Clinical trial checklist	February 2009

The following are key factors to consider for any type of clinical study provided by
Neil Patel, Pharm.D, pharmaceutical R&D operations, PriceWaterhouseCoopers

NOTE: Factors were identified and reviewed as part of a project PWC undertook to inform site and patient recruitment strategies. These are not listed in any particular order, rather are factors that are considered in tandem.

Is the study assessing an acute disease state, as opposed to chronic disease state?
An indication with rapid onset where it is problematic to identify and line up patients in advance, i.e. myocardial infarction. "Heart attacks aren't planned or accurately predicted." Acute disease can also be defined as an exacerbation of an existing chronic condition, i.e., asthma exacerbation.

Chronic disease refers to illnesses that are prolonged, do not resolve spontaneously, and are rarely cured completely, i.e., coronary artery disease, allergic rhinitis, type 1 diabetes.

Does the study design call for a significant deviation from the standard of care?
The study treatment regimen varies significantly from the existing standard of care. Therapeutic area leaders and thought leaders can be utilized to assess the extent of deviation planned in the study from the existing standard of care. The question to ask yourself is what is currently the treatment algorithm for the particular indication/clinical endpoint you are studying.

If the design of your trial is different from the current treatment algorithm then you should answer yes. Standard of care is synonymous with the term "gold standard," i.e., the gold standard antihypertensive agent for diabetes is an ACE inhibitor.

Will the patient population come from the inpatient setting as opposed to the outpatient setting?
The indication/clinical endpoint under study is generally treated in an institutional setting, i.e., hospital, nursing home, assisted living home and requires overnight stays.

The outpatient setting is for patients that do not require hospital stay. Therapeutic area leaders and thought leaders can be utilized to identify the norm for treatment venues.

Will the study benefit significantly by using large academic medical centers (AMC)?
The trials will utilize university associated hospitals or care facilities owned or operated by universities.

Is the disease/clinical endpoint of the study managed by a general practitioner, as opposed to specialist?
The generalist is a physician such as internal medical family practitioner usually in the community setting.

The specialist is a physician who specializes in a therapeutic area, i.e., gastroenterologist, endocrinologist, cardiologist. The key question to answer is what type of physician makes the diagnosis and manages the particular patient. Therapeutic area leaders and thought leaders can be utilized to identify the norm for treatment venues.

Is the study considered complex?
When thinking about this criteria it is important to be very critical of your study design to designate the study as "complex." A complex study is one defined as meeting any one of the following criteria: 1) a long study (treatment duration in excess of one year); 2) requiring multiple visits in a relatively short period of time; or 3) lengthy visits or overnight stays.

Does the study have a requirement(s) for special tests/instruments?
The study has a need specialized or esoteric testing not normally found in a central lab or in the physician’s office.

Examples include requirement for the use of a spirometer, endoscopy, special assay or genotyping.

Therapeutic area leaders and thought leaders can be utilized to identify the norm for treatment venues and modes.

Is the disease prevalence high?
A study where the indication under investigation has a relatively large potential patient population.

Does the study have a requirement(s) for special patient populations?
Special populations are defined as studies requiring pediatric, geriatric patient populations, specific minority groups, etc.

This criteria does not address special patient populations such as Type 1 diabetic patients with foot ulcers

Will research naïve sites be required?
Research naïve sites are those in which the Principal Investigator has never conducted clinical research before or has very little experience with GCPs, ICH guidelines.

Protocol has significant potential scientific interest to investigator community
The treatment regimen under study has potential to answer an important clinical question or truly advance the standard of care in terms of patient endpoints.

Therapeutic area leaders and thought leaders can be utilized to assess the potential here.

Will this study face significant competition from other studies?
Similar studies targeting the same patient population are currently under way or being planned, which may lead to intense competition for sites and patients.

Will patient retention be a potential significant issue in this study?
A study where patient compliance and/or retention may be problematic, such as long-term (treatment duration exceeding one year), intrusive (invasive procedures such as bronchoscopy) or one requiring multiple inconvenient visits.

Will enrollment of patients/sites be impacted by the time of year?
A seasonal indication where the disease etiology is affected by the time of year.

Examples include allergic rhinitis which is more prevalent during the spring allergy season when pollen counts are high or seasonal affective disorder (SAD) which has been linked to low levels of daylight exposure during winter months.

Does the company have experience in this type of study?
Sponsor or CRO has executed similar study design(s) and/or indications in past five years.

Does this study have relatively strict inclusion/exclusion criteria?
Strict inclusion/exclusion is defined as multiple, broad exclusion criteria and/or therapeutically narrow inclusion criteria which minimizes the potential patient pool significantly. In other words strict inclusion/exclusion criteria significantly reduces a relatively large patient population to a very small patient population i.e., Including only Diabetics with HbA1C<7. Therapeutic area leaders and thought leaders can be utilized to assess the feasibility of the patient population required for the study.