In 2024, The Medicine Maker celebrated its 10 year anniversary. As part of that, we reached out to over 100 industry experts to ask:
“Looking ahead to the next 5–10 years, what will be the key disruptors and/or what can be improved upon in the pharma industry?”
Here are the experts who focused on personalized medicine…
Changing the Status Quo of Drug Development – with Scott Lewis, Head of Antibody at Biosynth
For decades, the pharmaceutical industry has used a fairly steady modus operandi for new drug development:
research a disease or disease segment
identify a biomarker associated with this disease
develop a reagent that blocks the action of the biomarker
study what happens when you give this reagent to animals and then people
hope it does less harm than good
release it as a drug
This method is notoriously inefficient, expensive, and questionably effective. The signalling pathways along which cellular communication occurs in biological systems are intricate and unfathomably complex. Each protein can interact with many other proteins depending on the conditions involved (cell cycle, protein modification state, etc.), and all of those proteins can go on to interact with many others, and so on.
To think we can remove any one protein from this cascade and accurately influence things for a preponderance of people in a positive manner without repercussion is hubristic. This inefficiency in the current process underscores the urgent need for a new approach to drug development.
One way to dramatically improve pharma is the widespread use and implementation of personalized medicine. This is not a new concept by any means, but current efforts are largely fractioned and not coordinated for maximum benefit. Forming baselines for inherited genes, understanding the impact of mutations, gaining a greater understanding of knowing exactly what is causing any given symptom or issue, and then collating the information in a central location could provide incalculable gains and lead to vastly more effective diagnostics and treatments.
View From a Cancer Researcher – with Yael Mamane, Senior Director Bioscience and Translational Sciences, Sygnature North America
In the next decade, I envision a pharmaceutical landscape where precision medicine isn't the exception, but the standard for all drug discovery and development. Precision medicine begins with identifying the specific biological target driving a disease, whether it's centered on a certain protein, mRNA, gene, or other molecule. Next, we develop therapies that modulate this target precisely, minimizing harm to healthy cells and tissues. Crucially, we identify the patients most likely to benefit from these interventions, avoiding unnecessary side effects and wasted resources. Lastly, we need to think about combination therapy and poly pharmacology since complex diseases such as cancer and Alzheimer’s disease have multiple factors driving their pathology. Furthermore, combination therapy can act synergistically and prolong time to resistance, and therefore any new treatment paradigm will require a multi-pronged approach.
Why is this shift critical? I am a cancer researcher and my mother has colon cancer with a known KRAS G12V mutation. Every three weeks for the past four years I’ve seen her endure debilitating and painful side effects from receiving classical chemotherapy that was discovered over 50 years ago. From experiencing weight and hair loss, and tingling sensations due to nerve damage, to weakness and gastrointestinal issues after every chemotherapy session, her experience underscores the urgent need for targeted therapies that effectively target the root causes of her disease.
The good news is that we are making progress! Several companies are developing precision oncology drugs, such as KRAS inhibitors that target my mom’s mutation, which could offer new hope for my mother and patients like her – I only hope that they reach her in time!
To truly transform the industry and ensure breakthroughs for people like my mother, we must embrace precision medicine as a foundation of drug development programs. This requires a paradigm shift from target identification all the way through clinical trial design. We need a general overhaul of the medical system – everything! From diagnosis, data-sharing, and economics, to training doctors on how to test, communicating with patients, and implementing genetic and biomarker testing.
The Path to Better Healthcare – with Steven Bloom, CBO, Vincerx Pharma
Traditional medicine often uses a "one-size-fits-all" approach, where treatments are based on the average response of a population and often take a trial-and-error approach. On the other hand, tailoring treatments to individuals can be more effective and have fewer side effects by addressing the root of the problem.
By using biomarkers to identify the patients most appropriate for specific drugs, the industry can ensure that therapies are tailored to individuals who are most likely to benefit. The hope lies in enhanced patient response and treatment efficacy, and not to mention, advancing early detection of diseases. Identifying individuals at high risk for certain conditions through genetic screening and biomarkers allows for interventions (including with targeted therapies) to be made sooner, potentially preventing the disease or catching it at an earlier, more treatable stage.
Choosing the right patient, drug, and dosage is the key to not only improving patient outcomes, but also achieving successful and economical clinical trials that enable researchers to better assess the safety and effectiveness of new investigational medicines. Additionally, drugs that may have failed in one area of treatment might find success in another with better suited genetic profiles.
Overall, precision medicine holds the promise of making healthcare more effective, efficient, and personalized, fundamentally transforming the way the pharmaceutical industry develops and delivers treatments. It’s up to us to realize its promise.
Unravelling Genomic Secrets – with Neil Ward, VP, PacBio EMEA
Genomic sequencing has revolutionized the medical industry; from the introduction of exome sequencing in 2009 capable of pinpointing small genetic changes under 50 base pairs, to the 2014 arrival of short-read whole genome sequencing that enabled insight into the complex structural changes associated with diseases.
In 2022, the remaining eight percent of the human genome was sequenced via long-read sequencing, which runs DNA samples thousands of base pairs long to accurately detect larger, more complex genomic variants. With the depth and accuracy of long-reads, researchers have advanced understanding of disease mechanisms, leading to the identification of new drug targets, the development of novel therapeutics, and improved diagnostics.
Long-reads are becoming increasingly accessible and will allow us to understand which genes impact the metabolism of drugs, leading to greater personalization.
Growing Awareness – with Eleni Lagkadinou, Vice President, Head of Oncology Early Development, AbbVie
Over the last 10 years, there has been significant growth in the awareness and impact of personalized healthcare, with such medicines now representing more than one in three newly approved treatments by the FDA. It’s clear the previous development and commercialization model – where a blockbuster drug was developed as a ‘one size fits all’ therapy, or for a very broad patient population – is changing. This focus, along with expanded access to genomic insights, is contributing to the development of validated drug discovery targets – further supported by AI and machine learning. We are, in essence, approaching a time when truly personalized patient care is becoming a more tangible reality.
The progress in precision medicine is particularly evident in oncology, where ADCs have recently emerged as a new treatment option. In many ways, this development aligns with the broader shift towards biomarker-driven approaches. Testing patients before starting therapy has become increasingly common, thanks to improved infrastructure, integration into treatment guidelines, and faster testing turnaround times. In the next 10 years, our focus should be on partnering with healthcare providers to make it easier to incorporate this wealth of new information into training. It's also noteworthy that personalized care is now expanding beyond traditional areas like oncology to fields such as neurology, psychiatry, and reproductive health.
The Power of Precision – with Becky Upton, President of the Pistoia Alliance
In the past decade we have seen next-generation sequencing (NGS) democratize genomic analysis, driving personalized treatments and large-scale studies. While data privacy and interpretation have remained critical, bioinformatics tools have been evolving to enhance clinical utility. Cell and gene therapies have enabled precise genetic modifications, offering potential cures for genetic disorders and cancers. Advances in spatial biology and multi-omics integration have provided deeper disease insights. Meanwhile, mRNA techniques, proven with COVID-19 vaccines, have begun to revolutionize vaccine development and therapeutics, including regenerative medicine.
These technologies will evolve in precision and impact. Generative AI and large language models will accelerate drug discovery, clinical trials, and precision medicine, significantly reducing time and costs. Quantum computing, once it matures and demonstrates stable economic quantum advantage, will enable unprecedented simulations of molecular interactions, providing new insights for drug discovery. However, the industry must stay abreast of quantum computing developments to leverage advancements in a timely manner. Given the high costs for individual pharma companies to keep up with quantum computing developments, pre-competitive collaboration will be critical to pool resources and share benefits. By working together, biopharma can cost-effectively develop these technologies, providing platforms for healthcare innovation and more precise, efficient, and personalized healthcare solutions.
The Arrival and Maturity of Personalized Medicine and AI – with Dennis van Rooji, Managing Director, DT Consulting, an Indegene company
The pharmaceutical industry has become increasingly collaborative as it looks to harness innovative technology in the pursuit of improving patient outcomes and streamlining drug development. The past decade has already seen some transformative changes, and the next ten years will undoubtedly see many more.
Two milestone achievements that have profoundly impacted the pharmaceutical industry are the arrival of personalized medicine and advances in data generation and AI.
The tailored treatments that personalized medicine provides have revolutionized patient outcomes, most notably in oncology with CAR-T. Additionally, advances in genomic sequencing have facilitated the identification of genetic variations that influence drug responses, leading to the development of newly-accurate targeted therapies in oncology and beyond.
Meanwhile, the digital evolution of clinical trials has brought an exponential increase in data volume, driven by tools such as mobile devices and wearables. This data surge has necessitated advanced data management systems and paved the way for AI solutions, which – though still developing – are starting to enhance clinical trials, by efficiency and accuracy. For instance, machine learning algorithms can now optimize patient recruitment by identifying eligible participants more quickly through electronic health records and social media data.
The future of clinical development will be influenced by how effectively pharma leverages the key disruptors from recent years. Digital maturity of pharma’s clinical operations has stalled over the past two years, with clinical ops leaders often overlooking the need to get their data management capabilities in order. These are needed to power crucial advanced data analytics that could enhance patient experiences by allowing organizations to predict patient needs, personalize communication, and optimize care pathways. Techniques such as mapping patient journeys, similar to the customer journey mapping carried out in other industries, would help identify critical touchpoints and areas for improvement. These approaches could not only increase patient satisfaction and outcomes but also drive operational efficiency and innovation, in turn advancing pharma’s digital maturity.
The past decade has seen significant advancements in digital maturity and personalized treatment, yet the journey continues. Looking forward, it is crucial to focus on sustainable digital initiatives, patient-centric approaches, and cross-functional collaboration across pharma’s clinical operations and beyond.
Personalized medicine is set to have a broader and more scalable impact on pharma once companies’ digital and patient data strategies are effectively aligned. This will be key to enabling value-based care and population health models that focus on prevention rather than just treatment. Ultimately, embracing patient-centric digital strategies in clinical trials will help establish the necessary infrastructure to transform patient outcomes and revolutionize healthcare.
Predicting Risks to Health – with Vilma Methner, Senior Market Development Manager ATMP, Optima pharma
I see personalized medicine continuing to advance. For me, this includes personalized and biomarker analysis that can predict risks to personal health and provide both prophylactic and diagnostic recommendations. AI can evaluate and process the collected analysis data more quickly, design clinical studies more efficiently, and compare them with studies that have already been completed. This leads to significantly faster evaluations and avoids unnecessary costly studies.
Machines, robots, and process automation can also reduce production costs and make previously expensive individual therapies affordable. To this end, I hope that specialty facilities for specific diseases and their treatment will be strengthened. Better access to R&D results and SME innovations in practice and clinical application should also be made possible. This requires both start-up funding and technical equipment support, such as the establishment of hubs where researchers can work. It is equally important that the translation of innovations from R&D to the clinic and industry is as unbureaucratic as possible.
In the use of personalized medicine, manufacturing processes are often still performed manually. These processes require further automation. Automated manufacturing should enable the possibility of faster adaptation of processes and facilitate a variety of processes through the establishment of platform technologies.
AI Meets Personalization – with Maryam Ahmadi, Director of Science, Sphere Bio
For me, AI stands out as the most revolutionary innovation in the pharmaceutical industry of the past decade. Drug discovery, a traditionally slow, costly, and high-risk process, has been revolutionized by AI . Analysis of vast datasets, identifying potential drugs, predicting efficacy, and optimizing chemical structures with unprecedented speed has been unlocked using AI algorithms. Insilico Medicine’s AI-designed drug for idiopathic pulmonary fibrosis, reached phase I trials in just 30 months – an astounding acceleration.
Clinical trials have also been streamlined by AI, enhancing patient recruitment, real-time monitoring, and outcome prediction, significantly cutting time and costs. However, AI’s true power can be unleashed in personalized medicine. As a relative of a cancer patient, I deeply appreciate the technology’s potential. Advances in genomics and biomarker identification allow drugs to be tailored to individual genetic profiles, vastly improving efficacy and reducing adverse effects. AI processes vast patient data, identifying patterns for precise diagnoses and treatment choices, crucial for conditions such as cancer and rare diseases. This precision optimizes medication timing and dosage, revolutionizing patient care.
Looking ahead, the synergy between AI and personalized medicine will drive profound changes in the pharmaceutical industry. AI's capacity to learn from complex datasets will complement personalized medicine’s focus on individual genetic and health profiles, heralding a future of more effective, safer treatments developed rapidly and cost-effectively.
For the industry to harness these benefits, collaboration between academia and industry is crucial. Transparency, ethical considerations, and shared expertise will foster innovation, unlocking the full potential of these technologies. The next decade promises a healthcare revolution, propelled by the powerful forces of AI and personalized medicine.
A Non-Standard Future – with Chris Molloy, CEO, Medicines Discovery Catapult
Personalized dosing could revolutionize healthcare and vitalize the industries that provide it today and tomorrow. We generally talk about personalized medicine as a well-targeted drug, but that is a pyrrhic victory if that drug is not given to the right patient, at the right time, or at the right dose. Today’s treatment centers are designed to give standard doses of a drug to standard patients in a process – sometimes without recourse to the other drugs they are being given. Real patients have different livers, kidneys, immune systems, microbiomes, diurnal rhythms, and physical characteristics that will dramatically alter their reaction to, and success with, any drug.
Imagine a future where rapid home or high street diagnostics enable patient profiling so that their cyp-profiles, serum medicines concentrations, cytokine states, and microbiomes are known. Using this information, the right amount of drug can then be dispensed and delivered to them at the right time of day, at home, at work, or under controlled conditions in the high street. This can be supported with the appropriate clinical supervision online or on-hand. Not only will this increase cost efficiency for dosing of more complex drugs, it will increase compliance and efficacy, so that most medicines will work as they did in the trial. Moreover, it will open up new business models for pharma, diagnostics, data, and delivery firms. This is a new era of development that will transform progressive pharma companies from high-value producers into active healthcare providers.
Making Use of Multi-Omics – with Carleen Kluger, Group Leader Clinical Proteomics, Evotec
Multi-omics analysis of large patient cohorts, such as the UK Biobank, German NAKO, deCODE from Iceland or the biobanks managed by Fingenious, could revolutionize precision medicine and drug discovery.
Currently, characterization of healthy individuals and patients prior to any medical intervention, diagnosis or recruitment to a clinical trial is based on a small panel of selected biomarkers. In the future, I believe that a full multi-omics screen (comprising of genomics, transcriptomics, proteomics, and metabolomics) performed on minimally invasive samples, such as biofluids, would provide a far more comprehensive and beneficial picture. To unlock the full potential of these molecular fingerprints, population sized cohorts, with multi-omics data from hundreds of thousands of individuals, would need to be generated to provide the background on which samples can be matched. Although each omics technology alone provides significant insights, only together can they create a full picture. While a patient’s current health status (which is influenced by nutrition and lifestyle) can be assessed using proteomics and metabolomics measurements, genomics will remain indispensable for analysis of genetic risk factors.
Moving from separate databases from individual studies and trials to a continuous and extendible database would be a big step for the pharma industry. Generating a reference framework with a standardized multi-omics format to which individual patient samples can be mapped could be a huge step forward, enabling not only precise diagnosis and treatment, but also the design and development of new drugs. First steps are already being made to generate such data sets; however, a standardized multi-omics format, as well as easy integration of additional data or cohorts into already existing databases, is still missing.
This is a joint effort which the pharma industry needs to work on together to continue our journey for medicines that matter.
Excitement For RNA – with Sam Deutsch, CSO, Nutcracker Therapeutics
RNA has the potential to deliver a wide array of new, lifesaving therapies into the hands of patients. The challenges in RNA therapeutic delivery are difficult, but innovative platforms will provide the means to develop RNA therapeutics in a scalable and efficient manner, including in the personalized setting. In the years ahead, RNA as a therapeutic modality could become a first-line tool in treating many cancers.
The response to the COVID-19 pandemic proved what RNA can do, and we most certainly have the infrastructure to yield millions of doses. However, the next phase of RNA medicine will focus on smaller, more specific, and personalized scales. Flexible and scalable RNA manufacturing technologies will enable therapies that address the unmet needs of patients with diseases that are less prevalent than others.
RNA can encode for virtually any protein. I believe this class of molecule can play a major role in how we envision protein therapies, enzyme replacement therapy, gene therapy, and more. One of the advantages of working with vaccines and therapies at the RNA level is the simplification of engineering challenges, enabling modalities with complex mechanisms and unique properties.
The CRISPR Revolution – with Cillian McGorman, Commercial Operations Manager, ERS Genomics
CRISPR is a technology that has continuously made waves in our industry for the past 12 years since its inception. Although there has been mass adoption, the technique is still in its infancy, but has the potential to transform the entire industry.
CRISPR is being used not only for groundbreaking gene therapies, some of which hit the market in early 2024, but also to identify novel drug targets, study diseases, and contribute to the rapid development of vaccines and small molecule drugs. CRISPR enables researchers to explore the genetic basis of diseases, uncovering critical insights that drive the development of targeted treatments. By accelerating drug discovery and refining therapeutic approaches, CRISPR/Cas9 holds the promise of eliminating diseases at their source, bringing drug production costs to new lows, and underpinning the future of personalized medicine.
Passion for a New Healthcare Landscape – with Kasia Maj, founder and CEO, Excellio
The pharmaceutical industry stands on the brink of a revolution, where precision and efficiency will redefine how we approach treatment. Imagine a future where treatments are not just generic solutions, but finely tuned therapies that are delivered with pinpoint accuracy to the exact location in the body where they are needed.
Biopharmaceuticals and biosimilars will continue to disrupt the industry, offering more sustainable and environmentally friendly alternatives to traditional small molecules. Precision drug delivery systems, such as tissue-specific exosomes will enable their implementation, ultimately transforming the landscape of healthcare as we know it.
Picture a world where cancer therapies are directed solely at tumour cells, sparing healthy tissues and reducing the often devastating side effects of treatment. This not only enhances the effectiveness of treatments but also aligns with the growing demand for individualised medicine.
The future of healthcare lies in our ability to embrace these innovations, creating a system that is not just reactive but proactive, where therapies are tailored to the unique biology of each patient. This vision is not merely about technological advancement – it’s about igniting a new era in medicine – one that is compassionate, precise, and ultimately transformative.
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