Medicines developed through clinical trials: real examples

What are clinical trials?

Clinical trials are research studies that test how well new medical approaches work in people; each study answers scientific questions and tries to find better ways to prevent, screen for, diagnose, or treat a disease. A trial may also compare a new treatment against a treatment that is already available^[1]. That comparison is what allows regulators to judge whether the new option adds something for patients.

For a patient, the practical question is simpler: will this research reach the shelf? The examples below are medicines and therapies that reached the market – or are being tested now in people – and that exist because doctors, researchers and patients agreed to run and join trials.

How did gene and cell therapies become authorised medicines?

Advanced therapy medicinal products (ATMPs) are a highly complex group of medicinal products applied directly to patients, made up of gene therapeutics, somatic cell therapeutics and tissue engineered products. After many years of development, the number of marketing authorisations issued for these medicines is now rising significantly^[2].

Five gene therapeutics were authorised in 2021 and 2022 alone. CAR-T cells – genetically modified autologous immune cells (T cells) modified outside the patient's body – have hitherto been used in the therapy of certain blood cancers, such as leukaemias and lymphomas. They are now also being developed for the treatment of autoimmune diseases such as systemic lupus erythematosus (SLE)^[2].

The scientific basis for this class was reviewed publicly years before the first product reached patients. In 2010, the European Medicines Agency (EMA) assembled European and international experts to review opportunities and difficulties in developing stem cell-based therapies and to discuss regulatory challenges. At that time, some 40 clinical trials were already exploring the use of stem cells in the regeneration of lost or damaged tissue – in the heart, skin, bone, spinal cord, liver, pancreas and cornea – and in haematological or solid-organ malignancies. The majority of those trials used mesenchymal cells derived from adipose tissue, bone marrow, stromal cells and connective tissue, and a small proportion used haematopoietic stem cells. The EMA's Committee for Advanced Therapies (CAT) confirmed the classification of three different stem-cell therapies as ATMPs^[3].

Which medicines were recommended for authorisation in Europe in 2026?

During its July meeting, the EMA's human medicines committee (CHMP) issued positive recommendations for two medicines that address significant patient needs^[4].

• Susvimo (ranibizumab) - a new treatment option for people living with neovascular (wet) age-related macular degeneration; it introduces an ocular implant designed to reduce the burden of frequent eye injections^[4].
• Icotyde (icotrokinra) - the first oral anti-IL-23 medicine for moderate-to-severe plaque psoriasis, providing an oral alternative to anti-IL-23 injections for patients requiring psoriasis treatment^[4].

Both were described by the Agency as having the potential to address significant patient needs by offering new therapeutic approaches and improving treatment experiences. Also in July, the EMA announced the start of a phased review for daraxonrasib, a medicine intended for metastatic pancreatic cancer. A phased review aims to accelerate the assessment of a medicine by evaluating the data in phases, as they become available^[4].

Can an existing medicine gain a new use through trials?

Yes – and this is one of the most direct routes from research to a new treatment. In April 2020, dozens of clinical trials were testing drugs originally designed for other ailments, such as Ebola, HIV, malaria and arthritis, against COVID-19. On 16 June 2020, researchers revealed that dexamethasone, a cheap, widely available steroid, managed to reduce deaths in patients with severe COVID-19. Giving the steroid to critically ill COVID-19 patients on ventilators reduced deaths by one-third, and it reduced deaths in patients on oxygen by 20%. COVID-19 patients who did not need help with breathing did not benefit from the drug^[5].

Other examples show the same mechanism – a study decides whether an existing medicine helps or does not. Remdesivir, an antiviral drug developed by Gilead Sciences in 2009 and previously tested in more than 100 Ebola patients, was shown to help coronavirus patients recover more quickly, and based on that data the US FDA issued an emergency authorisation for its use. The drug has also received regulatory sign-offs in Europe and Japan. By contrast, other promising treatments fell short in studies, including the antimalarial medication hydroxychloroquine, the HIV medication Kaletra and the arthritis medication Kevzara^[5]. A trial that ends without benefit is still a result: it stops patients from receiving a treatment that does not work.

How does research reach people with rare diseases and children?

Clinical trials are the route to treatment in rare diseases, where the medical need is most concentrated. Rare diseases affect a very large group of people and the numbers set the scale of the problem: 50% of rare diseases affect children, 95% have no single approved drug treatment, 35% of deaths in the first year of life are due to rare diseases, and 30% of children with a rare disease will not live up to the 5th year of age^[1].

Children were historically treated with medicines that had never been properly studied in their population: more than 50% of medicines used for children were never or incompletely studied in this group. That situation was typical before the paediatric legislation and was marked by an absence of age- and development-related research, a lack of suitable products and recurrent off-label use^[1].

The European paediatric rule changed the picture. On 26 January 2007, European Regulation (EC) No 1901/2006 – the Paediatric Drug Regulation – took effect with the objective to improve the health of European children by facilitating the development, accessibility and safe use of new drugs for children aged 0 to 17 years, through clinical studies. The first marketing authorisation based on a completed paediatric investigation plan followed in 2009, the first Paediatric Use Marketing Authorisation (PUMA) in 2011, the first Commission Report in 2013, and the second Commission Report in 2017. A parallel path exists for rare diseases: orphan designations and authorisations have grown year by year, with oncology representing the largest therapeutic area – 45.2% of orphan designations in the 2000-2010 period, then 41%, 39-40% in the following years^[1].

Designing such studies is difficult. Clinical trials for rare diseases involve very small populations of patients, which implies the use of non-homogeneous groups for age and phenotypes. Large variation in severity, stage, irreversibility and age leads to a very large range at baseline for many measures of efficacy, making it hard to detect clinically important efficacy changes, and the lack of quantitative natural history information makes the analysis of trial data harder still. Traditional randomised controlled studies are not suited for small populations^[1].

What trials are running in patients right now?

Some programmes give a concrete picture of what is being tested in people today. In the current financial year, one hospital recruited the first participant in the world to three clinical trials, compared with two across the whole of the last financial year^[6]. These three studies cover very different areas:

• A CAR T cell therapy trial aiming to slow – or even halt – the progression of multiple sclerosis^[6].
• A study looking at how safe and effective the medicine Xromi® is for babies and toddlers with sickle cell disease^[6].
• A study testing a medicine called nirogacestat for treating desmoid tumours, which are rare, non-spreading but sometimes harmful soft-tissue growths^[6].

The same site recruited the first participant anywhere in the world outside the USA and Canada to a trial investigating whether a robotically implanted brain-computer interface can improve independence for people who are paralysed. It also recruited the first participant in Europe on four occasions and the first UK participant on nine occasions, with 16 recruitment "firsts" so far in the financial year. Patients treated in such trials gain earlier access to treatments that are still being evaluated. A hospital's ability to attract, set up and begin commercial studies at speed determines how quickly that access becomes available^[6].

How does a trial result become an authorised medicine?

Treatments trialled in hospitals go on to be approved by regulators, which is what allows wider access for patients. In one recent year, the first ever treatment for the rare neurodegenerative disease Friedreich's ataxia - for which researchers at the same institution led the national trial – was approved for use by the medicines regulator. Earlier in that year, a university spinout company received authorisation from the national medicines regulator for a next-generation CAR T-cell therapy developed to treat adults with an aggressive blood cancer, following trials carried out at the hospital^[6].

What role does the EMA play in Europe?

The Paul-Ehrlich-Institut, as the national competent authority for Germany, evaluates and approves clinical trials with novel medicinal products in Germany. It also carries out the evaluation of European applications for authorisation in the CHMP at the European Medicines Agency. It also provides national and international scientific advice on ATMPs and is responsible for ATMP vigilance. The evaluation of ATMPs is complex and therefore requires special knowledge held by such specialist bodies^[2].

Why is it important that trial results are submitted?

Transparency is part of how the system earns trust. In 2026, the EMA newsletter drew attention to an open letter calling for the submission of missing clinical trial results to the Clinical Trials Information System (CTIS), reinforcing the importance of transparency and trust in medical research. The same newsletter draws attention to a newly published paper outlining key conclusions and strategic recommendations from an EMA multi-stakeholder cardiovascular workshop held on 1-2 July. The paper explores modern strategies for refining clinical trial endpoints, improving study designs and addressing pressing unmet needs in cardiovascular medicines^[4].

How can you find a trial?

If you want to know whether a trial exists for your condition, start with the public registers. In Europe, missing clinical trial results should be submitted to the Clinical Trials Information System (CTIS)^[4]. Trials recruiting in Europe can also be searched on clinicaltrials.eu. A conversation with your treating doctor is the next step: eligibility depends on your diagnosis, stage of disease and previous treatments, and only the study team can confirm whether you meet the criteria.

When should you talk to a doctor about clinical trials?

Raise the topic with your doctor if your condition has no single approved drug treatment – the case for 95% of rare diseases. Ask also if standard options have stopped working, or if a new medicine is still under evaluation and might be available through a study. For parents, the question of age is relevant: paediatric development now covers children aged 0 to 17 years, so trials exist for groups that were previously excluded^[1].

Remember that a treatment tested in a trial is not the same as an authorised medicine. Dexamethasone helped only patients who needed breathing support, not those who did not. Hydroxychloroquine, Kaletra and Kevzara fell short in studies despite early expectations^[5]. A trial is how you find out – not a guarantee.

Summary

The medicines and therapies described here reached patients because someone designed a study, a regulator assessed the data and a patient agreed to take part. Some are authorised products, such as the CAR T-cell therapy approved after trials for adults with an aggressive blood cancer, or the first treatment for Friedreich's ataxia. Others are still being tested, such as the CAR T cell therapy trial in multiple sclerosis or the study of nirogacestat in desmoid tumours^[6].

Three practical points to take away. First, check the public registers and ask your doctor whether an open study matches your situation. Second, if you join a trial, follow the study team's instructions on visits and reporting – that is how the data becomes reliable enough for regulators such as the EMA and the CHMP to assess^[4]. Third, treat a negative result as useful information: knowing that a medicine does not help a given group is what protects patients from ineffective treatment^[5].

1. [1] Clinical Trials and Rare Diseases (accessed 2 June 2026) — https://health.ec.europa.eu/document/download/b9745898-264c-408b-97fe-ccd8a9331089_en
2. [2] ATMP – Advanced Therapy Medicinal Products on the Rise (accessed 2 June 2026) — https://zweijahresbericht-2021-2022.pei.de/jb2/EN/regulation/regulation-node.html?pos=6
3. [3] Summary report on the EMA workshop on stem cell-based therapies (accessed 2 June 2026) — https://www.ema.europa.eu/en/documents/report/summary-report-ema-workshop-stem-cellbased-therapies_en.pdf
4. [4] Human Medicines Highlights - August 2026 (accessed 2 June 2026) — https://ec.europa.eu/newsroom/ema/newsletter-archives/78235
5. [5] Ramsey Pflanzer L, Dunn A. Drugmakers are racing to use existing medicines to fight the coronavirus. Business Insider. 2020. (accessed 2 June 2026) — https://www.businessinsider.com/list-coronavirus-treatments-tested-in-clinical-trials-2020-4
6. [6] More UCLH patients getting early access to new treatments via research (accessed 2 June 2026) — https://www.uclhospitals.brc.nihr.ac.uk/news/more-uclh-patients-getting-early-access-new-treatments-research