Parexel has launched an expanded Managed Access Programs service to streamline patients’ access to compassionate use of investigational medical products.
The FDA allows companies to provide drugs and medical devices still under clinical investigation to seriously ill patients who cannot participate in the trials but might benefit from those treatments. The patient can either enroll in an existing expanded access program sponsored by the drugmaker or apply for single-patient access. The EMA also has a counterpart program. Parexel’s new system helps biopharma companies design these programs and deliver them to patients in need.
Because the drugs have not been approved, the program, called Compassionate Use or Expanded Access, imposes stringent policies on their applications in human. The person’s disease needs to be life-threatening, and currently available treatments haven’t been helpful. The patient must not be eligible for any ongoing clinical trials if there is any. The physician must agree that the experimental drug is the only option, and that the drug has the potential to provide more improvement than damage; what’s more important, the physician must be willing to manage the use of an unknown drug. Plus, the company that makes the drug is not obligated to offer it, and not every drugmaker is willing to provide it to a patient outside a clinical trial.
Even though the FDA says it authorizes over 99% of expanded access requests it receives, going through all those steps can be frustrating. It involves medical expertise and experience in managing these programs, including familiarity with the regulations and key agencies, said Peggy Schrammel, a Parexel VP who oversees the new platform.
“Parexel’s unique configuration of simplified, end-to-end services for Managed Access Programs includes seasoned operational teams, global regulatory expertise, proprietary technology, and global clinical logistics,” Schrammel said in a statement.
Parexel’s program, managed as part of the CRO’s MyAccessPrograms platform, does not stop after it helps a patient through that application process. Clinical logistics experts will continue to manage the supply of the medicinal product on a worldwide basis, safety experts will monitor pharmacovigilance and report safety events, and market access experts help clients determine the utility of collected data for potential real-world value.
The new service seems timely given the backdrop of the recently enacted 21st Century Cures. The act, signed into law by President Obama last December, allows more opportunities for inclusion of real-world data—compared to those gathered in randomized controlled clinical trials—into the drug-approval process, a provision that could encourage more companies to initiate expanded-access programs for possible real-world data.