Skip to main content

07-07-2011 | Article

Action needed to address approval of ‘poor value and unacceptably risky’ drugs


Free abstract

MedWire News: Researchers have proposed five practical measures that they believe will promote innovation in drug development and increase the value of therapies that reach market.

"New drugs have the potential for transformative innovation, and improvements in outcomes and adverse effect profiles, as well as simpler regimens that improve convenience and adherence, all reflect important increases in the clinical utility of new products," write G Caleb Alexander (University of Chicago, Illinois, USA) and colleagues in the Annals of Internal Medicine.

"These innovations and the cost-effectiveness of a drug would ideally guide its adoption and use," the authors continue. "However, information about the relative efficacy and safety of a drug is often learned only after market entry and, to complicate matters, other factors such as advertising independently contribute to prescription use," the researchers say.

Alexander and team therefore propose five practical measures that they believe would ensure that only the most valuable novel treatments reach market.

Firstly, they suggest increasing the quality of the evidence required for approval. Specifically, they suggest that the US Food and Drug Administration (FDA) should, without exception, require sponsors to submit the results of trials comparing novel treatments with the current standard of care, with comparator and margins of noninferiority dictated by external experts. The authors' second suggestion is that comparative efficacy and safety information be routinely and clearly included on prescription drug labels.

Thirdly, the researchers recommend a period of conditional approval during which further data collection is required and professional and patient marketing is not allowed.

The authors concede that these measures would increase development costs for pharmaceutical companies and, therefore, as a fourth measure, suggest incentives beyond the existing patent extension and orphan drug tax breaks to include patent exclusivity for drugs for common diseases that are genuinely innovative and have significant public health benefits.

Finally, the researchers suggest changes to drug reimbursement procedures to avoid "wasteful spending." The authors cite the US$2.8 (€1.9) billion spent on esomeprazole in the US in 2009 despite evidence indicating no benefit over generic proton pump inhibitors, and the fact that, up to a year after the FDA issued a safety advisory about atypical antipsychotics in elderly patients with dementia, no state Medicaid formularies had modified their prescription policies to recommend safer and cheaper alternatives.

Summarizing, the authors caution that "policy changes, such as those we propose, entail many challenges and must be carefully implemented and assessed.

"Despite these challenges," they add, "the USA has a long history of successfully improving the safety and value of prescription drugs, and substantial progress can still be made."

MedWire ( is an independent clinical news service provided by Springer Healthcare Limited. © Springer Healthcare Ltd; 2011

By Philip Ford