Overview of Basic Research

What is research?

Simply put, research is the scientific process of gathering, collecting and organizing information for the purposes of learning and sharing knowledge. One way doctors and researchers gain and share knowledge is by performing research studies.

What types of research studies are done at HCWP?

The types of research studies at HCWP range from non-drug studies to clinical (new drug) studies:

Non-drug studies

This means that no drug is given for the purposes of the study. These studies usually “watch and see” what happened/or happens to patients throughout time. Then doctors and scientists analyze the information. Typically when patients consent to participate, doctors review health records, lab results, treatment records and responses to treatments (like FVII, FVIII, FIX or humate P).

Observational studies: These studies enroll subjects and then begin ongoing data and information collection.

  • Example: American Thrombosis and Hemostasis Network database which will enroll thousands of patients nationwide and plans to analyze information like severity of hemophilia, treatments and lab results over time.

Registries and repositories: These studies enroll patients for a variety of purposes, which can include any of the following: (1) Collecting past and current health information for analysis (2) Determining interest or eligibility in other studies (3) Collecting and storing blood samples for future testing.

  • Example: HCWP Research Registry & Repository allows patient health information, extra blood (left over from clinical blood draws) to be analyzed. It also permits staff to contact patients to see if they are interested in certain studies.

Clinical Drug Studies: Research studies can be developed by doctors, scientists and other professionals or by pharmaceutical companies for testing a newly developed investigational drug. These studies are categorized by phase I, phase II, phase III and phase IV. All drugs in the United States go through phases I through III before they are approved by the FDA. The FDA reviews all study information and votes on whether or not to approve the drug. Only if the drug is approved, can it be prescribed by doctors in a specified clinical setting.

  • Phase I studies: Phase I studies are first in human studies. Once a newly developed investigational drug is tested in a lab and found to be safe in animals, it is permitted for human testing. Phase I studies enroll a small number of patients (usually fewer than 15) to determine if the study drug is safe for humans.
  • Phase II studies: Phase II studies continue to look at safety and if the drug works in humans. Phase II studies enroll more patients and usually require repeated doses of study drug.
  • Phase III studies: Phase III studies enroll even more patients so doctors and scientists can statistically evaluate safety, efficacy (the drug does what it is supposed to do) and side effects, in comparison with a standard therapy. After all the information is collected, the data are presented to the FDA for review.
  • Phase IV studies: This phase of research occurs after the study drug is approved by the FDA and becomes available for patients (the drug is now available to be prescribed by doctors). This phase monitors effectiveness in the general population and collects adverse events over time.