Article | February 9, 2012

Avoiding A Failed NDA Submission Via A Third-Party Gatekeeper

Source: Life Science Leader

By Diane Mauriello and William Cairns

Companies specializing in the development, registration, and manufacture of pharmaceuticals will require no less than flawless management of data and tactical tasks to meet stringent FDA NDA (new drug application) submission requirements. In the supercharged business and highly regulated life sciences industry, your company must engage the best skills and most highly experienced professionals who offer end-to-end solutions to assure that your new products will be brought to market as rapidly as possible, well managed, and with minimal risk.

Your In-House Team Wears Too Many Hats
Your staff is responsible for numerous day-to-day global housekeeping activities. They do not have the time, focus, or experience to manage all aspects of a submission successfully. Submitting an NDA is the conclusion of 10 to 15 years of R&D, clinical development, and scientific discovery, and the cost is in the millions of dollars. Outsourcing these services to industry experts is the best alternative — not only because of the savings involved in every area, but because of the expert subcontractor’s in-depth understanding and day-to-day use of the procedures, protocols, and techniques required to reach a successful submission end point.

However, there are negatives and unseen risks. Many people do not do due diligence and spend enough time identifying the correct outsourced expertise and do not work on how to manage the resource. They are mainly concerned with signing a check. Unfortunately, when they get the deliverables, they start to see the problems and deficiencies.

You must consider whom you will engage, how to qualify a prospective expert, how to insert that resource/team into your company, how to keep project management centralized, how to monitor work and quality, and so on. The most successful method for avoiding a failed submission is the engagement of a third-party gatekeeper. This is the high-level project management individual or team who details a comprehensive road map in the form of a master project planner, which includes all components to the project. The gatekeeper serves as the communicator, the leader, the team effort overseer, sponsor, and project participant. They lead the team and manage the issues, timelines, and durations linked to all near-term or downstream activities but not the relationships. It is essential to monitor and QC (quality control) all actions. It is not enough to monitor without quality checks and controls firmly in place. All deliverables must be checked and timelines verified to determine how each element will impact another in the building of a submission “pyramid.” Overlook these steps, and you risk the potential impact of delays to your filing date and you will be faced with very costly daily delays.

Your in-house team can meet a perfect on-time schedule of deliverables, some of which are substandard. The gatekeeper opens the attachments, checks the summaries, verifies a data point at random, and asks for a preclinical report — or better — the appendices. This leader wants to see all FDA or regulatory and compliance correspondence, user-fee receipts, detailed (not summarized) marketing initiatives, and a sample label of a suggested product profile. They will organize the global teleconferences every week, without fail ask the pointed questions, and clearly assign responsibility and accountability — not just sign the agreement and write the check.

For the post-approval and postmarketing processes, more work will be required to monitor and assess the new drug in numerous areas including safety, benefit to the patient, risks, and efficacy, for a 12- to 36-month period.

Be Ready To File The First Time
Utilizing the gatekeeper for up-front development of a crystal-clear clinical/regulatory submission strategy and strict implementation of the master project planner leads to a successful submission end point. Effective medical writing is essential. It is a must that all data be reviewed for gaps, correctness, and consistency for compliance to CFR (code of federal regulations) and EMEA (European Medicines Agency). The gatekeeper approach will manage the project and your team to provide quality, clear, and pristine data and documentation with the end result being a fileable clinical submission the first time to the agency.

About The Author
Diane Mauriello, Ph.D., is the president of Dante Resources, a global company of clinical and submission specialists.

William Cairns is the founder and president of BCM Group, an international consultancy working with U.S. and non-U.S. manufacturers of pharmaceutical and medical technologies at the commercial level to create business growth and industry presence in North America.