Outcomes # 1

Patient-Centered Outcomes in the Clinic

Original article published on Assay Depot Blog, on 26 Sep 2013 here.

In different regulatory and research settings, it is now being realized that the need to consider patient-centered outcomes is paramount. This three-article series will discuss in brief patient-centered outcomes in clinical research and practice.

Personalized medicine is here to stay. It entered the scene of clinical medicine with a bang, made possible by the tremendous and rapid advancements in the “omics” fields, most notably genomics and proteomics. Though game changing in most fields of medicine, personalized medicine is profoundly affecting clinical cancer management. The main reason for this is the genomic revolution that is changing our understanding of the pathophysiology of cancer and making it possible to design novel therapeutic strategies.

Based on our understanding that cancer is a disease characterized by rapid and uncontrolled cell growth, patients have traditionally been treated with cytotoxic chemotherapy drugs. These are drugs affect all rapidly dividing cells and thereby control growth of cancer cells. These agents are effective in many cancers but also have a number of serious and dose-limiting adverse effects. Further, cancers tend to develop resistance to these agents. Finally, not all patients respond to these drugs in the same manner.

A major reason why cancer patients respond to drugs differently is the great deal of heterogeneity even in the same types of cancers. Owing to comprehensive genomic and proteomic profiling, it is now possible to obtain detailed molecular features of a tumor. Based on these data, it is obvious that different proteins may drive cancers in different cells and these proteins can in turn be targeted clinically.

For personalized cancer therapy, therapy is tailored to the individual by using the newly developed molecularly targeted agents. As the name suggests, these drugs act on molecular targets in the cancer cells and prevent or slow down the growth of cancer cells. When selected appropriately, these new drugs can be more effective than a blanket treatment with a bunch of cytotoxic drugs. The search for the so-called magic bullet for cancer (to quote the famous scientist Paul Ehrlich) led to development of these targeted agents.

Most oncology drugs being developed, including the molecularly targeted drugs use survival as an endpoint to determine clinical efficacy. While this is desirable in order to increase the survival rates in cancer patients, it ignores a key aspect of patient care. These drugs are not tested to determine quality-of-life issues for patients. There is little consideration for how a patient “feels” and whether the drug improves the well being of a patient apart from increasing survival. This is critical, especially today considering how many patients survive cancer or live long lives with cancer. This has spurred a new focus on patient-centered outcomes in drug development and clinical management of cancer.

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