Delays in Cancer Drug Approvals Cost Lives

Roxanne Nelson

September 08, 2015

DENVER — A "huge" number of life-years is being unnecessarily lost because of delays in cancer drug approvals, according to research presented here at the 16th World Conference on Lung Cancer.

Every hour lost to the cancer drug regulatory process costs 29 life-years in the United States and 260 life-years worldwide, according to lead author David Stewart, MD, from the division of medical oncology at the Ottawa Hospital in Ontario, Canada, who presented the findings.

New anticancer drugs can prolong survival, but often in only a modest way. In fact, on average it is just a few months, Dr Stewart reported during a press briefing. "However, for some, it is 2 or 3 years, or longer, and despite being fairly modest [for most], it's very important to patients."

In addition, around the world, regulatory complexity has been steadily increasing. This has driven the cost of drug development to well above the cost of inflation, which contributes directly to high health costs, he pointed out.

"It has also shown a minimal demonstrable impact on clinical trial safety, despite the increased regulatory compliance," said Dr Stewart. "But the increased regulatory compliance slows progress and leads to increased suffering and premature deaths."

Although accelerated drug-approval processes initiated by the US Food and Drug Administration have sped up the process, just "removing the phase 3 trial component" required to get drugs approved does nothing to fix the escalating burden of clinical research bureaucracy."

These accelerated approval mechanisms will have progressively less impact unless the clinical research bureaucracy is fixed. "It's a shorter distance to the goal post, but the mud on the way to these closer goal posts keeps getting deeper and deeper," he explained.

29 Life-Years Lost for Each Hour of Delay

Dr Stewart and his colleagues assessed cancer therapies that went through phase 3 trials from 2001 to 2015. They excluded adjuvant trials and research for uncommon malignancies. To determine the number of life-years potentially lost for each year required for drug approval, they multiplied the improvement in median survival (in years) by the estimated number of patients worldwide who die annually from the cancer type.

There were 21 approvals for drugs designed to treat ten different malignancies. When the combined impact of all tumor sites and drugs were considered together, the authors calculated that there were 29 life-years lost in North America for each hour of delay in therapy approval (one for every 2 minutes of delay) and 260 life-years lost worldwide for each hour of delay (one for every 14 seconds of delay).

The authors note that these data do not take into account the impact of drugs that were prematurely abandoned, drugs that were unevaluable because of crossover or missing survival data, drugs that are still being evaluated, and drugs being developed for nonmalignant lethal diseases.

Clearly, the survival gains associated with those drugs are only modest, Dr Stewart reiterated. However, there would be a large negative impact associated with approval delays even if other factors — such as comorbidities, performance status, and ability to pay — limited the number of patients treated to a fraction of the total dying from a specific malignancy.

He emphasized that everyone "shares the responsibility" for this, including investors, research staff, institutions, institutional review board, sponsors, contract research organizations, regulators, tort systems, and the press.

We all share responsibility to solve this.

"We all share responsibility to solve this," he added.

During a discussion of the study, David Sturges, JD, from the Lung Cancer Foundation of America, did not offer any solutions on how to reduce the period of time between application for a new drug and its entry into the market.

However, he did note that he is "hopeful that time has reduced some of the problems, at least to a small degree." Sturges, who is a lung cancer survivor and advocate, pointed out, for example, that there was a "shrinking of time" between drugs that received approval in 2004 and 2006 and those that received approval 2008 and 2014. "Whether that means that our regulatory system has evolved, I don't know, but I am hopeful."

16th World Conference on Lung Cancer (WCLC): Abstract ORAL12.05. Presented September 7, 2015.


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