Transparency. Toto, We're Not in Kansas Anymore

Let's be honest. Transparency is usually a real sleeper. FDA talks about being transparent, issues some information that no one really wants and everything continues pretty much as it always has.

Not anymore. FDA may have just blown the lid off business-as-usual transparency. And FDA may be using this transparency initiative to garner additional regulatory authority.

In January 2009, President Obama issued a call for an "unprecedented level of openness in government." In June 2009, FDA Commissioner Hamburg launched a transparency initiative that is overseen by a task force comprised of very senior agency management – Principal Deputy Commissioner Sharfstein, five Center Directors, the Chief Counsel, Associate Commissioner of Regulatory Affairs and FDA's Chief Scientist. When these people speak, Commissioner Hamburg and the rest of FDA will very likely listen.

For nearly a year, the task force has held public meetings, opened an online blog and solicited comments on how FDA should improve transparency.

On May 19, 2010, the task force issued 21 proposals for how FDA should change the way it discloses information to the public about the products it regulates. The proposals cover adverse event reports, dockets management, enforcement priorities, import procedures, inspections, product applications, recalls and warning letters.

The logo on the task force report says, "Open Government. Transparent. Collaborative. Participatory." These words are not often used to describe today's FDA. And it remains to be seen how receptive the agency will really be to public participation in policy creation. Setting these concerns aside (if only momentarily), these proposals are remarkable for their breadth and boldness in re-evaluating long held beliefs about the need for confidentiality of a great deal of information about the products FDA regulates. If implemented, these proposals would be the most significant changes to FDA's policies for releasing information since enactment of the Freedom of Information Act in 1966. The proposals pertaining to the release of information about product applications are particularly interesting and described in more detail below.

But before turning to the product application proposals, we urge you to read this document and make your comments to the agency. The report is available on the home page of the FDA website, www.fda.gov. Comments will be accepted until July 19, 2010. It won't put you to sleep. And much as we love Kansas, we're not there anymore.

Product Application Proposals

Current FDA regulations and policies prevent the agency from making public much of the information it has about the products it regulates. For example, FDA cannot acknowledge even the existence of an investigational application, and it cannot discuss its reasons for refusing to approve or clear any product.

It has been a given for many years that disclosing this kind of information could give competitors an advantage by providing information about the development process of particular products and harm incentives for innovation. According to the task force report on May 19, 2010, industry commented that the current limitations on FDA's release of information strike the correct balance between disclosure of information and maintaining the confidentiality needed to foster innovation.

Other commenters disagreed. It was noted that the European Medicines Agency releases more information than FDA. Some commenters noted that releasing additional information may reduce duplicative studies, provide important safety information and actually encourage rather than discourage medical breakthroughs by allowing scientists to avoid repeating past failures.

Following this discussion, the task force made the following proposals:

1. FDA should disclose the existence of investigational applications, including the name of the application sponsor, the date the application was received, the proposed indications(s) or intended use(s) of the product and the proposed proper and/or trade name of the product, if available.
2. FDA should disclose whether an IND or IDE has been placed on hold, terminated or withdrawn, and whether such a hold is lifted.
3. FDA should disclose the fact that an NDA, ANDA, BLA, PMA or 510(k) application submitted (or resubmitted) to the agency at the time the application is received by FDA, including the name of the application sponsor, the date the application was received, the proposed indications or intended use of the product and the proposed proper and/or trade name of the product.
4. FDA should disclose that an unapproved NDA, ANDA, BLA or PMA, or uncleared 510(k) has been withdrawn or abandoned by the sponsor. If the product is associated with a significant safety concern, FDA should provide a brief description of the product, the use for which approval was sought or obtained and the identified safety concern.
5. FDA should disclose refuse-to-file or complete response letters in response to an NDA, BLA or supplemental NDA or BLA at the time the letters are issued.
6. FDA should disclose "not approvable" letters in response to a PMA or supplemental PMA and "additional information (AI)" letters in response to a 510(k) submission at the time the letters are issued.
7. FDA should disclose relevant summary safety and effectiveness information from an investigational application or from a pending marketing application, if the agency concludes that disclosure is in the interest of the public health, which includes when FDA believes it is necessary to correct misleading information about the product that is the subject of the application.
8. FDA should convene a group of internal and external stakeholders to discuss the possible uses of non-summary safety and effectiveness data from product applications.

And It's More Than Product Approval Applications

The 21 specific proposals in the report cover much more than just product approval application information. Other topics include:

Recalls - FDA wants to make public substantially more information about product recalls and the reasons for them. In doing so, FDA has indicated that it will seek expanded authority to require submission of detailed recall information to FDA which FDA will then make public. This is an example of FDA potentially using transparency considerations as a basis for expanding regulatory authority.

Recall termination - FDA proposes that it make public when it is determined that a recall has been completed. This is similar to the warning letter termination process and will pressure companies to promptly complete recalls – or to be prepared to explain to the public why the recall is still open.

Inspections - FDA also proposes publishing information on all inspections, including inspection results (Official Action Indicated, Voluntary Action Indicated or No Action Indicated). FDA would also publish summaries (apparently not by company) of common categories of findings.

Adverse events - FDA proposes expanding the amount of information made public about adverse events and, perhaps more importantly, to give any user the ability to generate their own specific summaries of adverse events. This will permit any person (including plaintiff lawyers and industry critics) to do their product trending and to make that public.

Enforcement - FDA proposes providing more information about enforcement actions and actions referred to the Department of Justice for prosecution.

Conclusion

FDA's Transparency Initiative is not business as usual. All stakeholders should review these proposals carefully to determine whether confidential information is at risk, whether the information disclosures being proposed benefit public health and public policy, and the impact on their activities.