How to Get Innovative Treatment When You Don’t Qualify For The Clinical Trial
Here at FindMeCure, we aspire to give you a nicely summarized info about clinical trials, drugs, and treatments in development and what kind of opportunities our database comes up with. However, there are cases when a potential cure is not (currently) being tested in an open clinical trial or people in dire need of the drug’s health benefits do not meet the criteria of the clinical trial even when the potential effects might turn out to be life-saving. In such cases, you might want to consider what is known as “expanded access”.
What is expanded access? Also referred to as compassionate use or alternatively, pre-approval access, expanded access is when an investigational drug is used outside of a clinical trial when there are people with life-threatening conditions who can’t enroll into the clinical trial because they don’t meet the criteria. In such a scenario, there are some propositions that need to be present in order for the use of the drug to be allowed: the drug or therapy should be proven safe for use outside of the clinical trial; the potential benefits should justify the potential risks and any such risk should be reasonable considering the disease treated; no other drug or therapy could be used as a comparable alternative to the IND (investigational new drug), and the drug developer should agree to provide access to the drug.Find a Trial
It is important to note that INDs have not been proven safe for casual use in the general population by the FDA (for the US) and so a potential cure might be effective or it might not give any positive or sufficient results at all. So, when considering requesting expanded access, patients should keep that in mind and weigh the potential risks against the expected benefits.
Keep in mind that depending on the country you live in, there might be slight variations in the following: regulations and criteria when applying for expanded access, considerations both on the side of the drug developer and your physician, application forms and so on. If you consider expanded access an option for you or a relative, it is best to talk to your physician first as they should be informed about the specific situation in your country.
Ok, but what if you qualify neither for a clinical trial nor for an expanded use program? Then you can apply for the so called “single patient access”. In such a scenario, your physician can ask the company developing the drug if it agrees to supply it, and if it does your physician and the company together can “ask permission” so to speak from the FDA. In emergency situations, the FDA can even complete all the paperwork required in 24 h. However, keep in mind that this process could also take a lot of time – your physician connecting with the company, providing all the information and consent-forms needed, getting FDA’s approval. Different drug companies have different politics and sometimes they don’t have that extra amount of the medicine being tested to allow expanded access even for a single person. Another thing to consider is
Different drug companies have different politics and sometimes they don’t have that extra amount of the medicine being tested to allow expanded access even for a single person. Another thing to consider is cost: while some companies can and will supply the drug for free, others charge for it and most insurance companies don’t cover such expenses (again, this depends on your country). So, yes, it happens, but as a kind of a last resort – look for expanded access programs first and after having exhausted your options, consider single person access.
Unfortunately, expanded access is not well documented yet and so far there are no statistics about how many people resort to it and for how many of them it works. But with anything else, we can all help change that by first talking about it and making it into a big enough issue so it demands research.
[…] “How to Get Innovative Treatment When You Don’t Qualify For The Clinical Trial” is a post from the 25th of August, which opens up the discussion of “expanded access” (also found under “compassionate use” or “pre-approval access”). Expanded access is “when an investigational drug is used outside of a clinical trial when there are people with life-threatening conditions who can’t enroll into the clinical trial because they don’t meet the criteria” and to learn more about it, we recommend reading the whole article and sending it to someone who might need it. It also goes into that “but what if I don’t qualify for expanded access either?” territory and gives you some info about what “single patient access” means. […]