first. With this approach, we believe that we can make a positive impact on their lives and help them achieve better health outcomes. We remain committed to developing and commercializing products that meet the needs of patients around the world.
Patients have always been at the center of our approach to drug development at Ashvattha as we explore ways to improve treatment outcomes and the patient experience. Findings from market research in wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME) validate that both patients and the retinal specialists who treat them are keenly interested in a safe and effective treatment alternative to repeat intravitreal injections. For patients, the prospect of avoiding injections into the eye which are uncomfortable, cause anxiety, and require burdensome office visits is very appealing. Physicians are also enthusiastic about a treatment that could potentially improve patient adherence and reduce the heavy burden on their practice with current in-office injections. These insights informed our approach to developing a less invasive treatment option that a patient can self-administer orally or subcutaneously on their own schedule and in the comfort of their home, potentially reducing the burden of treatment for both patient and treating physician. We developed our nanomedicine therapeutic (D-4517.2) using a technology that enables it to cross tissue barriers, including the blood–retina barrier. Furthermore, multiple preclinical studies have shown that this agent is able to selectively target activated cells involved in wet AMD and DME. We are currently conducting a phase II clinical study of D4517.2 in wet AMD and DME patients with a goal of demonstrating positive clinical outcomes as well as an improved patient treatment experience.
Medical training reinforces the importance of the patient voice. It emphasizes how active listening is necessary in order to capture the full clinical picture. This experience has instilled in me a resolution to always remember to seek the patient’s perspective, whether it is at the bedside or working in drug development in the biotech industry. In recent years, patient-centric concerns have taken center stage, and shared decision-making has become increasingly relevant when it comes to clinical trial design and therapeutic launches.
Patients have the opportunity to play a key role in the development of potential treatments by serving on advisory committees and development meetings and providing feedback on guidance documents. As chief medical officer, reviewing these data serves as a foundation for research and development plans that will deliver evidence that includes impact on health that has direct relevance for patients. We work closely with patient advocacy groups to hear the real concerns about access and convenience and factor these into our strategic vision.
Creating an open, welcoming environment for patients is also crucial to be able to hear directly from them about their views and needs. We recently unveiled art inspired by patients with hypoparathyroidism and local artists in Boston, Massachusetts, and Lyon, France, to provide an open forum for the community to come together and discuss the challenges of living with this disease and what is needed for patients to experience an improved quality of life.
Patients are the catalyst for drug development at Ambrx. Their needs inform and inspire our efforts.
Ambrx has developed platform technology that allows for highly-stable, site-specific antibody–drug conjugation (ADC) that prevents premature release of cancer-killing toxic payloads. As a result, our product candidates are precision-engineered to be more potent, safe, and tolerable for patients compared with other ADCs.
The advances we are pioneering at Ambrx come at a critical time in the ongoing battle against cancer. While overall cancer mortality rates continue to decline, future progress may be slowed by stubborn increases in incidence of certain cancers. Among those still on the rise are breast and prostate cancers.
A closer look at prostate cancer reveals that its incidence increased by 3% from 2014 through 2019, after two decades of decline. This translated to an additional 99,000 new cases. A commonly-used treatment for metastatic castration-resistant prostate cancer (mCRPC) is a radiopharmaceutical. This approach has shown effectiveness in reducing tumors. However, exposure to radiation is a significant safety and quality of life issue. Some patients must isolate for up to 15 days from loved ones. Time is their most precious commodity. We must aspire to do better.
Ambrx is committed to accelerating the development of new, safe, and effective treatment options that minimize debilitating side effects, optimize outcomes and maximize quality of life. We believe patients should not be forced to choose between effectiveness and safety. As such, we are determined to evolve the treatment paradigm beyond such trade-offs.
At AltruBio, our work is significantly influenced by an increased focus on patient-centric concerns. We are dedicated to improving the lives of patients with autoimmune and immune-mediated inflammatory diseases by developing safer and more durable treatments. Unlike many existing therapies that merely address symptoms or block immune responses, we focus on the underlying mechanisms behind these conditions, particularly the inflammatory cells driving them. By targeting these cells, our aim is to enhance patient quality of life by targeting the disease source without systemic immune suppression. The development of our potent and subcutaneously administered immune checkpoint enhancer (ICE), ALTB-268, aligns with our patient-centric approach and has shown sustained and durable remissions compared with existing therapies. Supported by the clinical validation of ALTB-168, we have evidence of its safety, tolerability, and efficacy across multiple indications. By enhancing its potency in eliminating chronic pathogenic T cells, we anticipate achieving equivalent efficacy to ALTB-168 as a subcutaneous form to allow patient self-administration without the burden of going to a hospital and/or clinic, thereby making ALTB-268 a more patient-friendly option. In addition to our product development efforts, we recognize the importance of optimizing clinical trial strategies to maximize therapeutic benefits, especially for specific patient populations. Therefore, we are actively conducting biomarker research to gain valuable insights that will inform treatment regimens and ultimately improve outcomes. Our unwavering commitment to patient-centric concerns shapes the way we work. By developing treatments that target the root causes of diseases and prioritizing patients' convenience in drug administration, we aim to make a profound impact on their lives.
Alto is committed to transforming the diagnosis and treatment of mental health conditions, and the patient is always central to this work. We recognize that the CNS has proven to be a particularly challenging area for drug development and believe conducting trials more thoughtfully and efficiently can maximize impact for patients. We understand that the patient perspective is often different from an observer or an objective measure (like a biomarker), and so we collect multiple patient-reported outcomes to understand the patient perspective in our trials. Our team is working to incorporate real-world data in our trial design and execution, build knowledge iteratively by using earlier stage trials to de-risk development, and stratify patients using machine learning and AI practices.
As part of our clinical trial innovation, we’ve launched fully remote studies to alleviate patient burden. These trials bring research studies to patients and allow more people to be involved in innovative research, particularly those across diverse populations and geographies who may not have the time or resources to reach a physical site. We are exploring ways of explaining the biomarkers to patients so that they are able to take ownership of this information. We also hope to democratize access to diagnostic tests that identify the most effective treatment for a person, making them available to patients at little or no cost in the comfort of their homes.
As an oncologist, waiting with patients who’d undergone chemotherapy and surgery for mKRAS-driven tumors to see if they’d relapse left me feeling helpless — I wanted to do more than wait and see. Patients with mKRAS pancreatic ductal adenocarcinoma and colorectal cancer who have minimal residual disease following standard treatment have very high relapse and recurrence rates but no treatment options during the monitoring period.
At Elicio, these patients are our primary focus, which is why we are exploring ways we can improve outcomes in the “wait and see” period. We have developed an investigational cancer vaccine using our proprietary platform that is designed to deliver immunotherapies directly to the lymph nodes, the “brain center” of the immune system, to elicit a robust immune response. We are evaluating our vaccine candidate in the monitoring window for patients with mKRAS-driven tumors at high risk for relapse due to minimal residual disease following standard treatment. Interim phase I data presented at ASCO show that our immunotherapy is safe and tolerable, able to reduce tumor biomarkers, and results in a strong immune response. In addition, most other KRAS-targeted therapeutics in development — particularly small molecule KRAS inhibitors — target only a couple of mutations, potentially limiting the number of patients that can be treated. Elicio is developing an immunotherapy that targets the seven KRAS mutations that drive 25% of all solid tumors, potentially defeating resistance mechanisms.
Having a patient-centric approach is critical for every company but is especially important for those working in the rare disease space. Our lead indication is telomere biology disorders (TBDs), which are a set of rare genetic diseases caused by shortened telomeres in all cells with particularly devastating effects in hematopoietic stem cells.
Patients living with rare diseases are often the most vulnerable because there may not be any treatments available, or, if they are available, they come with a significant cost and/or adverse effects. The leading cause of mortality for TBDs is bone marrow failure, with most patients suffering from it by age 30. Currently, there is no cure available except a bone marrow transplant, a complex procedure that often requires a harmful conditioning regimen. Our lead candidate, EXG-34217, has the potential to make a real difference for these patients, with respect to both safety and efficacy.
When these patients work with us in the clinical trial setting, we want to make sure that we make the process as seamless and comfortable as possible for them. For example, we take care of all travel and lodging so that patients don’t have to worry about the cost of those things. In addition, we’re working with one of the leaders in this space, Cincinnati Children’s Hospital, where we know patients will get great care from start to finish.
We’re committed to continuing to do this important work and partnering with patients throughout the whole process.
Ethris is committed to developing respiratory medicines for lung diseases with high unmet needs, placing patients at the center of our research and development endeavors.
Our dedication to patient-centric care extends beyond therapy development. We strive to ensure that the voices and experiences of patients are heard and valued throughout the entire drug development process.
In the case of diseases lacking approved therapies, it is crucial for us to thoroughly understand the experiences of patients, ensuring that our therapy effectively addresses their needs and alleviates their symptoms. One approach to achieve this is by conducting a natural history study to gain deeper insights into patients suffering from these conditions and align our development efforts with their needs. For instance, early next year, we plan to initiate a natural history study involving patients with primary ciliary dyskinesia (PCD), a serious rare lung disease currently lacking approved therapies. We are dedicated to addressing this condition through our mRNA-based therapeutic candidate, ETH42, with the aim of precisely expressing the protein that is defective in the disease to restore lung function.
In a disease like PCD, where no approved therapies currently exist, it is crucial for us to define the present needs and challenges faced by patients to determine what success would mean for them when introducing an effective treatment. Data obtained from the natural history study will aid in designing trials and personalized management strategies, ensuring that patients receive support throughout their entire journey, from initial diagnosis to successful long-term disease management.
In the pediatric patient population, drug development programs must carefully consider the needs of children and the preferences of those who administer the treatment. Parents and caregivers are not only looking for treatments that are effective and safe but also ones that are practical to give to a child and that do not cause discomfort, pain, fear, and anxiety. When a treatment is convenient for a parent to give and well received by a child, compliance increases, and the treatment is more likely to be successful.
Children with pediatric growth hormone deficiency (PGHD) currently have only one therapeutic option: injections of recombinant human growth hormone (rhGH) under the skin. Whether given as a daily injection or a long-acting weekly formulation, a child with PGHD may receive hundreds if not thousands of rhGH injections over the course of their treatment. These injections can be a heavy burden to both the patients and their caregivers and lead to reduced compliance and a missed opportunity for additional height gain. At Lumos Pharma, we are advancing an investigational oral therapeutic alternative to rhGH injection that stimulates the pulsatile secretion of natural (endogenous) growth hormone from the pituitary gland and works within the body’s intact endocrine feedback loop to reduce the burden of injections and help children with PGHD reach their maximum growth potential.
Patients who live with psychiatric conditions like anxiety and depression know that symptom-based diagnoses fail to capture the many and meaningfully differentiated aspects of their personal experience. Until very recently, healthcare providers have shared this understanding but have lacked evidence-backed tools and approaches to adopt a more patient-centric treatment paradigm.
Clinical trial design is just the starting point for developing a more precise and nuanced understanding of how these conditions affect patients. Proactively recruiting trial participants of different ages, races, ethnicities, and economic backgrounds provides the most robust basis for assessing the safety and efficacy of a medical product for anyone who might someday use it.
The critical next step for our industry is to develop and rigorously test digital medicine technologies that can monitor and collect data across the entire patient journey. Only once we have collected that data, reliably and comprehensively, can we hope to understand the insights that can enable more effective and patient-centric treatments.
In the development of psychedelic medicines, for example, recent research has supported what we’ve long hypothesized. Subjective aspects of the psychedelic experience appear to have reproducible and predictable subtypes that correlate with enduring improvements in mental health. As drug developers and treatment providers, we’ve only just begun the process of refining how we collect and analyze these types of data throughout the entire treatment cycle.
Digital medicine tools may prove vital in advancing novel, consciousness-altering treatments like psychedelic medicines, in which real-time data feedback is essential for patient safety and deriving beneficial outcomes.
Patients are always at the forefront of our work, and it is Portage’s mission to expand the number of patients who derive long-term benefit from immunotherapy. The advantage of targeting the immune system is that you can develop approaches that utilize the patient’s own body and natural defenses to cancer, not necessarily the tumor cell or location. At Portage, we utilize a precision immuno-oncology approach, focused on boosting specific aspects of the immune system that are identified by profiling immune cells in the tumor based on samples taken directly from patients. This enables us to select for specific patient populations with the highest probability of responding.
We leverage clinical data from our peers to help inform our clinical trial strategies, including our adenosine program. The adenosine field has been handicapped thus far by the failure to identify an enriched patient population based on receptor expression. Some approaches look at the tumor as a whole and do not distinguish tumor expression from immune cell expression. We are optimizing trials for patient biomarker selection, therefore identifying patients that have a higher potential to benefit from treatment with fewer side effects. We believe this approach will help us gain valuable insight to improve upon previous shortcomings.
Looking at the future of the industry, we believe that precision immuno-oncology will continue to play a critical role in drug development and commercialization. By staying at the forefront of this rapidly evolving field, we can better serve the needs of patients and contribute to the advancement of medical science.
Patient needs and concerns have always been at the forefront of what we do at Recce Pharmaceuticals. Our anti-infective pipeline focuses on addressing unmet medical needs and an impending global crisis for one of the biggest emerging concerns in patient care — antimicrobial resistance (AMR), most often acquired in a hospital setting. As per the Food and Drug Administration, an unmet medical need is a condition whose treatment or diagnosis is not addressed adequately by available therapy. Moreover, AMR arises from the overuse of antimicrobials and contributes to the development of multi-drug resistant bacterial species, often referred to as “superbugs.” AMR has significantly increased due to a surge in antibiotic use since the start of the COVID-19 pandemic and could result in killing 10 million people every year by 2050 if no preventative action is taken. One of the most significant complications from AMR is sepsis, a life-threatening inflammatory response to infection that has spread to the body. Throughout our clinical studies, we’ve established partnerships with hospital systems, patients and their physicians, and advocacy groups like Sepsis Alliance to ensure that patients’ concerns are heard. These partnerships allow us to bridge the gap between clinicians and patients. Our goal is to ensure that we are not only supporting the rights of patients to receive safe and adequate care but also establishing a collaboration that provides a framework of decision-making that supports a patient’s needs, wants, and preferences by increasing patient and healthcare provider satisfaction and quality of life for the patient.
Patients are at the center of everything we do at Revolo. As we advance our drug candidates through clinical trials, we are working to increase awareness of active clinical trials to ensure patients in need of new therapeutic options can easily learn more and determine their eligibility for enrollment.
In 2021, Revolo launched a phase II trial for our lead immune resetting candidate, ‘1104, in eosinophilic esophagitis (EoE), an allergic disease characterized by swelling of the esophagus that affects roughly one in 1,500 to 2,000 people in the United States. To reach as many patients as possible, we developed an approachable and highly accessible page on our website that included the eligibility requirements for the trial, why patients should consider participating, the purpose of the clinical trial, several frequently asked questions, a digestible explanation of our science, a map for the easy identification of the study locations, and a form for patients to reach out with questions. After developing the site, we then leveraged social platforms, such as Twitter, Facebook, and Instagram, to share the clinical trial information broadly with relevant audiences. From there, we engaged patient advocacy organizations to further broaden the awareness of our clinical trial. Combined, we were able to reach patients where they are, listen to their voices there, and ultimately reach more patients at a faster pace, providing a potential new solution for those in need sooner. Engaging patients where they are in an approachable and meaningful way is key to conducting a patient-centric clinical trial.
Here at Sernova, our goal and mission are to develop innovative treatment options for patients living with chronic diseases. Living with a chronic disease can be challenging enough, but having the burden of time-consuming monitoring and treatments and disease comorbidities adds to the diminished quality of life. We are seeking to improve the quality of life for patients by developing treatment solutions that are more accessible, require lower long-term costs, and positively impact the treatment experience for patients.
We are advancing our Cell Pouch System™, a small, implantable medical device with immune-protected living therapeutic cells, which provides a complete cell therapy approach and potential “functional cure” for insulin-dependent diabetes and other chronic diseases. For years, patients with diabetes have had to rely on daily insulin injections in combination with external insulin delivery devices while constantly monitoring blood sugar levels. Even furthermore, they also must worry about the high long-term costs of insulin and other secondary diseases caused by diabetes, such as blindness, heart disease, kidney disease and amputations. Our knowledge of these current challenges that patients face in treating diabetes strongly influences many aspects of our research and development. Ultimately, with our Cell Pouch System, we aim to bring meaningful value, in the form of a new treatment option, to the day-to-day lives of patients who are living with chronic diseases.
When developing new treatments, patient access and patient experience should be included as measures of efficacy — efficacy is more than just “who responds” or “who goes into remission.” If a transformative treatment outcome is unattainable by a significant majority of patients today, what good is it?
This understanding is what led to the founding of Umoja Biopharma. Umoja is an in vivo gene delivery company aiming to engineer cancer fighting CAR-T cells directly inside a patient’s body. Currently, CAR-T cell cancer patients face a daunting treatment journey of cell collection, manufacturing, delivery, infusion, and hospital stays that oftentimes take weeks if not months. Sometimes patients are too sick for such a process or may choose to opt out of treatment altogether after years of toxic chemotherapy and stem cell transplants. Umoja’s goal is to change this.
Umoja’s in vivo technology coupled with an in-house state-of-the-art lentiviral vector manufacturing facility was developed to increase patient access to CAR-T cancer treatment and remove lengthy wait times and the need for toxic chemotherapy. By generating CAR-T cells in vivo, off-the-shelf and patient-specific lifesaving treatment could be available for multiple cancer indications.
Umoja is committed to a future where the benefits of CAR-T cell therapies are available to patients at lower cost, faster speed, and broader reach — getting those transformational efficacy outcomes into the hands of more patients and their families.
We are lucky that at Be The Match BioTherapies we are constantly reminded that in order to achieve our nonprofit mission, patients and patient-centric concerns have to be our highest priority. So, there is nothing that influences our work more significantly, and I don’t expect that to ever change.
As the cell and gene therapy landscape evolves, we have to pay ever closer attention to the needs of the patients of the future. With cell and gene therapies already demonstrating impact in diseases where the patient numbers will far exceed the number of patients we are capable of treating today, we need a paradigm shift in the capabilities of technologies used in manufacturing. It would be so disappointing if we were unable to offer these transformative treatments to everyone who could benefit from them, because of a failure to develop sufficiently high-throughput and low-cost manufacturing approaches. So, from our perspective as technology developers, we’re already thinking ahead to where this needs to go and from the beginning have been developing technology with high-throughput manufacturing in mind, but in a step-wise approach that bridges where we are today to where we want to go.
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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