LIFE CARE PLANNING: What If Drugs or Procedures Needed Aren’t Approved in the USA?

Sandra W. Reed Life Care Planning

Jae Lee, a thirty year old Korean, in graduate school in the United States, had been suffering severe back pain. Doctors advised him the cause was a disc in his lower back that was impinging on a nerve and that surgery was needed to remove the offending disc. Instead of submitting to the surgery, Jae returned to Korea to get a second opinion. The doctor he consulted there told him the Korean medical profession no longer considered surgery the best treatment. The doctor believed that removing one disc would likely expose other discs to added stress, ultimately resulting in nerve invasion at a different level on the spine. The Korean physician prescribed a procedure in which an instrument much like heart catheters used here would be run up Jae’s spine to the nerve. With an attachment to this instrument, the nerve would be moved away from the disc so it no longer caused any pain. Jae had the operation and has been pain free for five years.

Plagued with constant pain and decreased mobility in her knees, Anna Watson had been told she needed a knee replacement, but was not a candidate at this time because of a blood-clotting condition she suffered. Mrs. Watson had heard of a procedure, available in Puerto Rice but not approved in the U.S., in which stem cells are injected into the knee to restore knee function without the necessity of a knee replacement. Travel to Puerto Rico would expose Mrs. Watson to a risk of blood clots as well. If the procedure had been available in Texas, Mrs. Watson could have taken advantage of it safely.

Karen Katz had landed in the emergency room several times in the last year due to asthma. As a result, her doctors had advised her to quit work and file for Social Security disability. None of the treatments she had tried over the years had controlled her chronic pulmonary problems. Karen, who loved her job, was desperate to find something effective enough to allow her to continue working. A colleague mentioned to Karen she had read about a new asthma study. The study was testing a new drug designed to reduce symptoms for those whose asthma was triggered by allergies. Karen contacted the research institute and was admitted to the study. Karen noticed some improvement after the initial injection and after three dosages, she was asthma-free. Although the drug hadn’t been approved by the FDA, as long as Karen was involved in the study, she had access to the drug.

Drugs and Procedures Not Readily Available in the United States

The first two examples above illustrate situations where treatments not available in the United States are available elsewhere in the world. In some instances, patients can travel to other countries and consent to a treatment there.

Patients who bring into the U.S. drugs legally prescribed in other countries but illegal here risk criminal charges. As in Karen Katz’s case, often the best route to obtaining an unapproved drug or procedure is to participate in a clinical trial testing it.

The Food and Drug Administration (FDA) assesses whether the benefits of new drugs and medical devices outweigh their risks. According to The New England Journal of Medicine, the average development time for these ranges from 2 ½ to 8 years. This lag time can be devastating to patients who are nonresponsive to approved treatments as well as to those patients with medical conditions for which no treatment had previously been devised.

Expanded Access to Unapproved Drugs

The FDA has attempted to address the lag time problem by allowing patients and physicians to petition for access to unapproved drugs. In granting a request, the FDA must determine that the condition is serious or immediately life-threatening, that no similar alternative therapies exist and that access will not interfere with clinical trials. For individual patients seeking to use an experimental drug, the FDA will allow usage if the drug does not impose a greater risk than the disease itself.

Most interestingly, The New England Journal of Medicine reports that the FDA has permitted almost all expanded-access requests. More good news: many companies do not charge for the drugs. The FDA allows companies to charge only the direct costs of the expanded access program. When the drug is approved, the price charged will be much higher than this direct cost. By not charging at all, the companies avoid having this disparity made public.

Impediments to Expanded Access

The manufacturers are not required to make the drug available simply because expanded access is requested. Most patients must make the request for expanded access themselves because physicians are often reluctant to take on the task because it will take approximately 8 hours of their time to complete the paperwork.

Legal challenges arguing that the FDA regulatory system violates patients’ constitutional rights have failed, with the Supreme Court finding no constitutional right to access to experimental drugs. Some patients who participated in clinical trials have been successful in asserting contractual rights to continued access to the drug after the trial ends but before the drug has been approved.

Colorado, Missouri, Louisiana, Michigan and Arizona have passed “right-to-try” laws attempting to override the FDA restrictions. These laws have their limits, in that some do not require the manufacturer to supply, some do not require the physician to recommend the drugs and some do not shelter physicians and manufacturers from liability. Furthermore, if the constitutionality of these laws is challenged before the Supreme Court, under the so-called “preemption doctrine,” the court is likely to rule that the state laws are of no effect because they are in conflict with federal statutes or regulations.

Sandra W. Reed is an attorney with Katten & Benson, an Elder Law firm in Fort Worth, Texas. She lives in beautiful Somervell County, near Chalk Mountain. If you have questions or wish to suggest a topic of interest, please contact her at 254.797.0211 or at