The U.S. House of Representatives just passed a bill regarding the right of terminally ill patients to try medication outside of the FDA program of expanded access.
In signing it into law, President Trump hails this as a victory for patient rights, and a way to simplify the process of getting medication to people who need it the most.
But is it?
Since 2009, the FDA has already had a program that has allowed for the use of research medications for those who are not eligible to receive the medication from a clinical trial, or for which no trial exists at that time.
Congress has just made it a little easier for the terminally ill to access experimental drugs, but the new law raises a lot of questions.
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This provided for the collection of data and careful maintenance of records in order to avoid experimentation on the sick and allow for useful information to be shared with those doing the clinical trials and the company that provided the medication. Strict protocols had to be provided, and dosing that was appropriate for the patient given their specific circumstances.
Almost all of the compassionate use applications were granted from the onset of the program through the end of last year.
The main requirement was that the patient already had the agreement of the company who made the drug and also a physician to prescribe it, along with documentation of no other alternatives. The risk of using the medication had to be less than the risk of not using it, and for terminally ill patients, this meant that there needed to be some hope of success. The medication, if made available, would be at a cost to the company or to the patient, as the FDA has no authority to regulate the coverage of such experimental medications to either CMS or to private insurance.
The new proposal brings several elements into question. The first is how pharmaceutical companies will respond to the requests to use their medications out of a study protocol or the usual FDA route. There is nothing mandating company cooperation for either program. The resources it takes to provide the medication may not be significant for a large company but this diversion of labor would be a big problem for a smaller company.
Although there is no liability to the company for the use of the medication, it’s not clear if the potential for bad publicity might make companies reluctant to even disclose what they are working on.
The definition of terminal illness is also at issue. In the medical world, terminal means having six months or less to live. But other chronic conditions could be considered terminal. Genetic disorders, even heart failure, could apply, and the current law doesn’t specifically define the timeline for terminal illness. FDA director Scott Gottlieb asked for further clarification in the final version of the bill.
The third question is the cost, not just for the medication, but for any complications that arise from the use of an experimental drug. Granted, the patients involved already have a limited life expectancy, but if serious complications arise, then it may be associated with significant costs to manage these, and most insurance companies do not cover experimental treatment in the first place.
With the unknown list of potential side effects, it’s not clear if there would be additional insurance coverage for complications from this type of treatment. Mandating coverage wouldn’t be fair to everyone else who pays premiums that go up annually for FDA approved care. Now they would be responsible for experimental complication coverage.
In addition, there may not be any clear knowledge of how to manage possible reactions to medications, especially with some of the newer genetic therapies that could be provided. Providers wouldn’t be liable, but might not have the knowledge to manage issues that arise in this uncharted territory of care. Without clear published research protocols, there may not be any references to the best course of action for unexpected complications.
Although patients are trying these therapies at their own risk, if the treatments work well, the good news is that previously terminal patients may have longer to live. That leads to the question of how to get the medication on a long-term basis.
Currently costs are to be paid by the patient or by the company on a pre-arranged basis. But if it does show success, this may limit the right to try to those who have the money to ask, creating another level of inequality of medical care in the U.S. This has not been addressed in the “right to try” legislation or the current FDA expanded access program.
Eliminating the red tape of getting a medication to those who need it the most seems like a great idea. But to do so without creating additional safeguards will only lead to problems down the road.
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The post Health Beat: Beware Of This Dicey New Approach To Experimental Drugs appeared first on Honolulu Civil Beat.