General Publications October 23, 2015

“Are Patient Advisers Contraindicated for Pharma Cos.?,” Law360, October 23, 2015.

Extracted from Law360

Today, patients and patient organizations are front and center in the drug development process, not only as stakeholders, but as trusted advisers of drug developers. Yet, communications between developers and these groups can be fraught with risk, including jeopardizing the informed consent process, promoting investigational products prior to approval and promoting off-label uses of already approved products. In addition, public companies have the added worry of inadvertently disclosing nonpublic information about their product development plans or progress. There are a number of best practices clinical-stage drug developers can implement to help manage their legal and regulatory risk exposure while still forging collaborative partnerships with patients and patient organizations to help advance their drug development programs.

As evidence of the growing role patients are taking in drug development, this summer, the U.S. Food and Drug Administration issued its first-ever draft guidance for industry whose initial draft was prepared by a patient advocacy group. The Parent Project Muscular Dystrophy (PPMD) patient advocacy group spearheaded the effort to prepare an initial draft of FDA recommendations regarding clinical development considerations and trial designs that drug sponsors might utilize when developing drugs for Duchenne muscular dystrophy and related dystrophinopathies. This level of patient engagement not only in prioritizing FDA’s resource-limited guidance agenda but also in preparing a first draft of what would ultimately become agency-issued guidance is new ground for patient engagement in drug development.

Drug sponsors have long been partners with the patient community as their commercial success hinges upon developing and delivering products to the market that will meet patients’ medical needs. But today, as patient groups find an increasingly stronger voice within the FDA to shape the agency’s thinking on drug development, sponsors must assign a greater priority to preapproval interactions and alignment with the patient community throughout clinical development. Given the sea of legal and regulatory risks sponsors must navigate, however, drug developers must carefully manage these preapproval interactions so as not to contravene agency regulations or draw enforcement scrutiny or lawsuits.

First, while talking with patients to understand a disease’s natural history, progression, outcomes and impacts on patient lives is critical for sponsors developing therapies for those patients to understand, sponsors can quickly run afoul of the FDA’s informed consent and recruitment authorities if those discussions involve the sponsors’ investigational drugs. The FDA’s regulations require institutional review board (IRB) approval of, among other things, information given to patients about investigational products as such information can be seen both by the FDA and IRBs as the start of the informed consent process.

The core focus of the informed consent process is to ensure against undue coercion for clinical trial participation, so discussions about new therapies with patient audiences can run the risk of unknowingly commencing the informed consent process. Where those communications are not properly balanced and, for example, imply a favorable benefit or outcome of a pipeline product beyond what is included in the informed consent or study protocol, a sponsor can jeopardize the ability of those patients to provide a proper consent to future study enrollment.

Some companies choose to navigate this regulatory risk by engaging with patient organization or advocacy group leadership personnel who are not themselves patients. However, even when communications with those leadership personnel are conducted under a confidentiality agreement, information, particularly positive information, can travel especially in today’s age of social media and especially in disease spaces where patients are desperate for new treatment options. Drug developers would be wise to limit information presented about pipeline products in confidential meetings to information already in the public domain about their products that company personnel have carefully vetted.

Second, and perhaps why sticking to carefully vetted information is even more important, is ensuring that communications to patient groups about pipeline products do not violate the FDA’s prohibition on preapproval promotion. The FDA’s authorities restrict drug developers from representing that a pipeline product is safe or effective for uses for which it remains under investigation. Moreover, where the FDA has already approved one use of a product, promotional communications about new uses undergoing investigation can raise a risk of off-label promotion that not only “misbrands” the product under the FDA’s authorities, but also produces potential False Claims Act liabilities, which some pharmaceutical companies have paid over a billion dollars to settle.

Over the last several years, the FDA has issued a handful of enforcement letters against drug sponsors and investigators for communications the FDA found to constitute violative preapproval promotion, including websites targeted to patients where the net impression suggested the product was already approved. Generally speaking, the FDA’s enforcement letters solicit a plan from the sponsor or investigator as to how it will cease use of violative preapproval promotional communications. Because these letters are posted publicly, they can create bad publicity and, where an approval already exists for another use of the product, the FDA’s enforcement letters may be of interest to government investigators looking into FCA complaints. Even under a confidentiality agreement, communications about unapproved or off-label uses can be risky, particularly when there is not a legitimate business reason for those discussions and when sales or marketing personnel lead those communications.

Lastly, public companies communicating about their pipeline products to patients or patient groups carry an added level of scrutiny by shareholders and prospective investors under the securities and exchange rules. Under these rules, public companies must disclose material developments about their pipeline products to their current and prospective shareholders but, under the FDA’s authorities, must do so in a manner that does not jeopardize the informed consent process or promote their products prior to the FDA’s approval. As a result, public companies must vet their material public disclosures and communications even more carefully. In addition, confidential discussions can be risky for public companies to engage in and can lead to inadvertent disclosure of nonpublic information.

There are several best practices drug developers can employ when engaging in preapproval communications with patients or patient groups to help minimize their legal and regulatory risk exposure. First and foremost, ensure the accuracy of preapproval communications, make clear that the product remains investigational and be transparent about the steps remaining prior to approval. In addition, always ensure that preapproval study data are presented with appropriate context (e.g., interim findings vs. final study data) and balance (i.e., both efficacy and safety data). So often, drug developers want to share the latest efficacy findings with patient groups but without an equal focus on the latest safety findings. Maintaining that balance is important not only should the FDA or an IRB find that the companies’ communications commenced the informed consent process as a recruitment material, but also should the FDA review whether the companies engaged in impermissible pre-approval promotion. And finally, companies must take care to avoid use of promotional terminology and conclusive statements about product safety or efficacy when communicating about their pipeline products as the FDA will be the final arbiter of which drug products are deemed safe and effective. Adhering to these best practices in preapproval communications, whether confidential or not, together with effective employee training, will help keep drug developers’ legal and regulatory risks in check. 

Media Contact
Alex Wolfe
Communications Director

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