Pfizer and other industry members are joining a push in favor of personalized medicine, a method of treatment which offers many advantages for clinicians and patients. Personalized medicine reduces health care costs, cuts the time it takes to bring drugs to market, and includes the ability to make more informed medical decisions and a higher probability of desired outcomes through better-targeted therapies.
According to Mace L. Rothenberg, M.D., senior vice president of clinical development and medical affairs for the Oncology Business Unit at Pfizer’s Global Research and Development, personalized medicine is well-suited for the treatment of cancer.
“Personalized medicine is a way to get drugs to market quicker and more efficiently, without the need for giant trials, taking in all comers, that have a high risk for failure,” Rothenberg said.
In the relatively young field of personalized medicine, physicians make the treatment of the disease as individualized as the disease itself, acknowledging that diseases can be as unique as the people who have them. By identifying genetic, genomic, and clinical information, physicians can accurately predict a person’s susceptibility of developing disease, the course of disease, and a patient’s likely response to treatment.
According to Alison Ayers, executive vice president of commercial development for Pfizer, in the future treatments will be personalized depending on which genetic mutations are found inside tumors.
“Oncology drug development typically takes 12 years from the first time a drug is tested in humans to achieving regulatory approvals. The development of personalized medicines should reduce this timeline by a few years,” said Ayers.
More Specific Trials
Typically, the early stages of drug development are spent searching for the tumor type and patient population where the drug works the best, requiring numerous exploratory trials to determine where the new treatment is going to have the greatest impact. Under personalized medicine, by contrast, researchers start with an idea of which patients the drug is most likely to benefit.
“We know what genetic malfunction we are addressing. Therefore, we should be able to cut down the phase I and phase II process because we will start development focusing on patients who have that specific genetic defect or biomarker,” Ayers said.
Although the early development phase time should be reduced, the phase III time may not necessarily be shorter, Ayers explains.
“We may be able in phase III to have fewer patients because we are expecting to show a much more dramatic effect and we won’t need as many patients for statistical significance,” Ayers said. “But if the biomarker is rare, we will need to screen more patients to find ones who will be eligible to go into the trials.”
Even with those limitations, however, Ayers maintains more targeted therapies for patients who have a specific molecular defect could cut years off the development process.
Adapting to FDA Requirements
Personalized medicine still has to go through the same Food and Drug Administration (FDA) safeguards as any other treatment, with comprehensive preclinical and clinical packages of data, a full safety database, and clinical study results in a significant number of patients.
The patients who have the genetic trait for which the personalized medicine is available will likely be the big winners under this new approach. Personalized medicine enables researchers to go right to the likely best treatment for a particular patient.
“For cancer patients, the first treatment offers the best chance of success. Personalized medicine allows researchers to identify the drug most likely to help them the first time around and that will have the greatest impact on survival,” said Ayers.
FDA Process an Obstacle
Currently there are only about 12 personalized medicines for cancer, but more are expected soon. One barrier is the FDA’s requirement to test a drug on each different type of cancer to obtain an approved indication.
“Development in each tumor individually may be impossible,” said Ayers. “We will need to have an active dialogue with the FDA to address what is the best approach to get these drugs to the patients who need them.”
Wave of the Future?
Grace-Marie Turner, president of the Galen Institute, notes getting a drug approved by the FDA is extraordinarily expensive and time-consuming. She says personalized medicine offers a solution.
“Personalized medicine is the wave of the future, and there’s no place where it’s more important than cancer care,” says Turner. “Personalized medicine provides the maximum benefit to cancer patients and the minimum of financial loss to drug developers. It’s not only humane but it provides huge benefits in terms of effective treatment and reduction of costs.”
Regarding whether there will be a place for personalized medicine under President Obama’s health care law, Turner expressed concerns regarding the Patient-Centered Outcomes Research Institute, a new agency modeled after Great Britain’s National Institute for Health and Clinical Excellence.
“Public policymakers should be doing everything possible to move personalized medicine forward, instead of setting up barriers,” Turner said. “British ‘clinical excellence’ involves government officials determining whether to allow access to new drugs and treatments for its citizens. But cancer treatment requires more personalized medicine and care than just about any other disease.”
“Will there be a place for personalized medicine and drug development under ObamaCare? It’s much less likely that we’ll have it,” Turner says.
Kenneth Artz ([email protected]) writes from Dallas, Texas.