Pharma needs to be more upfront about FDA drug rejections

June 15, 2015

FDA researchers are calling for greater transparency from drug companies when their drug is rejected by the agency, according to a study in the British Medical Journal.

FDA researchers are calling for greater transparency from drug companies when their drug is rejected by the agency, according to a study in the British Medical Journal.

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Complete response letters (CRLs) issued by FDA’s Center for Drug Evaluation and Research (CDER) indicate reasons for withholding approval of a drug and explain which additional data might support an approval decision. CRLs are issued for a variety of substantive reasons, however safety and/or efficacy concerns were identified in a majority of the CRLs reviewed as part of this study.

Dr LuriePeter Lurie, MD, FDA associate commissioner for public health strategy and analysis, and colleagues compared the content of complete response letters (CRLs) (n=61) issued by FDA’s CDER between August 11, 2008, and June 27, 2013, with the text of sponsor-issued press releases on FDA’s action. Sponsor-issued press releases describing CRL issuance were identified through publicly available sources, including sponsors’ websites and Internet search engines. Drug names also were entered into the Securities and Exchange Commission’s Electronic Data Gathering, Analysis and Retrieval System.

“The analysis of the content of press releases indicates that they are incomplete substitutes for detailed information CRLs contain,” Dr Lurie said. “While statements that were included in the press releases were typically accurate, they generally were an incomplete source of reasons for FDA non-approval of applications.”

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For example, he said that only a minority of the press releases clearly stated that receipt of a CRL meant that marketing could not commence and most findings associating the drug with a higher mortality rate went unmentioned in press releases.

“Disclosure of CRLs would allow FDA to increase the overall transparency of its regulatory process, provide greater awareness of the agency’s role in protecting public health and combat misperceptions regarding the basis for drug non-approval,” Dr Lurie said. “It also would allow for broader and more informed public discussion by relevant stakeholders. The potential benefits of publicly disclosing the agency’s detailed rationale for refusing approval include better informing the development of new drugs, facilitating a richer public health discourse and counteracting misconceptions regarding FDA’s reasons for denial of applications. However, the need for increased transparency must take into consideration FDA’s legal requirement to protect sponsors’ trade secret and confidential business information.”

According to Dr Lurie, the CRLs reviewed as part of the study, 48% of them cited deficiencies in both safety and efficacy and only 13% cited neither safety nor efficacy deficiencies. Others called for new clinical trials and higher mortality rates in treated subjects.

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