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Volume 38, Issue 3
The public and policymakers seek more transparency in pricing and product development.
There’s a mounting clamor for an end to the secrecy surrounding prescription drug pricing, promotional activities, regulatory decisions, and research findings. Stakeholders on all sides believe that more information on drug discounts and rebates is key to reining in payer and patient outlays for medicines (see sidebar below). States are expanding provisions in the Federal Open Payments or “Sunshine” program that tracks industry
payments and gifts likely to influence prescribers. And more timely and complete information on product safety aims to prevent patient harm, as do efforts to make drug labeling more informative.
The FDA recently adopted a policy to inform the public more quickly about adverse events and product recalls and the agency will watch to see if that generates unwarranted alarms or gets high-risk products off the market more effectively.
The full disclosure movement has gone global, moreover. The European Medicines Agency (EMA) seeks to provide more detailed clinical trial data, despite industry protests. And the China FDA recently proposed a plan to issue and disseminate more information regarding the regulatory review and approval of new drug applications.
A main transparency issue involves access to information on the status of drug applications and FDA’s decision-making process in both denying and approving submissions. Consumer activists and free market deregulators alike want to see the complete response letters (CRLs) FDA sends manufacturers that essentially delay or reject an application and outline what additional clinical or manufacturing information is needed to achieve approval. FDA currently posts summaries and some data when it approves a new drug or biologic for market. But current rules prevent agency disclosure of information on products that fail to pass muster, ostensibly to protect trade secrets or confidential information, and manufacturers want to keep it that way.
FDA Commissioner Scott Gottlieb addressed these issues at a January forum to discuss a “Blueprint for Transparency at FDA” issued in March 2017 by a group of experts organized by the Johns Hopkins Bloomberg
School of Public Health (view here). Gottlieb announced a new pilot to test the impact of FDA posting more detailed data from clinical study reports (CSRs) of approved drugs, asking that sponsors of nine new products voluntarily provide CSR data, protocols, and statistical analysis plans on their pivotal studies. FDA also aims to better track drug studies through the R&D process to review and approval by adding the ClinicalTrails.gov identifier (NCT) number to all clinical data submitted to the agency.
However, Gottlieb hedged about publishing CRLs, proposing instead to further explore FDA’s authority to disclose these documents, while evaluating the feasibility of redacting and releasing a subset of CRLs that raise important public health issues. While he acknowledged that some information in CRLs might enhance the appropriate use of marketed products, Gottlieb noted that redacting proprietary information from these letters is burdensome and that much of the data may not be useful. Meanwhile, FDA officials emphasize that pharma companies are perfectly free to publish CRLs and other confidential information on their own and should do so, especially information important to patients and prescribers.
The Hopkins Blueprint further recommends greater disclosure of FDA’s evaluation process for new drugs, generics, and biosimilars, including what products are in the review queue and why certain applications are not approved. The rationale is to help avoid research studies not likely to succeed, reducing costs and waste and unnecessary patient exposure to potential harm. The experts also want FDA to have authority to correct misleading information issued by manufacturers, such as incomplete factors underlying a CRL, and for the agency to disclose results from clinical trials for approved products when sponsors fail to do so.
These issues are not new and have been discussed and debated by FDA and stakeholders for years. The agency launched a Transparency Initiative in June 2009 under former commissioner Margaret Hamburg,
which has led to FDA online “dashboards” that track agency actions and programs, including inspections, recalls, imports, and compliance actions. A “Drug Trials Snapshots” initiative posts data from clinical trials on products approved in recent years. FDA has expanded access to agency enforcement reports and adverse event data and has made its guidance development process more visible and efficient.
Sponsors, furthermore, are listing more studies on the ClinicalTrials.gov website, although the record is weaker for timely disclosure of research results for newly approved medical products. Under pressure to share more research data to avoid repeated errors and waste and support more efficient clinical research, biopharma companies are providing qualified researchers with access to confidential clinical research data. And some sponsors are pledging to publish new research reports only in open-access journals.
While policymakers and industry struggle to refine transparency, continued expansion of information available through the Internet, social media, and smart phones-some of it inaccurate or biased-erodes traditional controls over public disclosure of data on drug testing and production. Reports of adverse events and enforcement actions emerge online quickly, as do claims of both patient harm and of miracle cures. These developments raise questions about the value of limits on what FDA can or cannot disclose regarding products or manufacturers and highlight the importance of more forthright industry disclosures about marketing and results.
Jill Wechsler is Pharmaceutical Executive’s Washington Correspondent. She can be reached at email@example.com