If people with rare diseases or incurable diseases do not qualify for inclusion in a clinical trial who otherwise appears to offer hope in their condition, consider the differences between the “extended access” and the “right to experiment” routes Understanding experimental drugs can become a priority for both patients and their doctors.
Understanding the differences is also important for those doing the studies, says Preethi Sriram, DHSc, MSN, BSN, a clinical researcher in Raleigh, NC who recently wrote for Clinical researcher Information on reused medication, advanced access / compassion programs, and other options for those with difficult-to-treat conditions to consider when participating in a standard clinical study is not an option.
“If a person does not meet the criteria for a clinical trial or does not meet the schedule for registration, or if a study is not available for their specific condition, there appear to be no treatment options other than the traditional standard of care, especially for those with rare diseases can be inadequate, ”says Sriram.
However, the US Food and Drug Administration (FDA) notes that expanded access, sometimes synonymous with the term “compassionate use,” is a possible avenue for people with rare diseases and / or life-threatening conditions to Access to medical research products, especially when no analogous alternative therapy is available. A condition of such access, according to Sriram, is that such treatment outside of the normal protocol of a clinical trial must not interfere with the normal development or marketing authorization of a product.
However, there is no guarantee that an extended access request will be granted. As such, patients may need a Plan B. “While the enhanced access / compassionate use process requires FDA approval, experimentation law bypasses this process and allows an individual patient or healthcare provider to contact a company directly for access to get his experimental treatment, ”says Sriram. Although not without complications of its own, the law allows manufacturers of an investigational drug to make it available to authorized participants through a cooperating physician without the risk of liability.
“To gain access, the person must meet requirements that are similar to the conditions for extended access. These include having a life-threatening illness, exhaustion of all other treatment options, inability to participate in a study of the product, and giving written consent to the healthcare provider to use the investigational drug, ”explains Sriram.
While the Trial Law Act states that the use of the investigational drug is exempt from FDA review or approval requirements, the drug’s sponsors and manufacturers must report the number of doses delivered to the agency, individuals treated, indications who the drug was used for and possible serious side effects. Sriram states that the FDA is expected to make this information available to the public.
“In either strategy, the sponsor or manufacturer can refuse access to the drug out of concern about possible negative results,” warns Sriram. However, she notes that the FDA is not aware of any cases from a literature review where adverse event information from the extended access ever prevented the agency from approving a drug and that the reviewers of the data understand the context in which the advanced access is performed and recognizes that it is outside of the stages of a controlled clinical trial environment. Regarding the cost to patients, she adds, “The cost of treatment for drugs used in these situations can be very high and insurance companies will not cover them even if they have been approved for use.”
Their review of the literature on the subject also shows that pharmaceutical companies are generally more familiar with the extended access / compassion pathway than the right to try. “Who actually gets the drug in these situations seems arbitrary and that should be an area for further study,” she recommends.
Edited by Gary Cramer