Development Stage Company

Companies that have not yet received (or sought) marketing approval have a unique set of needs from a safety perspective.  Generally, development stage companies tend to spend less time specifically focusing on safety, most often considering it simply part of the development process and oftentimes unfairly expecting a development team to handle safety strategy and execution.

There have been many recent situations where safety issues have interfered with the approval of compounds.  New safety techniques provide ample opportunity to determine the safety profile of compounds and outline a comprehensive post-approval safety process that will clarify any outstanding safety questions.

There are two general situations faced by development stage companies.  In the first, the company is built around the entrepreneurial scientist and executes activities using internal resources.  The second is where the company is based upon a financial model and outsources many aspects of development to remain, "virtual".

Key safety needs can differ depending upon the situation:

• Entrepreneurial science company

  • Perform comprehensive safety analysis early in development (mechanism based, class or similar compounds, all non-clinical and clinical data from precursor compounds and target compound.

  • Build a clinical development program to answer as many outstanding safety “questions” as possible

  • Author documents and design processes to ensure most efficient and cost effective use of resources

    • Attention to details when writing protocols or Data and Safety Monitoring Board charters can make a significant difference in cost while maintaining compliance

  • Use everything learned during the above to design the postmarketing Risk Management Plan (required for European submission) and Risk Evaluation and Mitigation Strategy (if required, for US submission)

    • Harmonizing these activities (or at least the conceptual thinking) will avoid duplication of effort and improve relationships where partnerships are involved

• Virtual development company

  • Select vendor that will be willing and able to perform an analysis of the context for the compound

  • Ensure that the development program will “test” all (or most) of the safety hypothesis identified during the analysis

  • Author documents and design processes to ensure most efficient and cost effective use of resources

    • Attention to details when writing protocols or Data and Safety Monitoring Board charters can make a significant difference in cost while maintaining compliance

  • Use everything learned during the above to design the postmarketing Risk Management Plan (required for European submission) and Risk Evaluation and Mitigation Strategy (if required, for US submission)

    • Harmonizing these activities (or at least the conceptual thinking) will avoid duplication of effort and improve relationships where partnerships are involved

SSI Strategy can serve as a VIRTUAL SAFETY HEAD for these companies.  This service offers highly qualified safety physician support for such activities as designing data collection schemes, reviewing protocols, attending KOL and investigator meetings, analyzing data, and authoring any required summary documents.

This includes individual patient safety analyses, summary of safety issues, and integrated safety summaries.  We will also participate in FDA advisory committee meetings as necessary.

Companies that are marketing newly approved products have a unique set of safety needs.  These companies are quite dependent upon their first or second approved product and there is typically significant corporate focus on each serious safety report.

These companies frequently struggle with questions about what capabilities to build and which to obtain from vendors (outsource).  In most cases, either direction can work for a company, the most important thing is for management to decide and move forward with executing.  SSI Strategy can assist with the decision as well as supporting the company in executing whichever decision is taken.

Once a company decides that it wishes to create an internal safety function, several key decisions and activities follow:

• Will the safety database be in house or externally hosted

• Which safety database best meets the company needs

• What dashboards should be created to inform management of activities

• Written procedures need to be developed, for example:

  • Information intake and handling

  • Signal detection

  • Regulatory reporting

  • Document change control and archiving

  • Training

  • Inspection readiness

For companies that choose to outsource, there are also many key decisions that follow:

• Which is the most appropriate vendor – although not as complex a process as will be necessary for large global pharmaceutical companies, the decision about provider is critical for the small pharmaceutical company because they will typically place significant trust in this provider.

• What specific activities will the provider handle vs the company?

  • Phone center

  • Medical information services (customer facing)

  • Case processing

  • Medical review of individual case safety reports

  • Regulatory reporting

  • Signaling

  • Report preparation

  • What type of oversight will be in place for the provider

    • Interactions

    • Dashboards/metrics

    • How will the contract be structured to ensure incentives are aligned