- We should look at cancer, which until recently was incurable. In the 1970s we had 40 cancer drugs registered, now about 15 are approved annually in the U.S. In the beginning it was difficult to deal with malignant tumors, and now we have programs that aim to completely eradicate the disease," stressed Prof. Richard Pazdur of the U.S. Food and Drug Administration (FDA).
As Prof. Grzegorz Nowakowski of the Mayo Clinic pointed out, there are already a great many therapies available to improve patients' lives. Their efficacy profiles are increasing, and availability is also increasing, as these therapies are less costly. He cited as an example lymphomas, in which chemotherapy is already being abandoned in favor of immunotherapy. However, there would be no such changes were it not for the fact that new drugs are being developed and tested more and more efficiently.
- We need to conduct extensive evaluation of clinical trials. Patients often say they are participating for the sake of science. They shouldn't think so. They are doing it for themselves. Diseases that were recently incurable are already becoming chronic," Prof. Nowakowski pointed out.
According to Prof. Beata Jagielska, director of the National Institute of Oncology in Warsaw, access to modern therapies in Poland has changed dramatically over the past few decades.
- Of course, it will never be the case that we can provide access to all the drugs that come along and are registered. Even if a drug is registered, it may not be covered by reimbursement, and it may not be as effective in the general patient population. That's what phase four trials are for, allowing us to see in a broad population how a drug really works, what toxicity and side effects it has," she pointed out.
- Clinical trials are the part of our business that allows us to qualify patients for modern therapies when we have exhausted our treatment options. We look for molecular abnormalities and try to direct patients to treatments that target the molecular abnormality," Prof. Jagielska added.
NIO-PIB in Warsaw has an Early Phase Research Branch dedicated exclusively to oncology, which was established in 2017 and was the first unit in Poland to conduct early stage clinical trials. Heading the Early Phase Research Unit, Prof. Iwona Ługowska, Plenipotentiary of the NIO-PIB Director for International Cooperation, explained that the possibility of testing new drugs in early phase clinical trials is an opportunity and hope for patients on the one hand, and a great unknown on the other.
- It's an unknown in terms of safety, but also in terms of response to treatment. When patients come to us, they don't want to know if the therapy is safe. They want to know if this therapy will cure their cancer. It is important that the patient needs to understand what kind of study they are participating in. We want to be effective at what we do, so these studies require a very high level of commitment. In the first few weeks, patients must be monitored very closely. A unique relationship is formed between them and us, because they usually come to us at a stage that precedes hospice treatment, but they are people in very good condition. It is very important that we can help people who are in great shape, but have few therapeutic options left," said Prof. Iwona Lugowska.
- The best therapies that patients can get are treatments in the clinical trial area. They are still misunderstood only as a last treatment option. We have trials that support the first, second or third line of treatment. They support and accelerate the pace of the patient's treatment," Prof. Grzegorz Nowakowski pointed out.
The challenge in the context of clinical trials, on the other hand, is undoubtedly the coordination and harmonization of the regulatory framework, at least at the European level.
- We still have differences from region to region. However, over the years we have also seen a lot of common ground when it comes to the regulatory framework. As early as 20 years ago, there were departments that worked to unify the therapy evaluation process. We should carry out unification work wherever possible. Coordinating and unifying the framework at the European level is also a very important step towards unifying it globally," pointed out Prof. Francesco Pignatti of the European Medicines Agency (EMA).
As Prof. Richard Pazdur added, it is important to emphasize that patients participating in clinical trials can feel safe, because at the preclinical stage, new drugs are thoroughly tested, in a way that is also strictly regulated. This helps ensure that the drug is indeed suitable for treating a particular disease.
