Biomedical Engineering Reference
In-Depth Information
Taking advantage of the opportunity to obtain “parallel scien-
tific advice” from both agencies can help to make the development
process more efficient and cost-effective by ensuring that all
planned investigation will be considered acceptable to support
applications in both the US and the EU. Clinical trials are very
costly, and discovering that the protocol of a completed clinical trial
is considered acceptable in only one market and not the other could
be a huge setback to a development plan. In the case study for
Ozurdex
®
, the same clinical studies were deemed acceptable to
support marketing approval in both the US and the EU, and this
allowed for a more streamlined, cost-effective drug development
and approval process than if different studies had been required for
each of these major markets.
Technical considerations that can help ensure a successful drug
development program include choosing the most clinically relevant
animal model(s) for preclinical testing, and designing all clinical
trials with great care. Some of the most important aspects of
clinical trial design include choosing the most appropriate control
group, choosing easily quantifiable and clinically relevant outcome
measures (particularly the primary endpoint), and the inclusion of
sufficient exploratory and subgroup analyses to help guide deci-
sions about how best to use the drug.
6 Conclusions
The increasing availability of safer and/or more effective medica-
tions helps drive ongoing improvements in patient care. The new
sustained-delivery implants offer particular promise for the treat-
ment of localized chronic diseases in general and chronic retinal
diseases in particular. The ability to bring these innovative new
drugs to market is dependent on several factors, including advances
in basic science, as well as well-designed drug development plans
and successful applications for marketing approval. The keys to the
successful launch of a new drug include identifying a promising new
drug candidate and then designing a drug development process
that takes the requirements for marketing approval into consider-
ation from the very beginning. This will motivate the appropriate
consultations with regulatory agencies in both the US and the EU,
and ensure that preclinical and clinical trial designs will be appro-
priate to support a successful application for marketing approval in
both regions. Ongoing efforts to harmonize the guidelines,
requirements, and document templates across regions are designed
to allow the drug development process to be more efficient and
cost-effective. The single most important determinant of a success-
ful drug launch—outside of the merits of the drug itself—is taking
full advantage of all of the resources and opportunities for commu-
nication with both the US FDA and the EMA.
