There are three things you need for successful research:
- A quality Protocol
- An engaged Clinician
- Motivated Participants/Patients
You may need others (and that will vary from project to project), but if you nail these three things, your research project is almost certain to succeed.
A Quality Protocol
The quality of your protocol will dictate the quality of your research. The research protocol contains all the information necessary to undertake the project. The Ethics Committee (HREC) reviews the protocol and, once approved, it cannot be deviated from without a formal amendment.
The recommended contents of a protocol are set out in section 6 of Good Clinical Practice1. However, it is possible to meet all the regulatory requirements of a protocol yet still have a poor quality protocol.
What makes a quality protocol?
The difference between a poor quality and a good quality protocol is somewhat subjective and the following list is based on my professional opinion and personal experience. A good quality protocol:
- Has a clear goal (primary endpoint) – one that is not confused or overwhelmed by secondary, tertiary or exploratory endpoints.
- The primary endpoint answers a valid and useful question that will inform clinical practice.
- Assessments and data collection inform the endpoints in a meaningful and measurable way.
- The population under study is clearly defined and reflective of real life.
- The assessments are not onerous for the patient (comparative to the disease under study and current standard of care)
An Engaged Clinician
One of the reasons I enjoy working on Investigator-Initiated Studies is that the clinicians are usually very engaged. The clinician usually writes, or helps write, the protocol, and, therefore, believes in the question the research aims to answer.
The types of research that result in an engaged physician include (again, from my own experience) research where:
- It is an area of unmet need (eg there are no treatment options available for this patient population; the disease under study is not well understood, etc.)
- Provides access to a therapy not otherwise available to the population under study
- The “standard of care” therapy is inferior (eg is not effective, has undesirable side effects, is complex to administer, etc.)
The reason for a motivated participant will vary depending on the population under study. For example, the reason for a healthy volunteer to be involved in research will differ significantly from a cancer patient with no treatment options left.
There are a few suggestions on how to ensure you have a motivated patient population:
- Consult your target participant population when writing the protocol (see this article here on reasons why).
- Try to be as flexible as possible with assessment windows to encourage patients to enrol and to stay in the study.
- Understand that participants are doing researchers a favour by being involved in the research and treat them accordingly.
So you see research is as simple as 1, 2, 3. Well, maybe not simple. But if you get the ingredients right, you will have a successful outcome.
1 Integrated Addendum to ICH E6 (R1): Guideline for Good Clinical Practice E6 (R2) dated 09 November 2016 (“ICH-GCP”)