Everyone wants their prescription medicines to be both highly effective and 100 percent safe.
But as this is clinically impossible in the treatment of our toughest diseases–cancer, Alzheimer’s, heart disease–there will always be trade-offs.
A drug that can save your life may also have a side-effect, and even well tested prescription drugs can affect different people in different ways, especially in already fragile patient populations.
So, who decides what risks are acceptable, and which drugs are approved and remain available to the public?
The answer, of course, is the federal government.
The U.S. Food & Drug Administration has full authority over which prescription drugs are available to whom, and how they are marketed.
And, to be clear, there is an essential need for strong federal oversight of the nation’s pharmaceuticals approval system.
But what are the roles and rights of patients in collaboration with their doctors to have a say in what risks they will assume? A growing number of people fear these rights are being ignored as our federal healthcare bureaucracy grows larger and more powerful.
The most recent example of this dilemma was the breast cancer drug, Avastin. Over loud protests from the patient community and scores of oncologists, late last year the FDA cited safety risks in its withdrawal of the drug as a treatment for women with metastatic breast cancer.
Patients and doctors wanted the drug to remain an option for women with late-stage, advanced cancer, many of whom had little other hope. But the FDA deemed it too risky.
We are seeing this now in the treatment of Multiple Sclerosis, a disabling disease affecting 400,000 Americans that worsens over time and requires aggressive pharmaceutical treatment to allow patients to live their most productive lives for as long as possible. Promising new treatments have emerged in recent years and more are in the pipeline.
But both in Europe and the U.S., regulatory agencies are tightening their control over access and limiting the ability of patients and physicians to make informed decisions based on an assessment of risk and benefit.
The new MS drug Gilenya was recently approved by both FDA and the European drug approval agency, and for the first time patients could take their medicine orally, versus an injection.
A 25,000 person clinical trial demonstrated safety, but among the thousands of initial Gilenya patients, one died within 24 hours of taking the drug.
Regulators in the U.S. and Europe are reviewing the case now to determine whether this was merely an unfortunate coincidence in a patient with severe disease and other health problems or if it represents a legitimate risk.
Even if some increased risk is established in the case of Gilenya or other therapeutics, patients and their doctors should maintain a significant role in assessing, accepting or rejecting that risk.
And FDA should ensure that this growing chorus of patients and caregivers who want into the decision-making process are given that chance.
It is a legitimate function of government to safeguard us from unsafe products. It is a disservice of a government grown too powerful to deny us the opportunity to make informed, private decisions with our doctors on the risks we are willing to take to live healthy, fulfilling or longer lives.
Kerri Toloczko is senior vice president for policy at Institute for Liberty and Director of its Center for Healthcare Security and Access.
