It sounds like a good idea. Patients who are terminally ill would be allowed to ask pharmaceutical companies to try drugs that are not yet approved. If a patient who is dying has tried other medications without success, why not let him or her take the risk on unproven drugs? That is the seductive — but misleading — appeal of the proposed federal Right to Try Act, perhaps why 37 states have passed their own version of it.
But, terminally ill patients already have access to drugs not yet approved by the FDA under the “Expanded Access” program. This new proposed “Right to Try” legislation is a bad deal for terminally ill patients as it would take away some of the protections they currently have under the existing program. And more concerning, it would allow pharmaceutical companies to sell experimental drugs with no oversight.
Here are some of the key differences between the Expanded Access program and the proposed Right to Try legislation.
Under Expanded Access (already in place), the FDA receives a request from a doctor on behalf of a patient to try a non-approved drug. The FDA reviews it within 4 days (on average) and within 1 day in emergencies. It is rare for the FDA to not give an OK. Because the drug is in clinical trials, the FDA has staff members working with the drug companies on the trial. Patients can get an unbiased medical opinion from FDA scientists about the chances for success and the risks of adverse events and debilitating side effects. FDA researchers can make suggestions about how the drug might best help that specific patient.
However, the pharmaceutical company makes the decision whether to release the drug or not. There is usually no cost to the patient because if pharmaceutical companies want to charge for the drug, the company has to reveal the actual manufacturing cost. And this is not something a drug manufacturer wants to do for competitive purposes.
The FDA follows the patient’s experience with the drug so that the knowledge gained can be shared with researchers. Good outcome or bad, at least something is learned and that patient’s life had value.
Under Right To Try laws, a patient asks the pharmaceutical company directly for access to an experimental medication. The drug company makes the decision whether to release the drug. In essence, there is no “right” to try. The patient has no one to represent his or her interests or to offer a realistic assessment of the drug. With no one to monitor the conversation, the drug company could be making unlikely and unfounded claims of the drug’s efficacy. While a pharma company could be working on a cure for a specific cancer or other disease, drugs in early trials have not yet proven to be effective or safe.
Sadly, some firms are using Right to Try to game the system for their economic benefit. One company has already announced plans to use the laws to offer its stem cell treatment well ahead of approval from the FDA.
The patient who is out of options might grasp at this last chance opportunity, but it comes with risks that may not have few, if any,benefits.
Drug companies can charge any amount they want to for a drug under the legislation. Families could bankrupt themselves trying desperately for a drug that has no chance of working.
When a drug is in a clinical trial, every person who takes it adds to the knowledge of how the medicine acts. That information is used to help future patients. But in Right to Try cases, the pharma company is under no obligation to report a patient’s experience with the drug and the FDA has no knowledge of who might be taking it.
The Right to Try Act puts patients in a worse position than they are today by minimizing the FDA’s oversight of use of experimental drugs. Under this legislation, pharmaceutical companies profit at the expense of terminally ill patients and avoid scrutiny from the FDA. Pharma companies could peddle drugs that will never be proven safe or effective to desperate people at the end of their lives — and make a profit on it.
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