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Neuroscience Trials of the Future Proceedings of a Workshop (2016) / Chapter Skim
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5 The Regulatory Landscape: International Opportunities and Challenges
Pages 43-52

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From page 43...
... . • Integrating clinical research networks is one way to ensure more efficient trials (Califf)
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From page 44...
... , one central goal is to optimize trial design approaches to get products to patients who desperately need them while ensuring that these products undergo appropriate evaluation for safety and effectiveness. REGULATORY PERSPECTIVES FROM THE UNITED STATES Robert Califf, Commissioner of Food and Drugs at the FDA, said that within the FDA, there is a shared view that the best medical outcomes occur when doctors and other health care providers and patients are armed with high- quality evidence to support what they do, and this is most likely to happen when the clinical trials and observational studies are actually done in practice.
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From page 45...
... Underlyying all of thhese steps, saaid Califf,, is measurem ment and educcation both inn the clinical ttrials arena annd in clin nical practice,, which he deescribed as a learning heaalth care systeem (see Fiigure 5-1)
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From page 46...
... The FDA has published numerous guidance documents to clarify the considerations that should be taken into account by sponsors through the approval process, beginning with presubmissions. They also strongly encourage sponsors to initiate dialog with them at the earliest stages, so that the agency can provide feedback and suggestions on the anticipated designs before a study is initiated and data collection has begun.
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From page 47...
... For example, AIFA has signed contracts with pharmaceutical companies that set payment based on treatment effectiveness (performance-based risk sharing agreements) , with companies refunding costs if the medication fails, said Pani.
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From page 48...
... Health Care Systems Research Collaboratory,4 which has initiated 10 demonstration projects spanning 12 NIH institutes and centers to reduce the cost of clinical trials by capturing electronic health record data. Another network, the National Patient-Centered Clinical Research Network (PCORnet)
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From page 49...
... While several potential barriers exist to sharing clinical trial data, several participants acknowledged the many benefits. For example, sharing clinical trial data • "has great potential to accelerate scientific progress and ultimately improve public health by generating better evidence on the safety and effectiveness of therapies for patients; • increases patients' contributions to generalizable knowledge about human health by potentially facilitating additional findings beyond the original, prespecified clinical trial outcomes; • could provide a more comprehensive picture of the benefits and risks of an intervention and allow health care professionals and patients to make more informed decisions about clinical care; and • could potentially improve public health and patient outcomes, reduce the incidence of adverse effects from therapies, and de crease expenditures for medical interventions that are ineffective or less effective than alternatives" (IOM, 2015, pp.
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From page 50...
... With the passage of the Orphan Drug Act in 1983, the FDA recognized the need for a different regulatory framework around rare disease entities. Since then, the identification of specific genetic mutations that could predict prognosis or response to the therapy, as well as the emergence of the Internet and the digital revolution, have highlighted the need for a new regulatory framework appropriate for the 21st century, according to Karl Kieburtz.
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From page 51...
... Drug Product Registries Registries are an enormously useful source of information that could generate many hypotheses that might lead to randomized trials, according to Laughren. For example, a registry was required for the clozapine9 registration trials, which enabled generating data regarding the hazard curve for the potentially fatal adverse event agranulocytosis, in which the level of white blood cells called neutrophils drops to dangerous levels, causing suppression of the immune system (Alvir et al., 1993)
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From page 52...
... Califf commented that there are excellent models in the United States, such as one developed by the Society of Thoracic Surgeons, where data are checked and audited. They were designed that way because surgeons realized they would not be reimbursed unless they produced reliable outcome data.
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Key Terms

  • data sharing
  • accessed june
  • clinical trial
  • regulatory framework
  • sharing data

  • clinical practice
  • trial design
  • optimize trial
  • research networks
  • regulatory perspectives

  • data warehouses
  • design approaches
  • monitoring registries
  • improve public
  • sharing clinical

  • clinical data
  • universal data
  • increased data
  • collecting data
  • allow health

  • data regarding
  • integrating clinical
  • rarified clinical
  • prespecified clinical
  • integrated health

  • health record
  • permanent national
  • generating data
  • claims data
  • rigorous data


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