John LaMattina , PureTech Ventures04.03.13
Over the last decade, pharmaceutical companies began to become very transparent about their R&D productivity by periodically publishing the contents of their R&D pipelines on the company websites. The extent of this transparency differs for each company but, for the most part, one can get a handle on the number and types of compounds that a company has in Phases I, II and III. This was an unusual step to take for companies who previously had strongly believed that keeping these assets under wraps for as long as possible was important for maintaining a competitive edge in bringing new medicines to market.
This changed when investors and Wall Street financial analysts began questioning the productivity of R&D organizations. To show stakeholders that R&D investments were paying off, companies began to divulge these pipelines. Of particular interest were compounds in the later stage of development, as these compounds were relatively closer to a potential New Drug Application filing than the earlier assets. Late stage compounds had the better chance to impact a company’s fortunes in the short term, and so analysts paid close attention to the progress and results generated in the advanced clinical trials of these investigational medicines.
Actually, analysts went further than just tracking the progress of these compounds. They also combined this information with data about the clinical trial design that appears on ClinicalTrials.gov. By doing so, they gained insights into the main goal of the trial, the number of patients being studied, the length of the trial, etc. In fact, it’s not unusual for these analysts to critique the trial design in advance of the results being known.
As a result, and perhaps unintentionally, it now became difficult, if not impossible, to hide negative trial data. A great example of this occurred last summer when it was announced that Lilly’s experimental drug to treat Alzheimer’s Disease (AD), bapineuzumab, had failed a critical late stage critical trial. The press release that announced these negative results unleashed a torrent of articles about the need for AD treatments, the challenges facing this field of research, and also all the other negative studies that had occurred previously with other experimental drugs.
But the publicizing of negative trial data is not unique to a high profile field like AD. Negative trial outcomes with Merck’s cardiovascular drug, Tredaptive, Amgen’s osteoporosis drug, romosozumab, and Lilly’s rheumatoid arthritis, tabalumab, have all been reported recently, resulting in these compounds being dropped from development.
Negative data generated for compounds already on the market also get widely publicized. Sutent is a Pfizer drug with excellent efficacy against gastrointestinal stromal tumors (GIST), renal cell carcinoma (RCC) and pancreatic neuroendocrine cancerous tumors. But it has also been studied unsuccessfully in prostate cancer, liver cancer and breast cancer. All of these failures received as extensive press as occurred with the successes.
Be assured that there have been a lot of notable recent successes with new drugs discovered by the pharma industry. So, why am I dwelling on the negative? I recently attended Dr. Ben Goldacre’s talk at the New York City Skeptics Society, where he expressed his concern about pharma companies hiding negative trial data. I fear that the audience left that session with the view that the pharma industry does all it can to hide negative data and touts only positive studies. Given the scrutiny that the industry now undergoes by regulatory agencies, patient advocacy groups, physicians, Wall Street analysts and the media, hiding negative trial data is next to impossible.
This does not address Dr. Goldacre’s concern that the industry has not been compliant with posting all trial results on ClinicalTrials.gov in a timely fashion, something that I recently addressed. However, the high visibility of the pharmaceutical industry and genuine public interest in new medicines to treat diseases like AD, cancer, osteoporosis, etc., all have led to results of clinical trials now being very public, regardless of whether they are positive or negative. It’s pretty hard to hide things anymore.
Dr. John LaMattina is a senior partner at PureTech Ventures, author of Devalued and Distrusted: Can the Pharmaceutical Industry Restore its Broken Image? and the former president of Pfizer Global R&D, where he managed more than 13,000 scientists & professionals worldwide. This piece is adapted from Dr. LaMattina’s blog on forbes.com.
This changed when investors and Wall Street financial analysts began questioning the productivity of R&D organizations. To show stakeholders that R&D investments were paying off, companies began to divulge these pipelines. Of particular interest were compounds in the later stage of development, as these compounds were relatively closer to a potential New Drug Application filing than the earlier assets. Late stage compounds had the better chance to impact a company’s fortunes in the short term, and so analysts paid close attention to the progress and results generated in the advanced clinical trials of these investigational medicines.
Actually, analysts went further than just tracking the progress of these compounds. They also combined this information with data about the clinical trial design that appears on ClinicalTrials.gov. By doing so, they gained insights into the main goal of the trial, the number of patients being studied, the length of the trial, etc. In fact, it’s not unusual for these analysts to critique the trial design in advance of the results being known.
As a result, and perhaps unintentionally, it now became difficult, if not impossible, to hide negative trial data. A great example of this occurred last summer when it was announced that Lilly’s experimental drug to treat Alzheimer’s Disease (AD), bapineuzumab, had failed a critical late stage critical trial. The press release that announced these negative results unleashed a torrent of articles about the need for AD treatments, the challenges facing this field of research, and also all the other negative studies that had occurred previously with other experimental drugs.
But the publicizing of negative trial data is not unique to a high profile field like AD. Negative trial outcomes with Merck’s cardiovascular drug, Tredaptive, Amgen’s osteoporosis drug, romosozumab, and Lilly’s rheumatoid arthritis, tabalumab, have all been reported recently, resulting in these compounds being dropped from development.
Negative data generated for compounds already on the market also get widely publicized. Sutent is a Pfizer drug with excellent efficacy against gastrointestinal stromal tumors (GIST), renal cell carcinoma (RCC) and pancreatic neuroendocrine cancerous tumors. But it has also been studied unsuccessfully in prostate cancer, liver cancer and breast cancer. All of these failures received as extensive press as occurred with the successes.
Be assured that there have been a lot of notable recent successes with new drugs discovered by the pharma industry. So, why am I dwelling on the negative? I recently attended Dr. Ben Goldacre’s talk at the New York City Skeptics Society, where he expressed his concern about pharma companies hiding negative trial data. I fear that the audience left that session with the view that the pharma industry does all it can to hide negative data and touts only positive studies. Given the scrutiny that the industry now undergoes by regulatory agencies, patient advocacy groups, physicians, Wall Street analysts and the media, hiding negative trial data is next to impossible.
This does not address Dr. Goldacre’s concern that the industry has not been compliant with posting all trial results on ClinicalTrials.gov in a timely fashion, something that I recently addressed. However, the high visibility of the pharmaceutical industry and genuine public interest in new medicines to treat diseases like AD, cancer, osteoporosis, etc., all have led to results of clinical trials now being very public, regardless of whether they are positive or negative. It’s pretty hard to hide things anymore.
Dr. John LaMattina is a senior partner at PureTech Ventures, author of Devalued and Distrusted: Can the Pharmaceutical Industry Restore its Broken Image? and the former president of Pfizer Global R&D, where he managed more than 13,000 scientists & professionals worldwide. This piece is adapted from Dr. LaMattina’s blog on forbes.com.