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Clinical trials are better, faster, cheaper with big data

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Clinical trials are better, faster, cheaper with big data


“One of the most difficult parts of my job is enrolling patients into studies,” says Nicholas Borys, chief medical officer for Lawrenceville, N.J., biotechnology company Celsion, which develops next-generation chemotherapy and immunotherapy agents for liver and ovarian cancers and certain types of brain tumors. Borys estimates that fewer than 10% of cancer patients are enrolled in clinical trials. “If we could get that up to 20% or 30%, we probably could have had several cancers conquered by now.”

Clinical trials test new drugs, devices, and procedures to determine whether they’re safe and effective before they’re approved for general use. But the path from study design to approval is long, winding, and expensive. Today,researchers are using artificial intelligence and advanced data analytics to speed up the process, reduce costs, and get effective treatments more swiftly to those who need them. And they’re tapping into an underused but rapidly growing resource: data on patients from past trials

Building external controls

Clinical trials usually involve at least two groups, or “arms”: a test or experimental arm that receives the treatment under investigation, and a control arm that doesn’t. A control arm may receive no treatment at all, a placebo or the current standard of care for the disease being treated, depending on what type of treatment is being studied and what it’s being compared with under the study protocol. It’s easy to see the recruitment problem for investigators studying therapies for cancer and other deadly diseases: patients with a life-threatening condition need help now. While they might be willing to take a risk on a new treatment, “the last thing they want is to be randomized to a control arm,” Borys says. Combine that reluctance with the need to recruit patients who have relatively rare diseases—for example, a form of breast cancer characterized by a specific genetic marker—and the time to recruit enough people can stretch out for months, or even years. Nine out of 10 clinical trials worldwide—not just for cancer but for all types of conditions—can’t recruit enough people within their target timeframes. Some trials fail altogether for lack of enough participants.

What if researchers didn’t need to recruit a control group at all and could offer the experimental treatment to everyone who agreed to be in the study? Celsion is exploring such an approach with New York-headquartered Medidata, which provides management software and electronic data capture for more than half of the world’s clinical trials, serving most major pharmaceutical and medical device companies, as well as academic medical centers. Acquired by French software company Dassault Systèmes in 2019, Medidata has compiled an enormous “big data” resource: detailed information from more than 23,000 trials and nearly 7 million patients going back about 10 years.

The idea is to reuse data from patients in past trials to create “external control arms.” These groups serve the same function as traditional control arms, but they can be used in settings where a control group is difficult to recruit: for extremely rare diseases, for example, or conditions such as cancer, which are imminently life-threatening. They can also be used effectively for “single-arm” trials, which make a control group impractical: for example, to measure the effectiveness of an implanted device or a surgical procedure. Perhaps their most valuable immediate use is for doing rapid preliminary trials, to evaluate whether a treatment is worth pursuing to the point of a full clinical trial.

Medidata uses artificial intelligence to plumb its database and find patients who served as controls in past trials of treatments for a certain condition to create its proprietary version of external control arms. “We can carefully select these historical patients and match the current-day experimental arm with the historical trial data,” says Arnaub Chatterjee, senior vice president for products, Acorn AI at Medidata. (Acorn AI is Medidata’s data and analytics division.) The trials and the patients are matched for the objectives of the study—the so-called endpoints, such as reduced mortality or how long patients remain cancer-free—and for other aspects of the study designs, such as the type of data collected at the beginning of the study and along the way.

When creating an external control arm, “We do everything we can to mimic an ideal randomized controlled trial,” says Ruthie Davi, vice president of data science, Acorn AI at Medidata. The first step is to search the database for possible control arm candidates using the key eligibility criteria from the investigational trial: for example, the type of cancer, the key features of the disease and how advanced it is, and whether it’s the patient’s first time being treated. It’s essentially the same process used to select control patients in a standard clinical trial—except data recorded at the beginning of the past trial, rather than the current one, is used to determine eligibility, Davi says. “We are finding historical patients who would qualify for the trial if they existed today.”

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This content was produced by Insights, the custom content arm of MIT Technology Review. It was not written by MIT Technology Review’s editorial staff.

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The hunter-gatherer groups at the heart of a microbiome gold rush

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The hunter-gatherer groups at the heart of a microbiome gold rush


The first step to finding out is to catalogue what microbes we might have lost. To get as close to ancient microbiomes as possible, microbiologists have begun studying multiple Indigenous groups. Two have received the most attention: the Yanomami of the Amazon rainforest and the Hadza, in northern Tanzania. 

Researchers have made some startling discoveries already. A study by Sonnenburg and his colleagues, published in July, found that the gut microbiomes of the Hadza appear to include bugs that aren’t seen elsewhere—around 20% of the microbe genomes identified had not been recorded in a global catalogue of over 200,000 such genomes. The researchers found 8.4 million protein families in the guts of the 167 Hadza people they studied. Over half of them had not previously been identified in the human gut.

Plenty of other studies published in the last decade or so have helped build a picture of how the diets and lifestyles of hunter-gatherer societies influence the microbiome, and scientists have speculated on what this means for those living in more industrialized societies. But these revelations have come at a price.

A changing way of life

The Hadza people hunt wild animals and forage for fruit and honey. “We still live the ancient way of life, with arrows and old knives,” says Mangola, who works with the Olanakwe Community Fund to support education and economic projects for the Hadza. Hunters seek out food in the bush, which might include baboons, vervet monkeys, guinea fowl, kudu, porcupines, or dik-dik. Gatherers collect fruits, vegetables, and honey.

Mangola, who has met with multiple scientists over the years and participated in many research projects, has witnessed firsthand the impact of such research on his community. Much of it has been positive. But not all researchers act thoughtfully and ethically, he says, and some have exploited or harmed the community.

One enduring problem, says Mangola, is that scientists have tended to come and study the Hadza without properly explaining their research or their results. They arrive from Europe or the US, accompanied by guides, and collect feces, blood, hair, and other biological samples. Often, the people giving up these samples don’t know what they will be used for, says Mangola. Scientists get their results and publish them without returning to share them. “You tell the world [what you’ve discovered]—why can’t you come back to Tanzania to tell the Hadza?” asks Mangola. “It would bring meaning and excitement to the community,” he says.

Some scientists have talked about the Hadza as if they were living fossils, says Alyssa Crittenden, a nutritional anthropologist and biologist at the University of Nevada in Las Vegas, who has been studying and working with the Hadza for the last two decades.

The Hadza have been described as being “locked in time,” she adds, but characterizations like that don’t reflect reality. She has made many trips to Tanzania and seen for herself how life has changed. Tourists flock to the region. Roads have been built. Charities have helped the Hadza secure land rights. Mangola went abroad for his education: he has a law degree and a master’s from the Indigenous Peoples Law and Policy program at the University of Arizona.

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The Download: a microbiome gold rush, and Eric Schmidt’s election misinformation plan

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The Download: a microbiome gold rush, and Eric Schmidt’s election misinformation plan


Over the last couple of decades, scientists have come to realize just how important the microbes that crawl all over us are to our health. But some believe our microbiomes are in crisis—casualties of an increasingly sanitized way of life. Disturbances in the collections of microbes we host have been associated with a whole host of diseases, ranging from arthritis to Alzheimer’s.

Some might not be completely gone, though. Scientists believe many might still be hiding inside the intestines of people who don’t live in the polluted, processed environment that most of the rest of us share. They’ve been studying the feces of people like the Yanomami, an Indigenous group in the Amazon, who appear to still have some of the microbes that other people have lost. 

But there is a major catch: we don’t know whether those in hunter-gatherer societies really do have “healthier” microbiomes—and if they do, whether the benefits could be shared with others. At the same time, members of the communities being studied are concerned about the risk of what’s called biopiracy—taking natural resources from poorer countries for the benefit of wealthier ones. Read the full story.

—Jessica Hamzelou

Eric Schmidt has a 6-point plan for fighting election misinformation

—by Eric Schmidt, formerly the CEO of Google, and current cofounder of philanthropic initiative Schmidt Futures

The coming year will be one of seismic political shifts. Over 4 billion people will head to the polls in countries including the United States, Taiwan, India, and Indonesia, making 2024 the biggest election year in history.

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Navigating a shifting customer-engagement landscape with generative AI

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Navigating a shifting customer-engagement landscape with generative AI


A strategic imperative

Generative AI’s ability to harness customer data in a highly sophisticated manner means enterprises are accelerating plans to invest in and leverage the technology’s capabilities. In a study titled “The Future of Enterprise Data & AI,” Corinium Intelligence and WNS Triange surveyed 100 global C-suite leaders and decision-makers specializing in AI, analytics, and data. Seventy-six percent of the respondents said that their organizations are already using or planning to use generative AI.

According to McKinsey, while generative AI will affect most business functions, “four of them will likely account for 75% of the total annual value it can deliver.” Among these are marketing and sales and customer operations. Yet, despite the technology’s benefits, many leaders are unsure about the right approach to take and mindful of the risks associated with large investments.

Mapping out a generative AI pathway

One of the first challenges organizations need to overcome is senior leadership alignment. “You need the necessary strategy; you need the ability to have the necessary buy-in of people,” says Ayer. “You need to make sure that you’ve got the right use case and business case for each one of them.” In other words, a clearly defined roadmap and precise business objectives are as crucial as understanding whether a process is amenable to the use of generative AI.

The implementation of a generative AI strategy can take time. According to Ayer, business leaders should maintain a realistic perspective on the duration required for formulating a strategy, conduct necessary training across various teams and functions, and identify the areas of value addition. And for any generative AI deployment to work seamlessly, the right data ecosystems must be in place.

Ayer cites WNS Triange’s collaboration with an insurer to create a claims process by leveraging generative AI. Thanks to the new technology, the insurer can immediately assess the severity of a vehicle’s damage from an accident and make a claims recommendation based on the unstructured data provided by the client. “Because this can be immediately assessed by a surveyor and they can reach a recommendation quickly, this instantly improves the insurer’s ability to satisfy their policyholders and reduce the claims processing time,” Ayer explains.

All that, however, would not be possible without data on past claims history, repair costs, transaction data, and other necessary data sets to extract clear value from generative AI analysis. “Be very clear about data sufficiency. Don’t jump into a program where eventually you realize you don’t have the necessary data,” Ayer says.

The benefits of third-party experience

Enterprises are increasingly aware that they must embrace generative AI, but knowing where to begin is another thing. “You start off wanting to make sure you don’t repeat mistakes other people have made,” says Ayer. An external provider can help organizations avoid those mistakes and leverage best practices and frameworks for testing and defining explainability and benchmarks for return on investment (ROI).

Using pre-built solutions by external partners can expedite time to market and increase a generative AI program’s value. These solutions can harness pre-built industry-specific generative AI platforms to accelerate deployment. “Generative AI programs can be extremely complicated,” Ayer points out. “There are a lot of infrastructure requirements, touch points with customers, and internal regulations. Organizations will also have to consider using pre-built solutions to accelerate speed to value. Third-party service providers bring the expertise of having an integrated approach to all these elements.”

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