Not Accessible: Data From Pivotal Trials of New Cancer Drugs 

Pam Harrison

August 04, 2022

Despite a commitment from the pharmaceutical industry to improve data transparency,  a new study shows that fewer than half of the pivotal trials of new cancer drugs allow access to individual patient data (IPD).

"Sharing of IPD is critical for highly used newer medicines when the evidence underpinning their use is almost exclusively derived from clinical trials supporting the medicines' approval," say the authors, led by Natansh Modi, BPharm (Hons), a PhD candidate at the Clinical Cancer Epidemiology Lab at Flinders University, Adelaide, Australia.

The study was published online July 28 in JAMA Oncology.

It found 304 trials that supported the registration of 115 new anticancer drugs over the last 10 years.

Only 136 of these trials (45%) had individual patient data available for sharing.

They included trials of three of the globally top-selling anticancer drugs (nivolumab [Opdivo], pembrolizumab [Keytruda], and pomalidomide [Pomalyst]), which together generated over $30 billion in revenue in 2020.

For these three best-selling drugs, 90% of the trial data was unavailable. 

"It is time that global regulators, governments, and journals establish mandates for data transparency on all major trials investigating registered medicine, [as] their purpose must be to protect and maximize public health and ensure the contributions of trial participants and their families reach their full potential," Modi added in a statement.

IPD can be used to "unlock insights into the risks and benefits of medicines in unexplored patient groups," he commented.

"If the data isn't made available, it can't be put to good use and trial participants and their families deserve better," he said.

Eligibility Criteria

The study evaluated the eligibility of independent, qualified researchers to access IPD from oncology trials that supported the US Food and Drug Administration's approval of new anticancer medicines within the past 10 years.

The medications were owned or co-owned by 49 pharmaceutical companies and their approval was based on the results of 304 industry-sponsored trials. 

The team reports that in the majority of cases (79%), the trials required an inquiry to the sponsor to establish whether the trial was eligible for data sharing.

The most common reason for not sharing IPD eligibility was that the trial was still ongoing, cited by 53% of industry sponsors. Because of this, the IPD for the results reported in the product label were not available for sharing.

"This is the same reason given by Pfizer and Moderna for the clinical trial data on their respective COVID-19 vaccines not currently being available," senior author Ash Hopkins, MD, leader of the Clinical Cancer Epidemiology Lab at Flinders University, said in a statement.

However, this excuse disregards any commitment to data transparency and it should not hinder the release of key data implicated in the global release of medicines to tens of thousands of people, he added.  

Data on Thousands of Patients

The authors note that IPD were eligible for sharing from 136 industry-sponsored trials that had results summarized in the product labels of 60 anticancer medicines approved by the FDA over the past 10 years.

"These trials included more than 70,000 patients and provide an immense opportunity for independent scientific investigations by regulators, clinicians, and researchers," the authors write.

On the other hand, IPD were unavailable for sharing from another 168 industry-sponsored trials that had results summarized in the product labels of 78 anticancer medications approved by the FDA over the past 10 years.

"These trials included more than 85,000 participants," researchers point out.

"It is of great concern that this wealth of anonymized IPD remains unavailable to independent investigation despite the rollout of medicines within the US and global cancer populations based on the product label results," the authors comment. As these trials form the basis of safety and efficacy claims for new medications, "we question whether it is justified that the data are unavailable to independent scrutiny."

Modi and Hopkins have reported no relevant financial relationships.

JAMA Oncology. Published online July 28, 2022. Full text

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