Call for Improving System of Patient Access to Investigational Drugs

I was part of this meeting and was very happy with the outcome. I hope the FDA and pharma listen to us. Representatives from the FDA, industry (Merck, Pfizer, Tibotec, Panacos and BI) , physician groups, Kaiser Permanente, researchers, and others were part of the discussion.

HIV Research Group Calls for Improving System of Patient Access to Investigational Drugs

With the first agents from 2 new classes of antiretroviral drugs poised to receive regulatory approval this year, the Forum for Collaborative HIV Research, an independent advocacy group, has called for reassessment of the way investigational new drugs are made available to patients in need.

Below is the text of the group’s June 14 press release:

HIV Research Group Calls for Improving System of Patient Access to Cutting-Edge Investigational HIV Drugs

With New Treatments in the Pipeline, Scientists, Industry, Patient Groups Say Change is Needed if All are to Benefit

WASHINGTON, DC (June 14, 2007) — With at least three promising new HIV drugs in development, radical reform is needed of “expanded access programs” that allow patients to take new drugs before final federal approval.

Such programs can be critically important to HIV patients who no longer respond to current treatments. However, the system for providing access to experimental drugs is fragmented and under-funded, and discourages academic health centers and private physicians from participating, according to a report released today by the Forum for Collaborative HIV Research, an independent public-private partnership comprised of academic researchers, patient advocates, government officials and industry representatives.

The report reviews these problems and recommends guidelines for fixing the system, including a recommendation that drug companies consider reimbursing health care providers for Expanded Access Program (EAP) costs.

“The goal of expanded access programs is to make promising drugs in the late stages of clinical trials available to patients who urgently need treatment and have exhausted all currently approved therapies,” said Ben Cheng, Deputy Director of the Forum. “Unfortunately, the current mechanism for early access to these promising drugs serves neither patients, companies, nor regulators.”

The United States lacks data regarding the extent to which patients have run out of treatment options, but one estimate in the report suggests that as many as 13 percent of HIV patients have run out of treatment options. EAPs have provided critical medical options for such patients since the early years of HIV treatment, when HIV activists first pressed the Food and Drug Administration [FDA] for such options. But today EAPs are riddled with problems, according to the report, “Rethinking the Approach to Expanded Access Programs.”

Among the main problems, EAPs are:

• Expensive to administer, limiting the ability of many healthcare sites to participate, as they must cover such costs themselves. The costs of administration are currently not reimbursed by drug companies, though they are when drugs are provided to patients in clinical trials.• Poorly advertised;• Usually dependent on only one novel drug in treating patients. Though policies may recommend using at least two active therapies, many patients are resistant to the other drugs that are available. This can lead to virtual monotherapy, and to the risk of developing resistance to the new treatment.• Relatively inaccessible to minorities, women and rural residents;

• Failing to take advantage on a valuable opportunity to gather useful safety data on emerging drugs.

The associated expenses and administrative obstacles can be particularly onerous at academic medical centers, where financial and bureaucratic hurdles are greater than in private practices or community-based clinics.

As a result, many academic centers have opted out of participation in EAPs. But treatment opportunities offered by the emergence of simultaneous EAPs for three new drugs (raltegravir [Isentress, formerly MK-0518], etravirine [TMC125], and maraviroc [Celsentri]) are leading some of these institutions to reconsider their decisions so they can offer the new drugs to patients who need them.

But, according to the report, administrators warn that the programs will remain in jeopardy until reliable sources of funding are found to cover high expenses related to data collection requirements and approval processes from internal review boards.

The report is being released at a pivotal moment in the history of HIV drug development, according to its authors. There are a number of drugs in the development pipeline that offer novel approaches to treating HIV. Among these are: a new protease inhibitor, new entry inhibitor drugs that block the CCR5 receptor on immune cells, and new integrase inhibitors.
“Patients groups are particularly excited that so many new options are becoming available,” said Veronica Miller, Director of the Forum. “But it is clear that changes must be implemented to make sure that we can expand access to these new therapies as they are proven basically safe and effective.”

The report’s recommendations are based on the outcome of a recent Forum workshop, which brought together representatives of academic health centers, advocacy groups, pharmaceutical companies, and the Food and Drug Administration. It is particularly timely, given that the United States Food and Drug Administration is in the midst of revising its rules for the regulation of EAPs for other diseases.

Report Recommendations

The HIV Forum report identifies several key directions for improving the EAP system, suggesting that:

• Policymakers explore a two-tiered approach to EAPs. One would be an actual research protocol designed to answer specific questions leading to drug approval. The other would be a simplified system, with fewer data requirements. Both would be would eligible for reimbursement from drug companies, as are traditional clinical trials;

• Pharmaceutical companies work together and with regulatory agencies to design EAPs that simultaneously use several different new drugs in order to limit the use of “virtual monotherapy” and lessen the chance of developing drug-resistant strains of HIV;

• Changes be implemented to decrease the burden of paperwork, among them, standardization of EAP data collection requirements and safety reporting;• Pharmaceutical companies collaborate to standardize their case report forms and adverse events reporting for their EAPs;

• Develop a centralized electronic database, and other technological tools, to provide real-time access to analyse results, including adverse events for the EAP.

“Every time there is an adverse event, physicians have to report it,” Cheng said. “But there is no way to find out whether this is happening in five or 50 percent of patients. None of the information is being analyzed until the program is finished.”

Since EAPs were first initiated for HIV drugs in the 1980s, they have saved or prolonged the lives of thousands of HIV patients. Since 1998, however, the use of EAPs for HIV drugs has decreased, and that the size of the patient population that currently needs access to investigational antiretroviral drugs is difficult to estimate. Refining this estimate will be an important aspect of reforming EAPs, according to the study.

The Forum for Collaborative HIV Research is an independent public-private partnership whose mission is to facilitate discussion on emerging issues in HIV clinical research and the transfer of research results into care. It is comprised of international experts from government agencies, pharmaceutical companies, academia, advocacy and community organizations, and private foundations. The Forum is housed in the Department of Prevention and Community Health at The George Washington University School of Public Health and Health Services. For further information, visit: and click on “Rethinking the Approach to Expanded Access Programs


SourceForum for Collaborative HIV Research. HIV Research Group Calls for Improving System of Patient Access to Cutting-Edge Investigational HIV Drugs. Press release. June 14, 2007.

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