Financing a Phase I trial in oncology is tough, but completing a successful trial for a novel drug candidate is even tougher. You will need specialists: study investigators, statisticians, independent experts and key opinion leaders, project managers, institutional review boards, data and safety monitoring committees, quality controllers, manufacturers, suppliers, compliance and regulatory officers. Just to name a few.
Then you will need to coordinate this diverse team that is often spread across multiple sites spread over different geographies, different countries, even different continents. At last the trial will begin enrolling patients for many months in a study that likely will continue for years, which leads to a new level of complexity: recruitment and retention. The risk is that failure to retain patients results in missing data, which can subsequently interfere with the interpretation of results.
“These trials are becoming more complicated, more expensive and less predictive, which is not a good combination,” said Edward Garmey as he concluded his introduction at the opening of a workshop he organized for participants at BIO-Europe Spring® in Barcelona.
A soft-spoken pediatric hematologist-oncologist with Alacrita Consulting, Garmey said that given these complexities, we should not be surprised by the data describing the woeful inefficiency of bringing cancer drugs to market, nor be surprised that drugs that do come on the market have a price tag of USD 10,000 per patient per month.
Facing this complexity, biotech companies need the technical and clinical research expertise of partner companies and increasingly, a large hospital research network.
At the Vall d’Hebron Institute of Oncology in Barcelona, Gemma Sala is a specialist who has spent 20 years on the front lines of oncology and today leads the Phase I Trials unit with the Barcelona Clinical Trials Platform that currently is conducting 100 trials.
“In the beginning it was only chemotherapy,” she said nostalgically. “Now it is combinations, complex treatments with requirements beyond toxicity to the efficacy of biomarkers. We have trials with fast dose escalation and multiple arms for the study, we have seen changes in endpoints and increased requirements for biopsy samples that are often very difficult to obtain. Data requirements have increased while the time to complete the data has become shorter.”
Patient profiles have changed, making them more difficult to identify and recruit, she added, the length of treatments has changed, and the number of patients participating has increased. A recent Phase I trial at her center included 450 patients.
In what she called the new reality, “pharma companies need to recognize that the trial site is also an expert in clinical trials and it is important for us to be involved from the first draft of the trial protocol to help with inclusion and exclusion criteria and the logistics of the study.”
The Business Development Manager at Vall d’Hebron, Carlos López described the professionalization of clinical trial sites as centers of excellence. In Catalonia, his institution has created a network of hospitals and clinics that resulted in a 25 percent increase to the potential population of patients.
This trend extends to a larger scale with recently formed Cancer Core Europe by six leading cancer centers with a visionary approach to take international collaborative research. Joining Vall d’Hebron in this network are the Cambridge Research Center in England, the Gustave Roussy Institute in France, the Karolinska Institute in Sweden, the German Cancer Research Center, and the Netherlands Cancer Institute.
López said he has established procedure templates and research agreements for multiple pharma companies in order to standardize the clinical trial processes and define clear points of contact. Currently he is extending this effort to include contract research organizations (CROs).
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