Early 2018, the US Food and Drug Administration (USFDA) announced steps toward enhancing the transparency of clinical trial information. This was aimed at providing more support to further advance innovations and scientific research. The USFDA took this decision on exploring new ways to share information about product approvals which can improve patient care and better inform people about the products they prescribe or intake. To increase transparency, the USFDA is focusing on the information usually provided in the clinical study reports (CSRs).
Clinical Data Report Pilot Program
The USFDA had launched a clinical data summary pilot on March 19, 2018, where the primary agenda was to post key portions of the CSRs — the documents which sponsors create for the USFDA after each clinical study. These portions contain complete summary of the entire result, the protocols and amendments, as well as the statistical plan. The reports are redacted by the FDA to exclude confidential commercial information, trade secrets and personal privacy information. Before the data transparency move, the FDA provided drug approval action package which shared a significant amount of information from the sponsor’s application but did not have provisions to provide a complete summary of results. The incomplete information made it difficult for academic researchers, regulatory agencies and other stakeholders to gain an in-depth understanding of the studies supporting the approval.
The pilot program started by posting summary reports of nine approved new drug applications of participating sponsors, some of which are Janssen, Johnson and Johnson, and Sanofi. Janssen Biotech volunteered as the first pilot participant for the approval of Erleada (Apalutamide, a non-steroidal anti-androgen) which will be used to treat non-metastatic, castration-resistant prostate cancer. The USFDA uploaded CSR information, FDA review along with label and other supporting documents to facilitate a deeper understanding of their approval decision. In addition to the pilot, the agency is also adding a unique identity to the materials i.e., the ClinicalTrials.gov identifier number, also called the National Clinical Trial (NCT) number. This number will make it easier to correlate the clinical listings on the website to the FDA announcements on specific drugs which can include product labeling and advisory committee meeting materials. Firms are expected to ensure that the required clinical trial data is published on clinicaltrials.gov, as delays will attract a fine of $10,000.
Impact on the Pharmaceutical Industry
This move impacts the pharmaceutical industry significantly. Now the pharma companies are expected to submit enriched data sets and hence there will be issues regarding data management. Each company has its own trade secrets and procedures, and therefore, they will have to scrutinize the information which will be available to the public post submission. Due to such development on data transparency, there might be a slowdown in the drug development process. Pharmaceutical companies that utilize “alteration” of existing drug as a life cycle management strategy, for e.g., using an anti-allergen and tweaking it to create an anti-asthma drug, will be impacted. By releasing the required level of information to academics and researchers, the business strategy of “evergreening” of a drug” will be under pressure. Also, the development pipeline of a company can be hampered if competitors start using the information for inventing a new process of the same drug.
Such transparency requirements help in better understanding the trial and the result. The data published under the “results” section was previously lacking the details required to satisfy stakeholders and sponsors. Now, with the release of result synopses and application of simple language in the “summary” of trial data, they can comprehend the development process and outcomes of trials much better. Patients will now receive a more detailed perspective of their clinical trial processes and hence can make better informed decisions. The populace that once had to depend on inaccuracies published via secondary resources can now access scientific and trustworthy information.