If the researchers aim to show whether a specific treatment achieves the
desired benefits, they may reasonably choose to conduct a randomized
clinical trial to ensure that uncontrolled variables that influence outcomes,
such as age or drug interactions, do not confound the study. However,
observational studies may be needed to determine whether the results of
randomized clinical trials that involved only a few thousand patients can
be generalized to the patient population at large and to realistic treatment
situations rather than carefully controlled ones. Furthermore, observational
research based on medical records will often be sufficient to determine a
treatment’s adverse effects. 80
Indeed, as a domestic example, an FDA advisory group on diabetes
recommended approval of a continuous-glucose-monitoring device that
relied on simulation data based on a small set of actual clinical data. 81 While
several members of the advisory group raised concerns about the use of this
type of data, an FDA official suggested that, if approved, there could be a
requirement to conduct post-market surveillance of the device to ensure
patient safety and compliance with recommended guidelines. 82 Thus, this
reliance on data—both as part of the approval process and to supplement
surveillance efforts once a device has gone to market—could be precedential
in future drug approvals.
Through efforts dating back to 1980, countries have attempted to find
ways to “strengthen collaboration and exchange information” through
international organizations, such as the International Conference of Drug
Regulatory Authorities, the World Health Organization, and regional
negotiations. 83 The International Conference on Harmonization of Technical
Requirements for Registration of Pharmaceuticals for Human Use (ICH) has
developed a global standard “for the design, conduct, recording, and
reporting of trials.” 84 Because ICH standards are guidelines, not regulations, 85
they allow for differences between countries as to how this standard is
applied. 86 Often, these differences reflect the result of cultural norms and
80. Id. at 507.
81. Miriam Tucker, FDA Panel Supports Dexcom CGM for Insulin Dosing in Diabetes,
MEDSCAPE (July 22, 2016), http://www.medscape.com/viewarticle/866492.
83. Kiichiro Tsutani et al., Harmonizing East Asia: The Evolution of DIA Japan and Its
Impact on East Asia 43 DRUG INFO. J. 325, 325–26 (2009); see also Li, supra note 66, at 3–4
(discussing the development and efforts of the Multi-Regional Clinical Trials Center at
Harvard University to encourage uniform standards for multi-national clinical trials);
Françoise Augier de Crémiers, The Birth of the ICH E3 and How it Led to the CTD, 3 GLOBAL
FORUM 19, 19–20 (April 2011).
84. Kuhnert, supra note 77, at 17.
85. Id. (“The guidelines are guidelines and not regulations; thus, they are intended to be
used in combination with regional requirements”).
86. Bollyky, supra note 75, at 721; see also Kelly Davis, Biopharmaceutical