One surprising aspect of the clinical trial landscape is just how few PhDs and physicians share their knowledge, either as industry insiders or academic researchers. The vastness of the global research enterprise does necessarily involve large numbers of other professionals. We know that. But hearing from the experts now and then is nice.

We get a large dose of such speakers every year at IIR’s annual Clinical Trials Congress. The 2009 event, in Philadelphia, had exactly what we had come to expect—high-level hand-wringing about the state of the industry, paired with ideas about what to do. 

The speakers yesterday framed the industry’s familiar productivity crisis (costlier trials, no blockbusters) in ways that seem more urgent. “Does it really make sense to have these large companies?” asked Joel Scherer, managing director of the Chorus project at Lilly. “We are slowly sinking.”

Nimble Niche

By which he meant, basically, the entire industry. He didn’t spend a lot of time on Lilly’s Chorus effort, but it appears to be an alternative R&D unit within the company. According to Scherer, it contributes perhaps a quarter of the candidates in Lilly’s early development portfolio. Much (perhaps all, according to its website) of the Chorus work is outsourced. The effort seems designed to make decisions far more quickly than in the rest of the company.

Scherer noted that his colleagues are trying to assess the viability of what Lilly calls a fully integrated pharmaceutical company. (This is the traditional big pharma model in which large companies do everything.) One new model is a firm that is the pivot for a network of other companies that each shoulder part of what large pharma companies formerly handled. Scherer conceded that he and everyone else in the industry are still formulating the right strategy. “We don’t have a play book,” he said. “The industry is trying new ideas. Some of them will work. Some of them won’t work.”

Data Dilemma

JNJ’s Centocor is, of course, a biotechnology island locked inside another large drug company. William Matthews is Centocor’s VP of biotechnology research and development management operations. “Our old model, our business model is not sustainable,” he told the Clinical Trials Congress. “The way we’ve designed trials in the past has been inefficient. We’ve got to think differently about how we think about trial design.”

At his firm, he said, colleagues are trying to minimize the amounts of unnecessary clinical data that is collected, a historic problem for the industry. (ClinPage has done an article and a podcast on what Genentech calls “smart” protocols.)

But the process of moving to a new model is not going to be simple. Matthews related that not that long ago, key stakeholders at Centocor could meet around a lunch table and agree on a plan. That’s not feasible today, with thousands of people in Centocor alone, much less tens of thousands at its parent company. Still, to a degree we have not often heard, Matthews put his finger on sheer size as something that may impede any needed industry transformation. “Our existing organizational structure, internally, can be a tremendous barrier,” he said. “We’re not a small company any more.  Our governance structure is a challenge.”

Information technology (IT) to help manage science or trials, he suggested, would be a splendid idea. If only it existed. He said his own firm’s IT group had proposed a number of solutions, but without success. The problems that industry faces, he implied, are simply too complex for IT to make a dent. Said Matthews: “We have never been able to implement them. We have struggled with them ever since I have been at Centocor. I would love to talk to people who have positive experiences, because I don’t.”

Futility Analysis

Surprisingly enough, the tone of the meeting grew even more somber with the presentation of Helen Sherman, chief pharmacy officer of The Regence Group. It’s a Pacific Northwest umbrella company for Blue Cross/Blue Shield insurers with 6 million members. Readers with a melancholy outlook may want to stop reading now, and go watch Comedy Central.

“I will be giving you a view point that is very different,” Sherman warned. Her company categorizes the published results of clinical trials into two piles. One is for studies that seem rigorous; the other is for junky, less useful research. The goal is to figure out which drugs are worth paying for—a radical idea in the free-spending U.S.

“We adopted high standards in reviewing evidence,” said Sherman. “We do audit each and every clinical trial. We use the Consort standards primarily. Is there proof in the data to justify the conclusions? Unfortunately, most of the time we find the data is unreliable.”

Economics 101

Sherman was crisp, clear and well-spoken. She was not an anti-industry crank. Her perspective was that the vast majority of clinical research sponsored by industry is designed in a manner that does not allow physicians or insurance companies to make objective, scientific assessments about the comparative merits of one medicine versus another.

We wonder if Sherman would say that $1 of the industry’s massive research budget is being spent to assess the comparative clinical or economic merits of medicines. Indeed, her presentation suggested that the industry may mostly be laboring mightily to find more convenient products, with simpler, daily dosing regimens, rather than safer, cheaper or more effective drugs.

Sherman’s assessment of the clinical benefit or larger societal value of industry’s research could not be more astringent. “Very few new products have the proof that they are more effective than existing products,” said Sherman.

It was news to us that the U.S. Congress is aware of all this. Politicians have appropriated more than $1 billion to help the federal Agency for Healthcare Research and Quality (AHRQ) begin to replicate cost-effectiveness assessments that are well developed in the U.K., Germany, Canada and other countries. The British National Institute for Health and Clinical Excellence (NICE) is the leader in assessing the cost-effectiveness of medicines. NICE has been in the news lately. It is often vilified as denying access to expensive drugs; its role in keeping a lid on the U.K. health care budget is less appreciated.

As demanding as the FDA is now, a new potential barrier for any product reaching the market is now on the horizon. Brandished by government or insurance companies, it would be some sort of hard quantifiable assessment of the economic value of a new medication. This new, fourth hurdle could add billions of dollars and years to the existing research paradigm of proving safety and efficacy—and winning regulatory approval. As a matter of legislation, adding any new, fourth hurdle would be certain to be a pitched political battle. That fight is one that pharmaceutical industry lobbyists could probably win in the end. So there is no need to panic. At least in the U.S.

Ray of Hope

But there is the rest of the world to consider. And some companies are already preparing for tough economic assessments of drugs. Pfizer, for one, is girding for the future rather than hiding from it.

Pfizer’s Gregana Zlateva, director of global outcomes research, gave one of the more impressive presentations of the day. Providing few details, she implied that that the big company is starting to incorporate economic value considerations into clinical trial design and pipeline development. She delineated the methods by which the values of medicines can be calculated and presented to regulators or insurers, much as efficacy and safety data are now marshaled. It can be done.

From her perspective, the assessment of any drug’s economic value appears to be an inevitable element in assembling a pipeline of products that payors will allow to be prescribed all over the globe. While this may be difficult, daunting territory for some in the industry, one message from Zlateva’s talk is that the consideration of economic issues is doable. “We can actually document and quantify the benefits of our medicines,” she said. “The question everybody has is, are these drugs really worth paying for? We’re trying more and more to have programs reflective of the marketplace as well as regulatory concerns.”