Good Clinical Practice

The evolving drug development paradigm: A contract research organization perspective

The biopharmaceutical industry must meet new expectations for clinical research by re-thinking how to conduct trials, while focusing on four key areas: Good Clinical Practice (GCP) adherence, study design, quality data and patient safety. Increased focus on rare diseases and personalized medicine, for example, necessitates a shift to meet specific profiles in finding new medicines to address unmet needs for these areas. These factors require sponsors and clinical research organizations (CROs) to reevaluate how to design and conduct clinical research.

GCP

Good Clinical Practice (GCP) is an international quality standard provided by the International Conference on Harmonization that defines standards on the conduct of clinical trials, as well as the roles and responsibilities of research sponsors, investigators and monitors. By following GCP, investigators and clinical study teams can work to gather quality data and uphold patient safety standards. Those running clinical trials must focus on the right safety parameters, the efficacy and what is needed from a regulatory standpoint. Ensuring a trial adheres to GCP is important for regulatory review.

While there is some room for interpretation on a few GCPs, most are clear:

• Informed consent is a must
• Safety management is non-negotiable
• Quality source documentation must be generated and available
• Administration and control of the investigational product is paramount to confirming efficacy as well as safety to support study claims
• Protocol compliance is expected and a key area of focus for regulatory agencies

Study design

Clinical research organizations should partner with sponsors early on to promote trial and protocol optimization to ensure a trial has the best opportunity for success. If possible, discuss study design risk management with the regulatory authorities before the trial begins. All involved should ensure the study design regarding efficacy procedures are efficient and focused on what is needed to support market authorization.

Key questions to ask include:

• Are clinicians brought into the process early enough so they can support and conduct the trial before the protocol is finalized?
• Is there a connection with the clinical/medical team at the sponsor and clinical research organizations with all the investigators?
• Can a protocol be aligned with routine, accepted medical practice?
• How do you address protocol management when there is no standard of care in some rare disease areas?
• Focusing on the right areas before work begins helps to deliver an executable trial.

Quality data

To gather the highest quality data, expectations should be well defined and communicated to investigators and site staff prior to the trial. Entry criteria should be met before a patient is enrolled, without ambiguous documentation or imprecise proof of eligibility.

Clear instructions must outline how to document protocol related procedures, particularly around consent, safety data and efficacy parameters. Often, there can be a disconnect with investigators and site staff on what needs to be documented. This disconnect can be avoided with up front communications.

Trials should be designed to avoid the temptation to pull more data than necessary from a regulatory strategy and submission standpoint. Identifying the appropriate level of data required for the protocol is a key step in the design process.

Patient focus

To truly evolve the drug development paradigm, sponsors and clinical research organizations need to emphasize the ultimate role of the patient. In certain therapeutic areas, patient advocacy groups are serving as the voice for those who need to be heard. Perhaps we can bring those groups into the design of trials. Particularly for rare diseases, trials that face difficulty enrolling patients, all parties could benefit by hearing from patients and their advocates.

As personalized medicine and rare diseases gain focus, classical clinical trials could become a thing of the past. Across the biopharma industry we need to listen to the patients.

However we evolve the drug development paradigm, at the end of the day the end result of our work is a novel therapy that will benefit patients.