An Institute of Medicine (IOM) study released in January calls for stronger Federal leadership and a long-term commitment to supporting research to spur the development of medications to treat drug addiction.
The congressionally mandated study took a broad look at current conditions for the development of medications to treat opiate and cocaine addiction and found that they are largely unfavorable. For instance, a scarcity of basic scientific knowledge about the biological mechanisms that underlie addiction as well as a complicated web of State and Federal regulations present significant obstacles to private sector activity in this area. As a result, since methadone's approval in the 1960s, the Food and Drug Administration (FDA) has approved only two additional medications to treat people addicted to heroin and other opiates. And, so far, no medications have been approved to treat cocaine addiction. The NIDA-funded study, Development of Medications for the Treatment of Opiate and Cocaine Addictions: Issues for the Government and Private Sector, recommends ways that NIDA and other Federal and State agencies could remove these obstacles.
NIDA officials expressed optimism that the IOM's comprehensive evaluation would lead to a better climate for developing drug addiction medications. "We believe that it highlights the factors, from a governmental, academic, and private-sector point of view, that are critical for the success of antiaddiction medication development, including those areas in which NIDA can be more effective," said Dr. Charles Grudzinskas, director of NIDA's Medications Development Division (MDD).
The report praised the progress that NIDA's medications development program has made since its inception in 1990 and cited the following program achievements as being "major accomplishments for a young organization":
- completion of the evaluation of LAAM (l-alpha-acetyl-methadol), the newest of three currently available opiate addiction medications. NIDA's New Drug Application for LAAM received FDA approval in just 18 days;
- evaluation of buprenorphine and several other opiate addiction medications in major clinical studies;
- establishment of a screening program to identify potential cocaine addiction medications;
- establishment of a network of experienced clinical investigators; and
- establishment of an excellent working relationship with the FDA.
The report called for stronger leadership from the highest levels of the Federal Government in promoting the use of medications to treat drug addiction. NIDA, in particular, should intensify its efforts to stimulate private-sector development of these medications, the IOM report said. A big step in this direction would be expanding the drug addiction knowledge base, paying particular attention to improving the scientific understanding of the biological processes involved in addiction. The report noted, however, that recent budgetary constraints have restricted the ability to demonstrate this kind of leadership.
Further research progress would be made if NIDA were to designate national drug abuse research centers that would integrate all aspects of drug abuse prevention and treatment research, the report contended. Such centers could take a broader approach to addiction medication research and would have more flexibility to pursue new research leads.
The report also called on NIDA to evaluate the medications development programs of other NIH Institutes. "Utilizing those methods of medication development used successfully by other NIH Institutes has always been very high on the NIDA medications development agenda," said Dr. Grudzinskas, who noted that MDD staff have been consulting with officials from the National Cancer Institute's Developmental Therapeutics Program and the Developmental Therapeutics Branch of the National Institute of Allergy and Infectious Diseases.
The IOM report cited the achievemments being made by NIDA researchers in developing medications to treat drug addictionThe IOM report also recommended changes that would make the regulatory and market environments for medications to treat opiate and cocaine addiction more appealing to the private sector. For example, current FDA regulations permit new medications that are being studied to be distributed outside of controlled clinical trials to treat patients with life-threatening diseases. The FDA also sanctions the distribution of experimental drugs to treat AIDS patients who no longer respond to standard therapies and who are not able to participate in clinical trials. The report recommended that these distribution opportunities be extended to experimental treatments for drug addiction.
The IOM report noted that these measures could encourage private sector development of addiction medications by allowing companies to begin to recover research and development costs earlier in the approval process. They also would provide companies with an early opportunity to familiarize patients and physicians with their products.
The report also encouraged the FDA to grant orphan drug status to addiction medications that meet the criteria for this designation. As currently defined by the FDA, orphan drug status confers exclusive marketing rights to companies that develop medications to treat diseases that affect 200,000 or fewer individuals. Many pharmaceutical companies now believe that the market for medications to treat cocaine addiction is too small to justify the expense of research and development. Granting orphan drug status to potential cocaine medications could stimulate their development, the report contended.
The IOM report recommended that Federal and State authorities work together to devise a more coordinated approach to regulating addiction medications. Because States can enact stricter or additional requirements beyond the minimum standards of Federal law, there are significant variations in statutes from State to State, and numerous differences between Federal and State provisions. This creates confusion for pharmaceutical companies and presents another major impediment to the discovery, development, and marketing of these medications.
Assistant Secretary of Health Dr. Philip R. Lee has asked an interagency committee to review the IOM report and make recommendations for addressing the regulatory issues that affect the development of medications to treat addictions.
"The committee will consider the IOM's recommendations that pertain to the tiers of Federal and State regulation in this area," said Nick Reuter of the FDA's Office of Health Affairs and the committee's executive secretary.
The committee was to report its findings to Dr. Lee by the end of March 1995, said Reuter.
The IOM study was mandated by Congress in 1992 when NIDA was transferred from the Alcohol, Drug Abuse, and Mental Health Administration to the National Institutes of Health. The IOM is part of the National Academy of Sciences, which was chartered by Congress in 1863 to advise the Federal Government on medical care, research, and education.