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Bolstering Patient Access: FDA’s Project Orbis Attains First Joint International Drug Approval

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New call center also supports investigational requests.

The Food and Drug Administration (FDA) achieved 2 significant milestones recently in its ongoing efforts to expand access to new and investigational drugs for cancer patients.

Project Orbis, created to support streamlined international approval of new cancer therapies, attained its first collaborative approval across multiple international regulatory agencies with a drug combination targeting a form of endometrial cancer. Earlier, the FDA also launched a new call center pilot program, Project Facilitate, to help healthcare professionals request access to unapproved therapies for patients with cancer.

Both programs are initiatives of the FDA’s Oncology Center of Excellence, a part of the Office of Medical Products and Tobacco in the Office of the Commissioner. The Oncology Center of Excellence seeks to expedite the development of oncology and hematology medical products and support an integrated approach to clinical evaluations involving cancer treatments.

Green Light for Combined Cancer Treatment

In September, a collaborative review conducted through Project Orbis by the FDA, the Australian Therapeutic Goods Administration, and Health Canada resulted in simultaneous approvals by the 3 agencies of Lenvima (lenvatinib) used with Keytruda (pembrolizumab) to treat patients with advanced endometrial carcinoma.

The combination is indicated for patients whose endometrial carcinoma is not microsatellite instability high (MSI-H) or mismatch repair deficient (dMMR), and who have disease progression following prior systemic therapy but are not candidates for curative surgery or radiation, according to the FDA.

The FDA initially approved Lenvima for a specific form of thyroid cancer in 2015 and Keytruda for advanced melanoma in 2014. Approval of the drugs’ combined efficacy was based on a clinical trial of 94 patients with endometrial carcinoma tumors who were not MSI-H or dMMR.

Of the trial population, 10 patients had a complete response or disappearance of all lesions on imaging, and 26 had a partial response or shrinkage of lesions by at least 30%, resulting in an objective response rate of 38.3%, FDA reported.

Endometrial cancer is the most common cancer of the female genital tract. Obesity, metabolic syndrome, and certain estrogen-promoting medications may increase the risk for the disease. Symptoms can include unusual vaginal bleeding or pain in the pelvis.

Overcoming Systemic Delays

Project Orbis’ joint international submission and review process is designed in part to help identify any regulatory divergences across national review teams, according to the FDA. The approach is intended to provide cancer patients with earlier access to products in countries in which significant delays in regulatory submissions may occur, irrespective of whether the product has received FDA approval.

Drug approval delays in other nations can sometimes result from differing national standards of care, which in turn can affect cancer clinical trials and potentially slow the development of anticancer products.

“We are pleased to be working alongside our Australian and Canadian colleagues to help make potentially life-changing treatments available to patients as quickly as possible while still ensuring FDA’s high standards of safety and effectiveness,” said Acting FDA Commissioner Ned Sharpless, MD.

“As Project Orbis expands, we look forward to welcoming additional international partners to collaborate with us in this important initiative as we work to help further serve the global patient community.”

Fifteen Years in the Making

The Lenvima-Keytruda approval marks the culmination of a 15-year effort by the FDA and its counterpart agencies aimed at establishing a global framework for aligning oncology drug approvals. In 2004, the FDA’s Office of Hematology and Oncology Products (OHOP) began holding regular teleconferences with other regulatory agencies worldwide to exchange information and collaborate on specific topics related to applications under review.

Today, OHOP holds a monthly teleconference with Australia’s Therapeutic Goods Administration, Health Canada, the European Medicines Agency, Japan’s Pharmaceuticals and Medical Devices Agency, and Switzerland’s Swissmedic. The FDA and China’s National Medical Products Administration also have initiated quarterly meetings to discuss non-product-specific regulatory issues facing worldwide drug development.

Although Project Orbis currently is focused solely on supplemental oncology approvals (new indications for previously approved therapies), the FDA said discussions with other agencies are planned to consider the possibility of cooperating on new drug applications (NDA) and biologics license applications (BLA). However, the agency noted that the process of collaboration on NDA or BLA reviews may be more complex due to the proprietary information involved.

New Physician Call Center

In another recent development, the FDA in June launched a dedicated call center designed to help clinicians request access to unapproved therapies for cancer patients. Known as Project Facilitate, the call center provides a single point of contact for physicians to receive assistance from FDA staff in the submission of an Expanded Access request for individual patients.

The FDA’s Expanded Access program offers a means for patients with serious or life-threatening morbidities, such as cancer, to try investigational medical products for treatment when no satisfactory therapies are available and enrollment in a clinical trial is not feasible. Investigational drugs have not been approved by the FDA, may or may not be effective in the treatment of the condition, and can result in serious, unanticipated side effects.

“The FDA has been working diligently to improve the Expanded Access framework, including development of an updated and more streamlined application form,” said FDA Acting Commissioner Sharpless.

“But despite recent improvement, we understand that for many patients or healthcare professionals, especially those not familiar with the Expanded Access program, the process may appear confusing or burdensome. Today’s launch of Project Facilitate is part of our continued commitment to Expanded Access, and we hope that this program will simplify the process for oncologists and ultimately benefit patients.”

Project Facilitate compliments the Expanded Access Navigator, a web-based resource developed by the Reagan-Udall Foundation to educate patients and providers about the Expanded Access program. The navigator includes information provided by companies about their Expanded Access policies.

Clinicians can contact the Project Facilitate call center at 240-402-0004 between 8 AM and 4:30 PM ET, Monday through Friday. They can also email the center via

Patients and families with questions can call FDA’s Division of Drug Information at 301-796-3400 or email

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