It’s never too early to start preparing a global regulatory submissions strategy.
When you’re planning a new type of trial – direct-to-patient (DtP), eclinical or virtual study – it’s especially important to communicate with regulatory agencies, and even ask for scientific advice, earlier in the drug development process.
In DtP trials drug therapies and medical equipment are delivered and administered in a patient’s home. Patients may take the medications themselves or rely on a home care provider for clinical support, to measure vital signs, prepare laboratory samples, and ensure safety.
From a regulatory point of view, DtP trials are a challenge because they are so new that no country has yet issued formal regulations.
Most countries lack regulations for handling investigational medicinal products (IMP) at a patient’s home. In addition, regulatory agencies are increasingly requesting temperature monitoring (E.U. GMP, Annex 15, GDP Guidance, etc.), which requires the use of temperature tracking devices and systems in the supply chain.
Since every country differs in its approach, the protocol you write must include a chapter that explains the ethical and medical justification for conducting the trial partially or completely remotely. The DtP protocol must be amended with an explanation of the process, ethical considerations, and justifications as to why the trial will support a patient’s safety and treatment.
Every country also has different data privacy rules. Patient data are classified as sensitive personal data, and the country in which the patient is located determines the rules. There are specific rules about how and where patient data are stored and can be shared.
In 2016, the United States and European Union agreed on a new framework for transatlantic data flow. That framework was designed to protect European patients’ fundamental right to transfer their data to the United States legally.
Because the supply chain for DtP studies can include several vendors handling sensitive personal information, all vendors—including investigators and CRAs—need to be trained in and understand patient privacy regulations. Interactive response technology systems (IRT) are required to keep patient details separate from study-supporting data so that only non-clinical service providers (e.g., couriers) have access to patient details. This helps avoid the risk of disclosing a patient’s identity to the study team inadvertently.
Finally, sponsors need to be aware of increased requirements for secure information (e.g., encryption) in each country. Should data be lost, security breach notifications must be made to local data protection authorities.
US data shows that of the 132 new product applications submitted to the FDA’s Center for Drug Evaluation and Research between 2008 and 2012, the 49 that followed a pre-IND meeting 49 had a median clinical development time (CDT) of 6.4 years. In contrast, the marketing applications that skipped that pre-IND meeting had a mean clinical development time of 8.3 years.
If you are about to start the clinical development process and are considering a DtP trial, engage your local regulatory agency, help them understand why a remote trial makes the most sense, then amend your protocol/filing documents based on the feedback you receive.
For more information on Direct to Patient clinical trials, download our white paper.
 “EU Commission and United States Agree on New Framework for Transatlantic Data Flows: EU-US Privacy Shield,” European Commission Press Release, February 2016, http://europa.eu/rapid/press-release_IP-16-216_en.htm.
 Vu and Pariser, 2014