From the series Industry and pharmaceuticals
The 20th century, particularly its second half, saw an extraordinary transformation in the field of pharmaceuticals. Starting with the production of penicillin, the first in a long line of antibiotics, the therapeutic capabilities of thousands of chemical compounds have been discovered, capable of changing the natural history of many diseases – that is, their normal course if left untreated. Industrial production has filled pharmacy shelves with ready-to-use remedies, or purported remedies, replacing the pharmacist’s ancient galenic formulations. Italy’s regulatory agency, Agenzia Italiana del Farmaco (AIFA), lists approximately 3,000 active ingredients across various therapeutic classes, for nearly 11,000 human medicines authorised for sale.
However, we should not forget that, for years, in our society of mass production and consumption, there have been alarming shortages of many essential and low-cost medicines. Furthermore, according to the World Health Organisation, one third of the global population has no access to basic medical care. The cornucopia of medicines is not within everyone’s reach.
The “standard” patient
The most recent advancements in medical science pose a different problem for the “pharmaceuticals of the 20th century”. First, a treatment does not necessarily imply a cure. An incurable disease still requires appropriate treatment to keep it under control, prevent complications, or at least alleviate the patient’s suffering. A cure, which involves correcting the causes of the disease, remains far off in most cases, even today. Paul Ehrlich [1854-1915], the German pathologist who launched Salvarsan against syphilis in 1909, founding modern chemotherapy, was looking for a magic bullet
that would destroy the pathogen without harming the body. Medicine has moved closer to Ehrlich’s dream
, but it remains largely unfulfilled. However, the new biotechnological therapies aim to make that dream a reality.
While the “conventional” therapeutic arsenal is well stocked, it suffers from individual variability in response. This is an ancient and well-known principle, too often neglected in medical practice. Commercially approved medications are, as a rule, subjected to clinical trials on larger or smaller samples of a population and are evaluated on the basis of statistical averages. However, a medication indicated for a specific pathology, on a one size fits all basis, will not have the same therapeutic efficacy and the same side effects for each subject. The response will differ among ethnicities, population subgroups, genders, and individuals. It has been estimated that a drug reaching the market will, on average, only work for half of those patients who take it. According to a study reported by the Personalized Medicine Coalition (PMC), the percentage of the population of patients for whom a drug of the therapeutic class corresponding to their condition is ineffective is 38% for depression, 40% for asthma, and even higher for diabetes, arthritis, and cancers [PMC, The Case for Personalized Medicine, 2014].
Tailor-made medicines
Among the many factors considered responsible for individual variability in response to medication, from environment to lifestyle, the genetic profile has attracted particular attention. Advances in genomics, accelerated by the sequencing of human DNA starting in the 1990s, have stimulated the development of new technologies. These allow, for example, the identification of a gene (or genes) responsible for a disease and the correction of harmful mutations, or the identification of the molecular basis of a predisposition to a disease. The PMC has announced a new era of personalised medicine
, an evolution from “reactive” medicine to predictive diagnoses, from standardised treatments to individualised therapies.
The scope of application is vast and, in some respects, still unclear. According to the European Union’s definition, personalised medicine (also, somewhat improperly, referred to as “precision medicine”) refers to a medical model using characterisation of individuals’ phenotypes and genotypes (e.g., molecular profiling, medical imaging, lifestyle data) for tailoring the right therapeutic strategy for the right person at the right time, and/or to determine the predisposition to disease and/or to deliver timely and targeted prevention
[Council of Ministers of Health of the European Union, “Council Conclusions on Personalised Medicine for Patients”, December 2015]. The scientific advisors to the US presidency clarify that it does not literally mean the creation of drugs or medical devices that are unique to a patient, but rather the ability to classify individuals into subpopulations that differ in their susceptibility to a particular disease or their response to a specific treatment
[President’s Council of Advisors on Science and Technology, “Priorities for Personalized Medicine”, September 2008].
Health “big data”
While the potential of personalised medicine is widely recognised within the scientific community, not everyone shares the same enthusiasm. Doubts are raised about its practical limitations, stemming from the complexity of genetic variations even among individuals, the multifactorial origins of many non-transmissible diseases, the limited number of qualified specialist centres, and prohibitive costs. Moreover, the excessive emphasis placed on the genetic causes of diseases threatens to overshadow other well-known factors, such as lifestyle and diet, which are considered more important for population health and more easily addressable. There is a risk of diverting precious funds that could be allocated to population health, writes the Journal of the American Medical Association (JAMA).
Many of the most common diseases have a multifactorial genesis, the journal notes, and excessive enthusiasm risks fostering excessive expectations that may later be disappointed [JAMA, “Will precision medicine improve population health?”, October 4th, 2016]. Similarly, The Lancet suggests that Although precision medicine will almost certainly be used in niche applications, if widely implemented, it could be a distraction from low-cost and effective population-wide interventions and policies
[The Lancet, “Is precision medicine the route to a healthy world?”, April 25th, 2015].
Furthermore, managing the genetic data of millions of people, made available through current databases and artificial intelligence, raises privacy issues. Implementation of precision medicine would need each person’s genetic profile to be obtained, raising complex ethical, legal, financial, and social issues
[The Lancet, op. cit.].
This is an issue that already concerns health data. In 2023, the English National Health Service (NHS England) signed a £330 million contract with Palantir, the US data analytics company co-founded by Peter Thiel. Palantir, known for its ties to the defence and intelligence sectors, will develop the Federated Data Platform that will manage patients’ health records [Financial Times, “US tech group Palantir wins lucrative NHS data contract”, November 22nd, 2023]. The fact that “sensitive” data is entrusted to a US spy technology firm
has raised protests from, among others, the British Medical Association [British Medical Journal, November 21st, 2023].
What the market says
“Personalised medicine” nonetheless promises a radical change in diagnostic and therapeutic techniques. The life sciences industrial complex is undergoing profound transformations, investing billions in genomic research and development (R&D). This field is deemed strategic by the major powers, which compete over their innovative capacity.
In 2005, molecular entities classified as “personalised medicines” accounted for 5% of new drugs approved that year by the Food and Drug Administration (FDA), the US regulatory agency. By 2019 this figure had risen to 25%, and by 2023 to 38%, according to the PMC. In 2008, there were five personalised drugs (indicating specific biomarkers) on the market; this number increased to 286 in 2020, more than half of which were only launched in the previous four years. These are used in various therapeutic classes, primarily in cancer treatments [PMC, “The Personalised Medicine Report 2020”].
The number of innovative “advanced therapies” (advanced therapy medicinal products, ATMP) is growing. The European Medicines Agency (EMA) categorises these into three main types: gene therapy, which uses genes to correct a genetic defect; cell therapy, which relies on the use of cells, whether genetically modified or not, for treatment, diagnosis, and prevention; and tissue engineering, designed to repair, regenerate, or replace human tissues.
By the end of 2023, 76 cell and gene therapies (including tissue engineering) had been approved for clinical use in at least one country – more than double the number of the decade prior, with twenty of these launched in just the last three years [IQVIA, “Strengthening Pathways for Cell and Gene Therapies: Current State and Future Scenarios”, March 2024]. Citeline, a Norstella company, further notes an additional 35 RNA-based therapies [ASGCT-Citeline, “Gene, Cell, & RNA Therapy Landscape Report”, January 2025].
IQVIA estimates that total spending on cell and gene therapy (CGT) reached $5.9 billion in 2023, a 38% increase from the previous year. It is a market that attracts investment and is characterised by intense acquisition activity and licensing agreements.
Towards Asia
Over the past five years, over 3,200 CGT clinical trials have been initiated, with industry-funded trials growing faster than those sponsored by government or academic institutions [IQVIA, op. cit.].
From a geographical standpoint, Asia’s weight is increasing. In 2008, most trials were conducted at sites that were at least partly American. By 2023, the US share had fallen to 41% for industrial trials and 35% for non-industrial ones. China’s share, which was 4% and 6%, respectively, over the last decade, now surpassing not only Europe but also the United States in both categories. Meanwhile, Europe’s share in industrial trials has declined from 25% to 10% over a decade, and from 20% to 7% for non-industrial trials.
This movement involves the entire pharmaceutical supply chain. Looking at the pipeline of drugs under development, it has been calculated that the US share in 2024 is 49.1% of the total, compared to 51.1% in 2023. China, on the other hand, has increased its share from 23.6% to 26.7%. South Korea is in third place with 3.2%, having overtaken both the UK (3.1%) and Germany (2.5%). China, notably, has four pharmaceutical groups among the world’s top 25 in terms of the size of their development pipeline. Jiangsu Hengrui Pharmaceuticals, a company with a turnover of $4 billion, has jumped from thirteenth to eighth place in one year, ahead of giants such as the American pharmaceutical company Merck & Co. and the French Sanofi. Jiangsu also has the highest percentage (94%) of completely “in-house” medicines compared to the other companies. This is a landmark moment for the Chinese pharmaceutical industry, which, 20 years ago, had little to no homegrown pharma R&D focused on original compounds
[ASGCT-Citeline, “Pharma R&D Annual Review 2024”].
The battle for the status of scientific and technological superpower is now also unfolding in the realm of medicine.