Clinical Trials – Discovering and Sharing Knowledge

02.02.13 | By Matt Bennett

Developing a new medicine to fight diseases such as cancer or diabetes can require 12-15 years of research and over $1 billion. A foundational aspect of the research is scientifically rigorous, well-regulated clinical trials.

Recently, critics such as Dr. Ben Goldacre have sought to cast doubt on this vital research, contending that information generated from clinical trials is incomplete and insufficiently shared.

Such unfounded yet sensational claims may generate headlines, but they do not help make new medicines. Clinical trials made possible the more than 340 new medicines approved by the Food and Drug Administration (FDA) over the last decade, and 35 novel medicines in FY 2012 alone. Criticizing the research process without acknowledging the vigorous oversight and commitment to safe and ethical conduct is a disservice to the tens of thousands of scientists exploring new medicines, the patients who participate in trials and advance our scientific knowledge, and the millions of patients hoping for cures.

Dr. Goldacre’s calls to release all patient-level trial data pose a great risk to patient privacy and could discourage individuals considering participating in clinical trials and companies who want to invest in research. Inappropriate release of trial data could lead to second-guessing of the already rigorous regulatory approval process, which would be disastrous for patients and doctors. The FDA has the most advanced review process for potential new medicines, and it is continuously improving its scientific knowledge and regulatory capabilities.

The FDA releases a comprehensive summary approval package with each new medicine, which includes analysis of all significant clinical trials used for the approval process. FDA constantly monitors approved medicines and updates approved labeling accordingly. The approved labeling contains the synthesis of all known clinical data about a medicine. The Drugs@FDA database enables searches for information about FDA-approved brand name and generic medicines and therapeutic biological products, including labels, patient information, approval letters, and other information

Our current system includes sharing trial results with government regulators, the academic and medical communities, physicians and the public through submissions to the FDA, presentations at medical conferences and publication in peer-reviewed journals. As of February 2, 2013, ClinicalTrials.gov listed 139,847 studies with locations in all 50 states and in 182 countries. There is an enormous amount of clinical data available today.

The current clinical trials process is working well, fostering new medicines and spreading new scientific knowledge to researchers, physicians, patients and regulators. These committed participants in the clinical trials system are always seeking to improve, welcoming of constructive debates. Dr. Goldacre’s claims may gain some publicity, but they cast accusations rather than seeking common ground.

Comment (3) Add new comment

Ben Goldacre (not verified) 05/09/13 12:05

It is remarkable that PhRMA have deleted the 20 comments that were on this February piece - mostly highly critical of PhRMA - and then begun promoting it again. I'll be addressing this, and other odd claims by PhRMA, at length. Dr Ben Goldacre www.badscience.net

Ben Goldacre (not verified) 05/09/13 02:05

I am reposting my comment of 2nd February 2013, which has been deleted by PhRMA this week, along with the other 20 comments on this blog post. Needless to say I have a complete archive. Only PhRMA can explain why they have deleted these comments before promoting this blog post again on Twitter. It is unfortunate that PhRMA's comment system appears to remove all paragraph marks and formatting. My comment of 2nd Feb 2013 read as follows: The pharmaceutical industry makes many excellent products, but there are also many well-documented problems in industry, and indeed throughout medicine. Industry should be able to engage in a serious discussion around how to fix these problems, rather than issuing outright denials. The best available evidence, from the most current review of all the scientific literature, shows that the results of clinical trials are routinely left unpublished, both for industry trials and independent academic trials. http://www.hta.ac.uk/fullmono/mon1408.pdf The new legislation that should have fixed this - requiring results to be reported at clinicaltrials.gov within a year of completion - has been ignored by the overwhelming majority of clinical trials, as is shown in this 2012 academic paper (60% of industry trials failed to comply with the law). http://www.bmj.com/content/344/bmj.d7373 These are impeccable sources: a systematic review, and a top five medical academic journal. To say that these concerns are "unfounded" is untenable, and it is surely unwise for PhRMA to risk its reputation by issuing such outlandish denials, when all the available evidence goes against them. More peculiar is Mr Bennett's claim that this is about individual patient data, rather than the summary results of clinical trials. ("Dr. Goldacre’s calls to release all patient-level trial data pose a great risk to patient privacy and could discourage individuals considering participating in clinical trials", etc). Lay readers may need some background here. Individual patient data in huge files is analysed to create the final overall summary results of clinical trials, showing which treatment worked best. I don't call for the sharing of individual patient data, anywhere in my New York Times piece. I simply call for the results of clinical trials to be reported publicly, in full, so that doctors and patients can use this information to make informed decisions about healthcare. http://www.nytimes.com/2013/02/02/opinion/health-cares-trick-coin.html The alltrials.net campaign, similarly, calls only for the reporting of results, not for confidential individual patient data. In doing so, it is supported by major medical organisations as diverse as the UK Medical Research Council, the Wellcome Trust, the National Rheumatoid Arthritis Society, and the US National Physicians Alliance. There is nothing controversial about recognising that the problem of missing trial results exists, and there is nothing controversial about calling for it to be addressed. It is notable that the ABPI (the UK pharmaceutical industry body, and the equivalent of PhRMA) have also tried to derail discussion on access to clinical trial results, using the exact same strategy as PhRMA, by pretending that the alltrials.net campaign is asking for confidential individual patient data. The ABPI know, as do PhRMA, that the campaign is simply asking for access to results. http://www.alltrials.net/news/abpi/ However, on this issue, both PhRMA and Mr Bennett do also seem to be very out of date, especially with regard to international developments in regulation. The European Medicines Agency hold huge quantities of trials, and they have already announced that they will be sharing all individual patient data, starting from 2014. There are many interesting challenges around how to do this, and how to ensure that individual patients' privacy is protected. I discuss this in my book, Bad Pharma; I discussed it as a panel member at the European Medicines Agency event that announced their new policy; and, along with colleagues, I'm on one of five European Medicines Agency advisory panels discussing how to manage these challenges cautiously. http://www.ema.europa.eu/ema/index.jsp?curl=pages/special_topics/general/general_content_000556.jsp The issue of trial results, meanwhile, is unarguable: we need the results of all clinical trials to make informed decisions. Industry, doctors, and patients will all benefit from working together to achieve full transparency on this issue, but for now, the results of clinical trials are being withheld from doctors and patients. This needs to be fixed, through constructive engagement. Pretending the problem simply doesn't exist, as Mr Bennett and PhRMA do here, is unethical, because it perpetuates the current situation, which harms patients. More than that, they do a great disservice to the many ethical professionals working in the pharmaceutical industry, who are striving for positive change. If PhRMA really believe the problem of withheld trial results does not exist, they should be willing to sign up and support the campaign at alltrials.net for all trial results to be reported.

Matt Bennett (not verified) 05/13/13 06:05

Ben, thanks for your comments. The entire PhRMA.org web site was redesigned in April, and the majority of the PhRMA.org content -- including Catalyst blog posts -- was moved to a new technical platform. Visitor comments were not moved for any old Catalyst posts, however – including the post you noted today. This was due mainly to a technical issue -- basically, comments are on a different system than blog posts, although they display on the same page. It was never our intent to censor any opinions. We truly want to engage in meaningful conversations with you and others, which is why we welcomed your comments several months ago and had the online conversation in February. PhRMA continues to encourage others’ opinions and foster an environment for open debate surrounding key issues in the biopharmaceutical industry.