FDA’s proposed Expanded Access Navigator points to progress, not panacea
In response to the increasing demand for access to investigational drugs, creation of a clearinghouse to provide information on the request process has been proposed by the Reagan-Udall Foundation for the FDA. Dubbed the Expanded Access (EA) Navigator, the educational tool is the government’s latest effort to increase the efficiency of the complex process of requesting unapproved drugs outside of clinical trials for “compassionate use.”
While the industry welcomes a Navigator, some logistical challenges were raised at a public workshop focused on the Navigator last week.
Consisting of a website and possibly a call center, the Navigator would provide the steps involved in making a single-patient investigational new drug request; a directory of manufacturers’ policies, procedures and points of contact; potential resources for healthcare providers who do not have ready access to an institutional review board through a hospital or research affiliation; and background information about a variety of issues such as drug development, clinical trials and the FDA review process. The overall goal of the Navigator is to “increase transparency and accessibility of information, and thereby increase understanding of EA to help guide patients and clinicians through the request process,” according to the project proposal.
A key question for many stakeholders is whether providing educational content on EA will significantly improve the process and meet patient expectations. The role of the navigator is “not to push companies in one way or give medical advice or be actively involved in decisions about individual access to a drug,” said Barbara Bierer, M.D., faculty co-director at the Multi-Regional Clinical Trials Center of Harvard and Brigham and Women’s Hospital and a panelist at the Navigator workshop. “A lot of people feel that simply giving information about where to find more information is insufficient. What is the role of the Navigator in terms of pushing the agenda forward? That’s where the questions are.”
“To the extent that a navigator can direct those seeking to make contact with the company to the correct website, phone number or person, this is a good thing that increases fairness and decreases frustration,” said Alison Bateman-House, Ph.D., Rudin Postdoctoral Fellow in the Division of Medical Ethics at New York University School of Medicine. “The day should be long over when those who have a brother-in-law working at the company or who have access to their state senator can get their request considered, while those calling the company’s 1-800 phone number end up in phone-tree oblivion.”
EA requests are usually made under extreme time constraints, when a person is left without options for treatment of life-threatening conditions. Under such emotionally-charged circumstances, the Navigator “will fall short of providing requestors what they really want,” said Bateman-House, “which is access to a drug.”
Even if a patient or healthcare provider is armed with information, “going through all the steps needed to get single-patient compassionate use of an unapproved drug can be frustrating and take a lot of time,” stated the American Cancer Society’s patient materials on the issue. “For instance, drug companies have different policies and processes, and there’s no way to force the drug company to supply the drug. There may be very limited amounts of the drug available for compassionate use. Drug companies may only have enough of the drug for use in the clinical trial.”
The Navigator addresses “a small piece in the EA process,” said Jonathan Jarow, M.D. senior medical advisor to the center director of the Center for Drug Evaluation and Research at FDA. Other efforts to make EA requests easier are currently in the pipeline, including a draft of a streamlined application form, expected to be finalized in the very near future, he said.
Jarow added that centralizing the EA process is a job the FDA can’t do alone. For the Navigator concept to move forward and ultimately be successful, “industry will have to be a highly engaged partner,” he said. A pharmaceutical company’s participation in the Navigator would be voluntary, and keeping information current would entail frequent updates on a wide variety of evolving details, such as name changes of investigational drugs, status of clinical trials and contact information within the company.
“My objections to the Navigator are 100% logistical,” said Bateman-House. “We do not want a webpage that has thousands of links to company webpages that will go inactive without frequent maintenance [or] lists of phone numbers that go out of service when small companies are acquired by larger ones,” she said. “We already have one of these, and it is called ClinicalTrials.gov.”
For its part, many in the industry expressed interest in contributing to the workings of the EA Navigator and managing expectations. “While, in general, Pharmaceutical Research and Manufacturers of America (PhRMA) is supportive of the centralized website concept to help patients and healthcare providers to access information ‘as quickly and directly as possible to the needed [expanded access] pages,’ we would like to reiterate that obtaining key stakeholders’ agreement on the final details of any such portal would be critical for ensuring the success and sustainability of the Navigator,” said PhRMA in comments submitted at the workshop. “The posting of information and links to biopharmaceutical companies’ websites in the Navigator should not serve as a guarantee of access to any specific investigational drug by any individual patient.”
“I think any centralized service, especially a call line, where patients and clinicians can be directed to proper company resources would be beneficial,” said Jodie Sherman Gillon, head, external/chief medical office at AstraZeneca. “It’s important to bear in mind, though, that the ultimate solution is to navigate patients through all available options, with the ideal being an approved product or clinical trial. We’re starting to have conversations on how to leverage technology to do that. But for now, I think the Navigator is a quick win.”
This article was reprinted from Volume 20, Issue 20, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »