In a regulated environment, regardless of the type of healthcare product development pursued (for example, of a drug, biologic or medical device), studies need to be conducted to demonstrate the healthcare product’s quality, safety and effectiveness/efficacy with respect to the proposed indication.

Although the exact data requirements may vary depending on the product design, the proposed indication and/or the targeted jurisdiction, studies that support submission applications must be carefully designed, conducted, analyzed and recorded  in compliance with relevant regulatory requirements and good industry practices (for example., Good Laboratory Practice [GLP], Good Manufacturing Practice [GMP], Good Clinical Practice [GCP], and Good Documentation Practices [GDP]).

Clinical studies and the regulatory submission

Studies to be included in regulatory submissions should follow a written protocol that outlines the purpose and details of how the study will be conducted and the justification for the approach taken. Record the study results and data analysis in a study report. These documents should be appropriately reviewed and approved by relevant personnel prior to finalization of the document. Any amendments introduced after the document sign-off should be properly evaluated, documented and approved.1

The types of studies to be included in a regulatory submission may include the following:

  • Validation studies (for example, process validation, sterilization validation)
  • Stability studies
  • Bench testing (for example, mechanical, physical or chemical testing)
  • Biocompatibility studies
  • Non-clinial studies (for example, acute toxicity, chronic toxicity, reproductive toxicity, genotoxicity, carcinogenicity)
  • Pharmacokinetic and pharmacodynamic studies
  • Biopharmaceutical studies (for example, bioequivalence studies)
  • Clinical studies (for example, feasibility, safety or efficacy studies)

Study protocol and study report: Companion documents

Consider developing the template of your study report while building the study protocol. By having the end in mind, you can plan and design the study (for example, the duration, patient population, sample size, statistical analysis plan) so that specific data and results (for example, the meaningful clinical end point) that answer particular questions (for example, whether healthcare product A effectively prevents disease X) can be collected, anaylzed and reported. By clearly defining all of these elements in the protocol, the study can be properly conducted and the results will avoid censure related to bias or data manipulation. The study protocol is crucial to establishing the foundation for the results and conclusions of individual trials―and ultimately, those reached in the licensing application. 2

Should the study protocol or the finalized study report need to be changed, generate an amendment that includes:

  • A description of the change
  • The rationale to support the change
  • An impact analysis (if relevant)
  • Identification of the affected documents and their sections
  • Signature(s) of the individual(s) who give approval
  • Date of the approval of the change
  • Date of when the change takes effect

In the case of a clinical study, if a study application has been approved under Investigational New Drug (IND), Investigational Device Exemption (IDE), Clinical Trial Application (CTA), or Investigational Testing Application (ITA), any significant amendment (that is, changes that significantly affect the safety of the trial subjects, or quality of the investigational product) should also be submitted to the corresponding ethics board(s) and regulatory agency(ies) for approval prior to its implementation.3 Make sure to review all the submission requirements for amendments relating to the conduct of clinical trials in all relevant jurisdictions.

Tips: The use of protocol and report templates allows upfront research to be conducted and recorded  in compliance with regulatory requirements and guidance documents. When compiling information, organize it into sections and subsections, with logical groupings of related data clearly identified through the use of headings.1 

Disclaimer

The information presented in these articles is intended to outline the general processes, principles and concepts of the healthcare product development lifecycle. Since regulatory requirements are ever-changing, it is current only as of the date of publication and not intended to provide detailed instructions for product development. Every healthcare product is unique and therefore so is its associated product development lifecycle. Specific advice should be sought from a qualified healthcare or other appropriate professional.

Published: October 17, 2012

References

  1. United States Code. (2011, April 21). Title 21, Section 820.40(b), Quality System Regulation: Document controls. Retrieved May 30, 2012, from http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=820.40.
  2. Linberg, S.E. & Yurko-Mauro, K.A. Chapter 17: The clinical protocol. In S.E. Linberg (Ed.) Expediting drug and biologics development: A strategic approach. (n.d.) Watham, MA: PAREXEL International, pp. 311-35.
  3. International Conference on Harmonisation. (1996, June). E6(R1): Good clinical practice. Retrieved July 12, 2012, from http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E6_R1/Step4/E6_R1__Guideline.pdf.