FDA Eases Rules on Access to Experimental Drugs

October 8, 2009

Under new FDA rules going into effect this month, more patients will be able to access experimental drugs without taking part in clinical trials. The new rules are meant to clarify a formal process in place since 1987

Under new FDA rules going into effect this month, more patients will be able to access experimental drugs without taking part in clinical trials. The new rules are meant to clarify a formal process in place since 1987 for releasing experimental drugs to patients in dire need. Janet Woodcock, head of the agency’s Center for Drug Evaluation and Research, explained: “We have always been afraid that only those in the know about this can get these drugs.” With the now-formalized rules, first issued as a draft proposal in December 2006, “we believe a large number of clinicians, investigators, patients, and patient groups who weren’t totally aware of these programs will now become aware of them and will have access,” Dr Woodcock said.

In addition to clarification of the standards-and creation of a Web site to explain the program to laypersons-the agency is making the standards less restrictive. For example, the standards now apply to anyone with a serious disease “regardless of whether the patient would currently be considered seriously ill with that disease.” The FDA estimates that under the new rules, perhaps as many as 3000 patients annually would get experimental drugs outside of clinical trials. That would be 5 times the current number but still few enough to prevent “disrupting the gathering of information,” FDA chief Margaret Hamburg assured drugmakers anxious about the new rule’s effect on clinical trial participation.

Besides gaining agreement from the maker of the drug, patients who want the experimental medication will also have to convince the FDA that they are facing a grave medical condition for which there is no reasonable treatment alternative. The rule also opens the way for the FDA to give approval for an entire group of patients to receive the new compound. For the first time, the FDA is willing to let a drugmaker charge for the experimental drug-but only if it can show that the charge is no more than the cost of making the drug and administering the program.

Washington Offers Grants to Teach e-Prescribing
One of the least controversial ideas in health care reform is broad adoption of electronic prescribing and other advanced information technology (IT) as a way to cut costs and improve quality. However, despite widespread support, individual providers have been slow to adopt state-of-the-art IT, in large measure because they are unsure how to integrate it into their practices and are loathe to take time off from practicing medicine to learn, even with the promise of higher Medicare payments for those who use electronic health records.

The Obama Administration is now addressing that problem head-on with a 2-pronged approach. The Agency for Healthcare Research and Quality (AHRQ) announced in early September that it is launching a study to identify the barriers to e-prescribing and the conditions that would accelerate its adoption.

The agency says that its study will be the first to gather data from practicing physicians and pharmacists in the same community who actually use e-prescribing to see what led them to adopt it and how they use it, particularly how the systems’ information on a patient’s health plan formulary influences prescribing decisions. Interviews at 110 organizations are planned over the next 2 years.

However, the White House is not waiting for the AHRQ study results before it tries to spur health IT adoption. It is offering $589 million to set up about 70 Health Informa- tion Technology Regional Extension Centers around the country to promote the advan-tages of electronic data processing and teach providers how to use it. “The regional extension centers will offer hospitals and clini- cians hands-on technical assistance to support meaningful use of certified electronic health record systems,” explained David Blumenthal, the federal health IT coordinator. His office is also offering $564 million to establish state or regional health data exchange networks.

OIG Okays Plan to Subsidize Part D Copayment
In general, it is a violation of the Medicare law for anyone to provide to a beneficiary an inducement to sign up for a Part D plan. However, the HHS Office of Inspector General (OIG) has given its blessing to a state proposal that would do just that. The reason: the OIG concluded that there is little risk that the scheme would pressure the beneficiary to select any particular plan.

As always in its advisory opinions, the OIG did not publicize the specific identity of the entity asking for the exception, but it was revealed to be a state planning agency for addiction and mental health services. The agency wanted to encourage financially needy Medicare enrollees to enroll in a Part D plan by promising to pick up some-and in some cases all-of the copayment that is usually the patient’s obligation. Key to the OIG decision is that while the agency would help the client sign up for Part D, it would leave him or her free to select any plan, and the subsidy would not be contingent on using any particular pharmacy. Moreover, the agency will not advertise the subsidy plan but offer it only to those already using a mental health center that operates under a state contract.

The OIG reasoned that without the subsidy, those with addiction or mental health problems were likely to forgo getting prescribed drugs and thereby would impose treatment costs on the state that could well exceed the cost of the subsidy.

California Probing High Denial Rates for Health Claims
In September, California Attorney General Jerry Brown launched a probe of the payment practices of the state’s 7 largest health insurers. The investigation began with an analysis by the California Nurses Association of data from insurers that showed an overall rejection rate of 22%. “These high denial rates suggest a system that is dysfunctional, and the public is entitled to know whether wrongful business practices are involved,” the attorney general asserted.

His new investigation is independent of ongoing surveillance by the state Department of Insurance, which has scheduled a hearing on its allegations that PacifiCare violated state laws governing claims handling in at least 133,000 instances. Last year, the company paid $3.5 million in fines for similar violations. In the California Nurses study, based on reports the insurers file with the state Department of Managed Health Care (DMHC), PacifiCare had the highest rejection rate in the first 6 months of this year: 39.6%. Aetna had the lowest rate: 6.5%. (Unlike the other 6 big insurers in the state, Blue Shield of California does not report claims denial data to the DMHC and, therefore, was not included in the analysis.)

While some of the denials are for treatments not covered by the patient’s plan, far more are because they duplicate claims already paid or because the patient is no longer a member, the insurers say. “It appears that a good deal of the so-called denials are merely paperwork issues,” said Nicole Kasabian Evans, spokesperson for the California Association of Health Plans. She predicted that Brown’s probe will not show “widespread denials of insurance coverage.”

In Other Legislative and Regulatory News . . . Pfizer agreed to pay a total of $2.3 billion to settle claims that it promoted prescription drugs for unapproved uses. This is the largest settlement ever won by the US Department of Justice. The case primarily involved the drugmaker’s salespersons urging doctors to use Bextra (valdecoxib) for acute pain from causes other than the osteoarthritis for which the drug is an approved treatment. The settlement also covered allegations of overpromotion of the antipsychotic Geodon (ziprasidone), the antibiotic Zyvox (linezolid), and the antiepileptic Lyrica (pregabalin). The settlement consists of $1 billion in civil damages for federal health plan payments for unauthorized uses of the drugs, a $1.195 billion criminal fine to be paid by the parent Pfizer, and a $105 million criminal fine to be paid by its Pharmacia & Upjohn subsidiary.

The FDA accused Johnson & Johnson of “unsubstantiated efficacy claims” and concealing risk information in advertising its athlete’s foot treatment Ertaczo (sertaconazole nitrate) to medical professionals. The agency took issue with language claiming that “tinea stops here,” because the drug is not approved for some forms of the disease or for use in preadolescents or immunocompromised patients, and it pointed out that Johnson & Johnson’s own studies show the drug delivers a complete cure in no more than 25% of cases. A letter from the FDA’s drug-marketing reviewers Andrew S. T. Haffer and Catherine B. Gray asked the company to stop distributing all “violative promotional materials for Ertaczo.”

A study by the New England Healthcare Institute pegs the extra annual medical costs directly attributable to patients’ not taking drugs as prescribed at $290 billion. The analysis-a compendium of 7 medical literature reviews and interviews with 16 health care organizations-found the major problem in nonadherence is in patients with chronic illnesses, as opposed to those with an urgent medical problem. Simplifying the medication regimen-especially changing to once-a-day dosing-would improve adherence, the investigators conclude, as would better prerelease education at hospitals and pharmacies, more case management, and lower drug prices. “Better health and medication adherence is a fundamental missing link to better outcomes,” declared the executive director of the institute, Valerie Fleishman.

Americans are getting the word that there is serious risk in abusing prescription drugs. This point was made by Eric Broderick, acting head of the US Substance Abuse and Mental Health Services Administration (SAMHSA). His evidence was an SAMHSA survey released September 10 showing that last year, 2.5% of the population said they had abused prescription drugs, down from 2.8% in 2007. The figure had been rising for a number of years. The new study also showed a substantial 1-year drop in the number of persons using methamphetamine, from 529,000 to 314,000.

An analysis by the Pew Prescription Project addresses Forest Laboratories’ market-ing strategies for its antidepressant Lexapro (escitalopram). Using data from Minnesota, where drugmakers are required to report in detail their payments to health care providers, the nonprofit Pew group found that 62 doctors in the state received at least $1000 from Forest in speakers’ fees and expense reimbursements last year. The company’s payments to Minnesota doctors topped $750,000-noteworthy because Forest has a far smaller drug portfolio than the 3 manufacturers that outspent it: Eli Lilly, GlaxoSmithKline, and Pfizer. Such financial relationships should make a patient “more cautious about the way in which prescribing decisions are made,” warned Pew prescription project director Allan Coukell.