As a pharmacist with clinical experience and a scientist in a management position, I am in a unique position to see the disconnect between scientific, patient-related, medical, and operational components of a study. I know that identifying and understanding the perspective of key stakeholders throughout all stages, from concept to closeout, will improve the quality and efficacy of the trial.
Creating an environment where patients and data quality are the main focus of the study requires teams to work collaboratively and innovatively.
Getting Input Early During Protocol Development
It is essential to get patient input early and frequently during study protocol development. During this stage, it is also necessary to rely on feedback from the other stakeholders. This feedback can lead to a study design and decisions that will support:
- The highest quality possible for study conduct and clinical data
- Improved patient experiences
- An elegant study design that works to answer clear and concise questions relevant to the patients and disease outcomes
- Practical operation components
The question on what the study goals are and what information it will provide starts with the patients (including caregivers and health care provider), product, and the clinical team (which includes clinical sciences, clinical operations, medical and safety, statistics, data management, and regulatory).
Fine-Tuning The Various Aspects of the Study
There must be an amalgamation of the different team members for this to be effective, each with a specific emphasis. The clinical scientist’s focal point is on creating the hypothesis and designing a trial that supports the drug’s mechanism of action and the pathology of the disease.
The clinical operations must concentrate on the study administration to ensure that trials run smoothly and in accordance with the study protocol. The medical teams will focus on the clinical presentation of the disease and the safety of the study participants.
The data scientist and statisticians will guide power assumptions and oversee data quality and trends during the study design, operational conduct, and reporting.
Early and continued multidisciplinary discussion between stakeholders will lead to higher-quality studies, more efficient timelines, and predictable costs. Gathering patients’ feedback early on will improve patient recruitment. Retention in the study will reduce the need for protocol amendments.
Improving Recruitment for a Study
Gaining a better and more current understanding of the patient population via patient, caregiver and healthcare professional interviews can improve recruitment for a study. A proper understanding of the patient population can lead to a better grasp of the operational challenges and how to overcome them with tailored recruitment strategies.
It is essential that development of the inclusion criteria for studies be realistic. We should be open about challenges that may arise and adapt as the study evolves. It is also important to appreciate that the environment in which a new study is running, including approvals of new drugs, will impact the characteristics of the population.
The clinical team must make their best effort to anticipate who they need in the study. However, they must also recognize that they will make mistakes and must be willing to amend the inclusion criteria for their studies. To mitigate the impact on the quality of the study, the team must make significant efforts to resolve any challenges related to the inclusion criteria.
Drug developers must also create study budgets that can support tailored recruitment strategies, using all the tools at their disposal (e.g., social media, recruitment applications and platforms, television & radio).
Patient retention in a study will improve if we consider the patient experience early in the study and protocol development. While patients and their families commit to research to support finding therapies and finding cures, if the trial is too demanding, exhausting, and does not have supportive strategies for the patients and caregivers, dropout rates are likely to increase. Managing dropout rates is critical as they affect the study, data, and quality of the results.
The Work Continues After the Study Begins
It is crucial to appreciate that the study initiation does not mean the work is over. In fact, it has just started.
The different stakeholders need to work as a united front to oversee operations and how they impact the patients and the study data quality. This can be done blindly to confirm the assumptions built in the study.
With the advancement of technology supporting clinical development, stakeholders should strive to build seamless collaborative platforms. The best protocols and studies focus on addressing clear and concise hypotheses, are operationally feasible, and keep patients at the center of decision-making.
Groups that appreciate how critical it is to work within a united and collaborative framework will create the best chances for effective drugs to succeed and help patients live better and healthier lives.