FDA seeks to expand access to experimental drugs

A new agency proposal would also clarify when companies can charge for an investigational product.

By Susan J. Landers — Posted Jan. 1, 2007

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Gaining access to a promising but still experimental treatment can present a bewildering path to physicians and their very sick, often desperate patients. The Food and Drug Administration proposed to shine light on that trail with new clarifying rules issued last month.

The agency intends to make experimental drugs more easily available to physicians and to seriously ill patients who have exhausted all other treatment options. The proposals also clarify how manufacturers can recoup the expense of providing the still-unmarketed products to those in need, said Janet Woodcock, MD, the FDA's deputy commissioner for operations.

"There have been barriers to patients seeking access, because the provisions were somewhat confusing," said Dr. Woodcock. "We felt there was unequal access."

Patients have been able to obtain experimental treatments for decades. For instance, in 1987 the FDA created a regulatory mechanism to broaden access to drugs beyond the scope of clinical trials. Treatment INDs -- investigational new drugs -- allow those with serious and life-threatening illnesses to use such products. As a result, thousands of people with HIV/AIDS, cancer and cardiovascular disease have taken advantage of promising therapies.

Participating in a trial is the surest route to investigational pharmaceuticals, but sometimes a patient doesn't qualify for a trial or can't travel to a distant site, said Dr. Woodcock. In such cases, treatment INDs could allow access. Many large drug companies also run compassionate-use programs to provide investigational drugs to such patients.

Balancing act

In developing its new regulations, the FDA tried to strike a balance between improving patients' access, maintaining a watchful eye on safety and making sure the scientific integrity of the clinical trial process remained intact, said Dr. Woodcock.

The proposal is now in the midst of a 90-day comment period. "Depending on how much comment we receive, we may have further public discussions," said Dr. Woodcock.

Patient access is already the subject of a lawsuit brought against the federal government by the Abigail Alliance for Better Access to Developmental Drugs. The case is scheduled for a March hearing before the U.S. Court of Appeals for the District of Columbia. The suit will continue to go forward, despite the FDA's new proposal, said Richard Samp, chief counsel at the Washington Legal Foundation, who is representing the alliance. The suit argues that terminally ill patients should have the right to take drugs that cleared phase I clinical trials, show some effectiveness and carry an acceptable level of side effects.

"My feeling is that the FDA's heart is in the right place. They are concerned about access to developmental drugs by terminally ill patients. So we certainly encourage the dialogue," said Samp. However, he takes issue with the FDA being the final arbiter in such matters. "We think the FDA ought to be allowed to establish reasonable rules, but once they set those rules they should step back."

Under its proposal, the FDA would continue to decide whether an individual patient should receive an experimental drug.

Ellen Stovall, president and CEO of the National Coalition for Cancer Survivorship, was pleased to see this guidance provided in what has been a murky area, but she did not want to comment on the lengthy proposal until the coalition had reviewed it.

The FDA also proposed revising regulations that govern when it is appropriate for companies to charge for an experimental product. Current rules have proven difficult to interpret and resulted in confusion over what costs should be recovered, according to an agency statement.

"It's not about making a profit," said Dr. Woodcock. The intention is to encourage small companies and research institutes, which are often short on resources, to supply promising products to patients.

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Explaining access

The Food and Drug Administration has developed new regulations to clarify patients' access to investigational new drug treatments. The new rules would:

  • Modernize applicable regulations to include all circumstances under which access to experimental drugs is permitted, including:
    • Single patients in nonemergency and emergency settings.
    • Small groups of patients.
    • Larger groups of patients under a treatment IND.
  • Make experimental drugs more widely available by establishing criteria that link the level of evidence needed to support the use of an experimental drug to the seriousness of the disease and the number of patients likely to be treated with the drug.
  • Revise the current regulation regarding manufacturers' recovery of the costs of an experimental drug to:
    • Clarify that such charges are permissible in a clinical trial only to facilitate development of drugs that promise significant advantages over existing therapies.
    • Clarify that allowing charges for treatment use of an experimental drug is intended to facilitate and encourage access to drugs that might otherwise not be made available.

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External links

Food and Drug Administration's proposed rules on charging for investigational drugs and on expanded access for treatment (link)

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