In the United States, only 31.4 % of phase II/III clinical trials in oncology are successfully completed1. The causes of this dramatic economic and time-consuming loss are, among others, protocol design and patient recruitment challenges.
Protocol design has become increasingly complex, often including a growing number of endpoints, eligibility criteria and study procedures. Notably, 90% of protocols require at least one substantial amendment2.
These scientific complexities are further compounded by operational challenges, such as the coordination of multiple countries and sites in a highly competitive landscape. In oncology specifically, protocols have higher executional variable values than non-oncology protocols, including the average number of countries, investigative sites and planned visits1. Nevertheless, patient recruitment also remains one of the biggest barriers to clinical trial success, often due to a lack of early patient-centered planning which leads to difficulties in enrollment and longer duration protocols (1.5 times longer in oncology than other disciplines)1.
In 2012, MEDSIR was established as a new-model company that provides end-to-end management of strategic clinical trials in oncology, from study design to publication. The concept is simple yet powerful: to transform drug development strategies with oncologist’s innovative ideas into a high-quality clinical trial.
And how does MEDSIR overcome the milestones described above? By thinking out of the box. MEDSIR-led clinical trials are originated from investigator-initiated ideas in collaboration with healthcare companies, to ensure a truly patient-centered strategy along with drug development excellence Each trial is driven by the leadership of the investigator and supported by a multidisciplinary team of experts who are involved in every step of the process. Protocol design integrates clinical trial expertise from the earliest stages, helping to prevent common pitfalls related to recruitment and study feasibility, while also supporting any amendments that may arise during the study. MEDSIR’s operational team ensures that patient recruitment is performed successfully across all sites and countries within adequate timelines. Once the study is completed, a team of data scientists conducts rigorous statistical analysis of the collected data. Lastly, results are disseminated by a specialized scientific impact team through presentations at leading international congresses and publications in high-impact journals rewarding the investigators’ team work.
Recent studies conducted within the company highlight how investigator-initiated trials have provided meaningful outcomes for cancer patients. These include therapy de-escalation strategies, as shown in the PHERGain study, and approaches aimed at improving treatment tolerability, as observed in the METALLICA and PRIMED trials.
Early breast cancer represents a large portion of diagnosed cases and about one in five are classified as HER2-positive (HER2+), a key protein present on the surface of cancer cells that promotes cell growth. Standard pharmacological treatment for HER2+ early breast cancer includes chemotherapy combined with targeted anti-HER2 therapies. The PHERGain study, investigated the possibility of safely omitting chemotherapy in a selected group of patients using a response-guided approach. Participants were randomized into two groups: group A received standard chemotherapy plus anti-HER2 drugs (trastuzumab and pertuzumab), and group B started with anti-HER2 therapy alone and chemotherapy was added only if insufficient response to the treatment. Results showed that a subset of patients with HER2+ early breast cancer can safely omit chemotherapy without compromising efficacy. These findings support the use of personalized treatment strategies in early HER2+ breast cancer. The latest results of this study were published in the prestigious journal The Lancet3.
In addition to strategies aimed at de-escalating therapy, an independent research line involves the improvement of drug tolerance. Patients positive for hormone receptors, negative for HER2 and PIK3CA mutated advanced breast cancer can be treated with alpelisib and fulvestrant after progression on endocrine treatment. However, one of the most common adverse events related to the treatment with alpelisib is hyperglycemia, which can lead to permanent discontinuation of the treatment. The METALLICA trial, showed that the addition of prophylactic metformin to the standard regime reduced the incidence rate of hyperglycemia and sustained treatment continuation. These findings reinforce the importance of personalized, preemptive toxicity management in maintaining treatment efficacy and minimizing disruptions—and ensure long-term feasibility and safety for patients.
Another standout study, PRIMED, evaluated a strategy to improve the safety profile of an antibody-drug conjugate: sacituzumab govitecan. This drug is approved in the United States and Europe for the treatment of patients with pretreated triple-negative breast cancer and HR-positive/HER2-negative advanced breast cancer. However, this treatment is commonly associated with high rates of neutropenia, a drop in white blood cell count, and diarrhea, which often disrupt treatment. By using prophylactic drugs, including the concomitant use of granulocyte colony-stimulating factor (for neutropenia) and loperamide (for diarrhea), the study achieved a reduction in the incidence of these adverse effects supporting the prophylactic use of these drugs to prevent and minimize neutropenia and diarrhea.
METALLICA and PRIMED are examples of targeting unmet needs not covered by traditional clinical trials and generated significant scientific impact with results published in eClinicalMedicine, a journal from The Lancet group4,5.
Conducting clinical trials in oncology is undeniable challenging, but there are effective ways to bridge the gap between drug development and real-world implementation. MEDSIR’s model has provided meaningful results and is trusted by over 40 healthcare companies. Studies like the ones described above and others (55 trials ongoing, 187 sites, >2455 patients treated) illustrate how MEDSIR aims to continue generating relevant real-world evidence for clinical practice and improve patient’s life.
References
1. Getz K, Smith Z, Kravet M. Protocol Design and Performance Benchmarks by Phase and by Oncology and Rare Disease Subgroups. Ther Innov Regul Sci. 2023 Jan;57(1):49-56. doi: 10.1007/s43441-022-00438-5. Epub 2022 Aug 12. PMID: 35960455; PMCID: PMC9373886.
2. Getz K, Smith Z, Botto E, Murphy E, Dauchy A. New Benchmarks on Protocol Amendment Practices, Trends and their Impact on Clinical Trial Performance. Ther Innov Regul Sci. 2024 May;58(3):539-548. doi: 10.1007/s43441-024-00622-9. Epub 2024 Mar 4. PMID: 38438658.
3. Pérez-García JM, Cortés J, Ruiz-Borrego M, Colleoni M, Stradella A, Bermejo B, Dalenc F, Escrivá-de-Romaní S, Calvo Martínez L, Ribelles N, Marmé F, Cortés A, Albacar C, Gebhart G, Prat A, Kerrou K, Schmid P, Braga S, Di Cosimo S, Gion M, Antonarelli G, Popa C, Szostak E, Alcalá-López D, Gener P, Rodríguez-Morató J, Mina L, Sampayo-Cordero M, Llombart-Cussac A; PHERGain Trial Investigators. 3-year invasive disease-free survival with chemotherapy de-escalation using an 18F-FDG-PET-based, pathological complete response-adapted strategy in HER2-positive early breast cancer (PHERGain): a randomised, open-label, phase 2 trial. Lancet. 2024 Apr 27;403(10437):1649-1659. doi: 10.1016/S0140-6736(24)00054-0. Epub 2024 Apr 3. PMID: 38582092.
4. Llombart-Cussac A, Pérez-Garcia JM, Ruiz Borrego M, Tolosa P, Blanch S, Fernández-Ortega A, Urruticoechea A, Blancas I, Saura C, Rojas B, Bermejo B, Ponce Lorenzo J, Gion M, Cortez-Castedo P, Llabres E, Galve E, Cueva JF, López A, Alonso-Romero JL, González-Santiago S, Martínez de Dueñas E, Ciruelos E, Martrat G, Gener P, Alcalá-López D, Sampayo-Cordero M, Gómez-Peralta F, Cortés J. Preventing alpelisib-related hyperglycaemia in HR+/HER2-/PIK3CA-mutated advanced breast cancer using metformin (METALLICA): a multicentre, open-label, single-arm, phase 2 trial. EClinicalMedicine. 2024 Apr 11;71:102520. doi: 10.1016/j.eclinm.2024.102520. PMID: 38638399; PMCID: PMC11024566.
5. In Press. https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(25)00241-X/fulltext
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