Creating a Comprehensive Drug Development Strategy | Page 8

Figure 7.

Brief Overview of Clinical Development

OBJECTIVES

POPULATION

DESIGN

Clearly, a clinical development strategy should include detailed plans
about study designs, identification of investigators, statistical analyses,
and timelines. However, two important but often overlooked topics
are contingency planning and risk management.
It can be argued that successful drug development is all about

PHASE I

contingency planning. What if the API can only be made in small
Establish
pharmacokinetic
profile & dosing
regimens
Assess safety
& tolerability

batches? What if the formulation isn’t stable? What if regulatory

12 to 40 subjects
Healthy volunteers,
patients with
advanced disease
maybe used for
life-threatening
conditions

Open-label
Uncontrolled

PHASE II
PHASE III

Establish
long-term safety
Expand
indications

if trial data is inconclusive? The list of “what ifs” can easily grow to
happen. Spend less time on freak occurrences (e.g., the ship carrying
the API from China sinks) and more time on highly probable events

20 to 400
patients with the
targeted disease

Test special
populations
Prove safety
& efficacy

enrollment is slow? What if a competitor product launches first? What
an unmanageable size, so concentrate on items that are most likely to

Establish
proof-of-concept
Test safety in
affected patients

authorities don’t agree with your planned approach? What if trial

Randomized
Controlled

(if used for regulatory
submissions)

300 to 1,500
patients with
the disease
The size of the safety
population depends
on the seriousness
of the disease

(e.g., development timelines are lagging).

Contingency planning. “What if?”
Spend time planning highly probable events.

Randomized
Controlled
(either placebo
or standard of care)

Finally, consider your risk management plan (RMP) or risk evaluation
and mitigation strategy (REMS), which can be defined as “a set of
pharmacovigilance activities designed to identify, characterize, prevent,

Clinical Development
Clinical testing establishes that a drug is safe and effective for human
use. Many volumes have been written on clinical development, and
this paper presents only a brief overview of the objectives, population,
and study design associated with each phase (Figure 7).

or minimize risks related to the medicine; to assess the effectiveness
of those interventions; and to communicate those risks to patients and
health care providers.3” Put more simply, this type of document is meant
to ensure that the benefits of a medicine outweigh the risks. Creation
of a RMP/REMS is often a collaboration between clinical and regulatory
teams, and external input from experts and regulatory agencies is
typically needed.

clinipace.com

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