March 3, 2021
By: Gurudath Gurjal, freelance clinical research expert at Kolabtree, provides 3 tips to design effective clinical trials.
Clinical trials are the most efficient and primary technique researchers have at their disposal when trying to understand whether a drug, treatment or device is safe & effective in humans. Clinical research is just one part of the entire drug development process1 and clinical trials make up the bulk of the clinical research part.
Clinical trials are further divided into different phases, based on their objectives, design, complications, expected end results, etc. Well-designed clinical trials bring out the most authoritative and accepted knowledge for the betterment of mankind – be it newer drugs/devices or existing treatment options or even if it is for further exploration of a disease area.
Industry-sponsored clinical trials (regular phase trials) form just about 5-10% of global clinical trials. The remaining, i.e. over 90% of all clinical trials comes from individual researchers2. In my experience of over 15 years in the clinical trial industry, I have seen hundreds of clinical trials across phases, indications, geographies, and applications. One of the key differentiators for successful trials is the design of the trial itself.
If you have designed an effective clinical trial, you have covered significant ground in the process and you know the way forward. Therefore, designing your clinical trial in the most efficient way to optimize it to its fullest potential takes utmost importance. While industry-sponsored trials may have multiple teams that are dedicated for clinical trial designing and conduct, as an independent researcher3, you may not have that luxury.
As an independent researcher/medical practitioner, it is essential that you start with the 3 basic/obvious aspects for any clinical trial – Objective, Methodology & Outcome. However, to get the overall picture right, here are 3 TIPs to be considered to design effective clinical trials.
- T – Tools & Techniques
- I – Investments
- P – Plans & Procedures
Let’s look at each of them in detail.
- TOOLS & TECHNIQUES
This essentially deals with how data will operate in your trial and how you will manage it.
- Will you need specialized lab/diagnostic tests & tools for your trial? Are these validated?
- If yours is a device study or device-based study, you are already in this game!
- One of the first aspects to consider when designing your clinical trial is be sure of data collection & reporting. There are tons of tools for data collection, management, and reporting – and many of these are open-sourced and free to use.
- Will your trial data be paper-based or electronic? Paper-based tools may seem like the obvious choice for most independent researchers, but open-source electronic tools are available that can be customized for type & size – giving you great advantage in the long run.
- There are tools even for subject recruitment. Everything from informed consent (eICF) to source documents (eSource), case report forms (eCRF) to e-Diaries to trial master file (eTMF) is now available in electronic format. US FDA and EMA have adopted to electronic trial data for over a decade now.
- Depending on the enormity of the data/subjects in your trial and your expected outcome, what statistical tool will you be using? While SAS® is the preferred and most widely acknowledged, statisticians these days are adopting other tools too.
- Although this list can be exhaustive, the golden rule is to ensure that any tool you plan to use in your trial should comply with regulations & guidelines, must be validated and most importantly ‘help you in your trial’ – not create a barrier!
The most obvious and important aspect – something that you will have to be sure of even before you start designing your trial – is understanding what investments you need to make.
- Will your trial be self-funded, or do you have other resources – your university or department?
- Can you apply for grants by the government under various research schemes – NIH & NHS have literally hundreds of clinical programs that may cover your trial design?
- Have you considered approaching the industry for funding – even if not completely, almost every mid to large pharma organization has funds allocated for Investigator-Initiated Trials (IITs). If your trial is aligned with the clinical development plan of a product, this could be a great way to optimize – not just partially or fully sponsoring your trial, the pharma company could even allocate some of their teams to help in your trial design & conduct.
- Investments are not limited to funding alone – your trial team is also a critical investment! Clinical trials are always about teamwork. Even if you are the only investigator, you need at least a nurse/coordinator to take care of mundane & administrative tasks. Invest in the right people and the rewards will be phenomenal. Remember to take care of their EGOs if you are working with colleagues who are at the same level or higher than you!
- If you are desperately wading in unknown waters all alone, consider hiring a clinical trial consultant. Consider this consultant as an external coach/mentor who can guide you in almost all aspects of your trial – from design to conduct, data monitoring to reporting and may be even help in publishing in top journals. The investment is small, but the returns are high!
PLANS & PROCEDURES:
Failing to plan is planning to fail! Clinical trials are all about plans – subject recruitment plan, randomization plan, data collection plan, monitoring plan, etc. A clinical research consultant can help you with aspects of planning.
- Identify what regulations and guidelines are applicable to your trial – design your study to meet these regulations & guidelines for better acceptance while publishing.
- Timelines are important part of your trial plans – be realistic but avoid procrastination. Effectiveness of your trial is also time dependent.
- If your plans are designed well, they become excellent tools during trial conduct. Templates for various trial plans are freely available over the internet – do not fret, be smart.
- Finer details like subject-specific procedures must be elaborated in the trial protocol – design these pragmatically.
- Your protocol is the biggest trial plan in itself – do your research well before you start designing your trial! A freelance clinical trial protocol writer can help you with this aspect.
- Be innovative in designing your clinical trial4. Think outside the box and try to come up with novel study designs that meet the needs of the evolving world. If the last 10 months are any indication (who thought a brand-new vaccine would be approved in a few months?), it’s all about breaking barriers to achieve success!