News comes from the Food and Drug Administration (FDA) fresh at the start of 2007. Health-freedom adherents, for the most part, firmly believe that the FDA and its bureaucracy – among other things – have no common sense.  There are ample examples that support this conviction, especially when it comes to dietary supplements, nutritional foods, and the exercise of healthcare freedom of choice.  A recent decision, though, shows that the FDA does actually have the ability to make an intelligent decision, at least one out of 100 times.  The Agency has proposed to change its position on access to drugs and medical treatments, i.e., experimental or investigational new drugs undergoing the drug-approval process.

 

A proposed regulation will give people and their physicians the ability to access drugs and therapies undergoing FDA approval, but only when a serious disease or condition has been diagnosed and when no other treatment options are available.  This ability currently exists, but applies only to persons who participate in a controlled clinical trial sponsored by a drug company, and only when offered by a physician investigator participating in a sponsored study.  On average, according to the FDA, only 659 people each year have been allowed expanded access.  This regulation will change this.

 

The genesis for this decision goes back to 1987 and the FDA Modernization Act.  And, more recently, this is an outcome of Congressional lobbying by the NHF and a few other groups for the Access to Medical Treatment Act (AMTA) legislation.  This legislation would give people, under the supervision of a licensed healthcare provider, access to drugs and treatments approved and in use in European countries, Canada, and Japan but not FDA approved for use in our country.  AMTA also seeks to allow access to drugs and treatments in the FDA-approval process, but not yet approved for use for people with serious or chronic diseases or conditions.  One of the arguments for the AMTA legislation was the need to change the FDA policy addressed by this regulation.  In essence, this FDA decision fulfills one of the policy goals of the AMTA legislation, but with some qualifications/limitations not intended by the freedom-of-choice legislation.

 

This action by the FDA reflects a small success for the lobbying efforts of the NHF and other supporters of the AMTA legislation.  The AMTA bill has not passed Congress.  In communicating our concerns to the FDA, and in getting a few key members of Congress and their staffs to raise the treatment access issue with the FDA, one of the health-freedom principles set forth in AMTA should be accomplished if the FDA gives final approval later this year.  Senators Hatch, Harkin, and Grassley as well as Congressmen Burton, DeFazio, and a few other Representatives had a hand in getting the FDA to act on this issue.

 

The proposed regulation eliminates the previous barrier for access to drugs and therapies undergoing FDA approval for persons not fitting the tightly designed clinical-study protocols, nor wanting to participate in them.  It clarifies and expands the current requirements for who can have access to drugs and treatments in the Investigational New Drug (IND) approval process.  It will allow an individual physician, in consultation with a patient, to become an independent “sponsor/investigator” to an ongoing clinical trial sponsored by a company, without requiring direct participation as an investigator in a clinical trial study.  Likewise, a person would be able to receive an experimental, not-yet-approved drug or treatment, but only for serious or immediately life-threatening diseases or conditions for the diagnosis, monitoring, and treatment in these types of medical situations.  There are other requirements, such as patient informed consent, filing documentation with the FDA for an expanded-use authorization, receiving a “right of reference” from a sponsoring company, and having an Institutional Review Board (IRB) do case monitoring.

 

The regulation does not allow unfettered access to unapproved or yet-to-be-approved FDA drugs and treatments for any medical/preventative purposes.  Nor does the proposed regulation address the issue of the use of, for example, an herbal, botanical, or dietary-supplement ingredient or product in conjunction with an approved pharmaceutical drug as a new medical treatment.  Supplements are foods, but when combined with a drug for a preventative or curative purpose, a new regimen to treat a variety of medical conditions could be claimed under current policy by the FDA to be a drug-treatment protocol.

 

The regulation does – in a relatively small segment of our population – try to reconcile an out-of-date and inconsistent FDA policy with recognition for the need for individual medical treatment choices.  It may very well help hundreds or thousands of people each year who suffer a serious or terminal disease or condition.  It will give physicians more options for treating patients to improve the quality of their expected life.

 

The proposed regulation will not help the hundreds of Americans who go to Mexico, Canada, or a European country each year for a safe and effective treatment to mitigate or cure a medical condition because they and their physicians cannot have access to it in their own communities.  This issue is still to be resolved, via regulation or federal legislation.  We need to continue to advocate and lobby Congress on this matter. Otherwise, it could take the FDA another twenty years to make an intelligent decision to change policy to better reflect the real world of medical practice and the right of Americans to make their own informed decisions on health care.

 

The FDA press release and proposed regulation can be downloaded at  http://www.thenhf.com/%20Experimental%20Drug%20Access.pdf.