PHOTOGRAPHY: GETTY IMAGES ILLUSTRATION: PAUL A. BELCI
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Few things unite the biotech industry more strongly than the desire for greater harmonization in regulatory and administrative
requirements between the European Union (EU) and United States. Harmonization of regulatory guidelines and development principles
through projects such as the International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals
for Human Use (ICH) is extremely valuable. But even with significant achievements such as the Common Technical Document (CTD),
major administrative differences remain and continue to frustrate.
As highlighted by the "Framework for Advancing Transatlantic Economic Integration between the European Union and the United
States of America," signed by Presidents Bush, Merkel, and Barroso in April 2007, the desire for greater harmonization has
support at the highest political level. Of course, the practicalities of transforming this goodwill into tangible solutions
for industry are extremely challenging, particularly when the key guiding principles for the proposal are that: No change
to legislation should be required, and the simplifications should maintain or increase current levels of public health protection.
In November 2007, the European Commission (EC) hosted the Transatlantic Administrative Simplification Workshop, which it co-chaired
with the FDA and organized with help from the European Medicines Agency (EMEA) and the Heads of the EU National Medicines
Agencies (HMA).
Administrative Simplification: International Proposals
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The Workshop provided transatlantic pharmaceutical regulatory partners with the opportunity to hear the pharmaceutical and
biotechnology industries' proposals for administrative simplification through transatlantic cooperation. More than 24 ideas
for simplification presented by industry from both sides of the Atlantic highlighted the scope for administrative simplification,
which were then categorized into "work done," "work in progress to be intensified," "proposals for careful consideration,"
and "visionary proposals involving possible legislative changes" (see Table 1). The more pertinent ideas for clinical research
follow.
Work done
The first achievement to date is the formal adoption of a common application form by the FDA and EMEA for sponsors seeking
orphan drug designation. The idea is to allow sponsors to apply simultaneously to both jurisdictions with one application
format, the impact of which should be comparable to the CTD for marketing applications. The format includes sections with
common information required by both the EMEA and FDA, and sections with region-specific information.
The need for a common application form was due to the fact that submitting differing application formats to the EMEA and FDA
represents a substantial burden for sponsors, particularly for small- and medium-size companies. Applying in both territories
at the same time should not only reduce resources spent and thus costs on the sponsor's side but also benefit patients with
rare diseases as a result of earlier designation. However, the first practical experience with the common form has shown that
there is still a need for further improvement in the form's design.
For example, the form used by the FDA is an electronic version with editable fields, which do not always allow for the entering
of EU-specific information such as phone numbers composed of more than 10 digits.In contrast, the EMEA form is a text version
that seems more user-friendly. Also, there are still too many fields that need to be completed with information specific to
the FDA and EMEA only, which not only makes the form longer than the previous EMEA one but it also implies that different
content for the FDA and EMEA has to be entered.
Despite these teething problems in finding an optimal format, the common orphan drug designation application form is a step
forward and provides an environment where the FDA and EMEA can share experiences, gain an understanding of the similarities
and differences in their designation of orphan drug status, and ultimately continue the process of administrative harmonization.
