Info & Opinion
April 25, 2019
With news about big data, Aetna, Covance, GNS, Scrip, Quintiles, PPD, Icon, BioClinica, Merge, Medidata and GSK.
With news about FDA, CTMS, PMG Research, Inclinix, EMA, Hemofarm, Parexel and the Korea Drug Development Fund
David Underwood of Quanticate says some firms are giving short shrift to the basics of clinical trials
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There is a new federal effort to organize disease registries. It's a meta-repository of all disease registries—a big list. The FDA will be advising on its design but play no formal role in creating or running it, according to Richard Gliklich, president and CEO of Outcome.
His company is gathering expert brainpower on the project and will build the registry. The details of the design are still to be finalized. The Cambridge, Mass., firm specializes in running patient registries and other databases. It typically supports hundreds of them at any given time.
The grant is from the Agency for Healthcare Research and Quality (AHRQ), the primary federal clearinghouse for figuring out if treatments work and are cost-effective. To the extent that any part of the U.S. government is doing research on how to compare the costs and clinical profiles of two drugs, AHRQ is it.
Gliklich says the registry of registries will serve several roles. It will be a single place for patients and doctors to search for registries, preventing the need to Google "diabetes registry" and sort through eleven trillion search results.
On the technical side, the meta-registry will attempt to standardize sets of data elements and definitions. That could ensure that scientific findings from different registries can be more intelligently compared to each other. On the scientific front, the new registry will allow organizations that have not published the medical insights from their registries to do so.
Finally, the registry of registries could help the scientific community, drug industry and insurers, illuminating the ways that drugs work in community practice. Outside the precisely controlled auspices of a clinical trial, where variables like concomitant medications and other ailments have been excluded, medicines often work very differently.
Yes, traditional efficacy data will always be crucial, Gliklich allows. "We also need complementary information that generalizes those results to the group the physician or payor is responsible for," says Gliklich. Perhaps cardiovascular patients in Utah have genetic traits that make a particular medicine more effective. That's where registries can help.
As some readers may recall, Gliklich and top lieutenants at Outcome literally wrote the AHRQ textbook on post-approval research, a topic ClinPage covered here. That volume will be updated as part of the registry effort.
No Regulatory Burden
And while much is unknown about the new registry, Gliklich says there will be no legal burden on industry to provide additional information. "It is not a regulatory imperative. Voluntary participation has to be driven by incentives," Gliklich says.
In time, the project could identify overlapping registries. In orthopedics, for example, Gliklich says that a relatively cursory search turned up more than 50 registries. He adds: "There are a lot of activities going on all over the country that are not necessarily aware of each other. They could have benefited from collaborating." That's especially true for small academic groups or patient advocacy organizations that may not have adequate funds for a registry in the first place.
Still, no registries will be obliged to join forces. The meta-registry will simply allow the physician who wants to start orthopedic registry #53 to think a bit about whether she might want to contribute to another project. "The goal is not to force collaboration," Gliklich says.
In a wider context, it's clear that the registry of registries, by standardizing methods and comparing published data, could focus post-approval research. "It's better to have fewer well-designed registries. You probably don't need hundreds of single-institution registries when you could consolidate them," said Gliklich. In some cases, similar data definitions or clinical benchmarks may allow registries to be electronically or statistically consolidated. Says Gliklich: "People are looking for standardization. No one wants to reinvent the wheel."
Gliklich allows that some medical specialties have tended to create bigger, more sophisticated registries. He's confident that the dissemination of best practices will expedite that process across more areas of research, and lead to smaller numbers of higher-quality registries.
He says that when there are multiple drugs available for the same indication, clinicians frequently don't have large amounts of data to help them choose the best medication. Sponsors themselves have been so focused on finding efficacy and safety data that the comparative merits or flaws of various products in a particular population have been an afterthought. "A lot is likely to change over the course of the next 2-3 years," says Gliklich. "There will be a bigger drive to get efficacy and comparative effectiveness information into the literature to support those types of decisions."
"When you see efficacy studies and compare them to the general population, there is often significant difference," he continues, owing to factors such as ethnicity, genetics or demographics. Regulators and clinicians would like more data from the real world, even if that data is not as immaculate as what trials produce.
Indeed, American officials are trying to catch up to comparative effectiveness efforts in the U.K. and other European countries that have traditionally spent more resources assessing whether new medicines are cost-effective. Gliklich sees the U.S. moving in the same direction. "The comparative effectiveness arena is where safety was in 2005. It's going to grow. It is tracking very similarly," Gliklich says.
Although there is not yet any formal regulatory demand for comparative effectiveness data, he says that some sponsors are already designing trials to create it. Notes Gliklich: "The smarter pharmas are considering how to get information on those comparative endpoints earlier in development. Getting an understanding of how patient quality of life is affected in Phase II, for example, is becoming more important."
In some ways, he says, sophisticated sponsors are putting themselves in the shoes of someone looking at the fourth drug in a particular class, and trying to design registries and trials in ways that will produce information to help government and private payors make a more complex, multidimensional decision about that product than is possible when only safety and efficacy are considered.