Drug companies in the U.S. and Europe, facing the prospect of EU officials forcing them toward more transparency, have now agreed to share some clinical research data. Does this mean a new era of openness or is this just lip service?
The European Federation of Pharmaceutical Industries and Associations (EFPIA) and the U.S.’s Pharmaceutical Research and Manufacturers of America (PhRMA) released a joint set of principles last week outlining a plan to allow greater access to clinical drug trial data so that qualified scientists could, in theory, analyse the results and confirm or refute the drug companies’ claims.
It is believed that less than half of research trial data is currently published, with critics saying the data is withheld so that pharmaceutical companies can overstate the effectiveness of their drugs, downplay side effects and maintain a competitive edge over each other — all at the expense of patients.
So strong are the concerns that the European Medicines Agency (EMA) has begun to formulate regulations that would force the companies to publish such data. Many have resisted such interference, as had PhRMA and EFPIA , with PhRMA on record as late as this January formally opposing any wider changes to data publishing.
However, with the publishing of a new set of transparency principles, both companies appear to be at least offering tacit agreement with the EMA initiative. So what are the principles, and what is going on here?
The New Principles PhRMA and EFPIA Have Agreed On
Due to be implemented in January 2014, the Principles for Responsible Clinical Trial Data Sharing: Our Commitment to Patients and Researchers outline that data requests will be allowed under certain conditions.
All such requests will first be referred to review boards established by each company. Those boards may include independent scientists and health care professionals, as well as company officials, to offer a balanced assessment of data requests. However, the individual makeup of each review panel will be set by the companies and there will be no mandate that companies have to include outside reviewers.
Researchers will at the same time have to demonstrate that they have the qualifications to handle the data and that their use of the data will fulfill a legitimate research question, though exactly how that is defined appears quite nebulous at this stage.
Other commitments outlined in the document include an agreement for companies to work more openly with regulators to provide a detailed summary of relevant information for patients who participate in clinical trials. Moreover, companies will also agree to make available to the public summaries of clinical study reports when new medicines are approved by Europe and U.S. regulators.
The principles encourage publication of positive and negative results from what are known as all “phase III” trials, where a prospective treatment is given to large groups of people to confirm its effectiveness and monitor side effects — this even if those results do not provide evidence for the effectiveness of a given drug or show signs of above acceptable side effects.
The principles explicitly state, however, that no data will be shared that could compromise pharmaceutical industry competitiveness. The principles also reserve far reaching powers to protect data that companies deem could compromise patient confidentiality.
Given the wider pharmaceutical industry has resisted attempts to force more transparency in data publishing, why have PhARMA and EFPIA suddenly changed course? And is this really all it seems?
What Does this Change Mean for Patients and the Pharmaceutical Industry?
In short, not very much.
It is important to point out this voluntary change would appear to be an attempt to forestall the industry being forced into more stringent measures.
This is not to minimize the changes the new principles could create, but to put them in context: the industry seems to be attempting to retain its freedom by self regulating more closely.
Also, this announcement appears to be part of a carefully coordinated exercise in resisting the more stringent EU regulations.
The Guardian this week alleged PhRMA and the European Federation of Pharmaceutical Industries and Associations (EFPIA) have engaged with patient groups to try to drum up resistance against the EMA forcing a wider release of data.
This came to light after the Guardian saw communications from Richard Bergstrom, director general of EFPIA, that went to directors and legal counsel at drug companies at Roche, Merck, Pfizer, GSK, AstraZeneca, Eli Lilly, Novartis and several smaller companies.
The email describes a four-pronged campaign that starts with “mobilising patient groups to express concern about the risk to public health by non-scientific re-use of data”. Translated, that means patient groups go into bat [sic] for the industry by raising fears that if full results from drug trials are published, the information might be misinterpreted and cause a health scare.
A source in the European parliament, who is close to the negotiations over the clinical trials directive, said he had experienced intense lobbying from patient groups. “We’ve witnessed this sort of activity in recent months, and it’s a concern if the pharmaceutical industry is behind some of it. They are trying to weaken some of the transparency proposals and that’s clear from the amount of lobbying we’ve had,” he said.
Concerns have previously been raised about how the pharmaceutical industry has courted patient groups, using their assumed status as representing wider patient concerns to lobby for the so-called Big Pharma causes.
Seen through this lens, PhRMA and EFPIA’s new principles may ring rather hollow now that it appears while they have been trumpeting their move toward openness, they have also been making a concerted effort to raise spurious concerns about wider transparency; EU regulators have made several assurances that any patient confidentiality concerns could be handled.
All that said, those in the medical community have said the principles, if acted upon, could be significant.
“What they are doing would have been thought to be inconceivable even a short time ago,”¯ said Dr. Harlan M. Krumholz, a cardiologist at Yale who recently oversaw an outside review of a treatment by the device maker Medtronic. “If these companies truly fulfill these promises, then they will have made an important contribution to science and the common good.”¯ But he said their efforts would need to be monitored.
So what does this mean for us, the patients?
It seems the principles outlined above add up to very little right now.
If adhered to, however, they really could represent a large step in the right direction to ensuring more openness and greater oversight.
This should be a first among many steps, though, and not an end goal.
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