The Wolf at the Door: An Inspection Readiness Crisis Case Study

How LMK’s TMF Consultants inspired a TMF leader to look beyond the wolf at the door and achieve TMF success with meaningful TMF metrics.

TMF leaders face a growing challenge—a crisis of inspection readiness. Despite rapid advancements in eClinical and eTMF technology, GCP inspection findings continue to rise, a product of increasing clinical trial complexity and regulatory expectations. The consequences of the looming inspection readiness crisis are immense. They directly threaten patient access to novel, lifesaving treatments by delaying, or even terminating, the most promising clinical development programs. As one TMF manager discovered, however, the best antidote to the paralyzing fear of inspections may be to redefine how you measure TMF success.   

The Challenge: A 483 Has Teeth

I remember the crushing sense of nausea that came with receiving the 483. It was a sensation that had been building since the murmurs of an unannounced inspection had become more than just an unsettling rumor.

I knew the outcome of this inspection was not going to be good. The TMF to be inspected, I heard, was in rough shape. The faces around me were vacant, drawn, or in muted panic. It was like there was a wolf barking the door—but we had no choice but to let the wolf inside.

For those who are blissfully unaware, FDA Form 483, informally called a 483, is a form issued to an organization’s “management at the conclusion of an inspection when an investigator(s) has observed any conditions that in their judgment may constitute violations of the Food Drug and Cosmetic (FD&C) Act and related Acts.”[1]

Lists of the 483s from each calendar year are located on the FDA’s website. They are issued under many programs according to the diverse array of products FDA is tasked with regulating, but they all have one thing in common: somebody was violating the regulations.

The implications of a 483, both professionally and personally, are terrifying. Many industry articles cite the cost of a ‘moderately bad’ 483 as around $250,000. Follow a 483 to its conclusion, however, and the costs can quickly spiral out of control.

Given the high stakes, fear and anxiety in response to a 483 are natural. The harm done by a 483 can be enough to end a study, cancel a clinical program, or bankrupt a company. In extreme cases of fraud or criminal intent, fines or imprisonment could even follow.

Consider Emergent BioSolutions’ COVID-19 vaccine 483. The company was required to dispose of a batch of 15 million doses of a highly anticipated vaccine in the middle of the COVID pandemic. A $628 million deal between the company and the U.S. government was risked. Extrapolating further, the incalculable costs of delayed vaccinations, damaged public reputation, reduced market standing, and incurred organizational distraction were likely orders of magnitude larger.

My 483, fortunately, was of the moderately bad variety. While the inspected TMF was not my direct responsibility, I was part of the clinical documentation team, the inspection response team, and the resulting clean-up crew. Sure, it wasn’t directly my fault, but since we were a small firm, I still had plenty to lose from the 483, as did everyone else in my company.

The 483 we received, as I alluded to, was nothing unheard of. It revolved around the failure of one challenging study’s TMF. While I will limit the details for anonymity, the findings were squarely in line with the most common TMF issues:

  • Documents were late or completely missing.
  • Documents were stored in non-TMF locations.
  • There was no real TMF plan.
  • TMF indexing was poor.
  • TMF QC processes were incomplete.

Again there’s no real mystery or deep complexity to these inspection findings. They reflect a breakdown of the three elements of TMF health: timeliness, correctness, and completeness.

As someone who has received a clinical trial documentation-related 483, I am in good company. TMF inspections are producing 483s at an increasing rate.

A recent study conducted by the FDA and European Medicines Agency (EMA) showed great alignment between the GCP inspection deficiencies between the two agencies, especially regarding the high incidence of records and essential document related findings.

The study identified investigators, sponsors, and CROs that had inspections common to FDA and EMA. Of the CRO inspections, for the documentation deficiency area, all 23 inspections had findings. Despite operating on different continents, both agencies identified documentation findings at 16 of the 23 CRO/Sponsor sites inspected by both agencies (a 70% concordance rate between the two regulatory bodies). The study also found that about 33% of FDA Sponsor/CRO inspection findings in the dataset were related to documentation, while 45% of EMA’s Sponsor/CRO findings were related to documentation.

The purpose of pointing out the high incidence of TMF inspection findings across the regulated world isn’t to escape a feeling of personal responsibility (although it does help), rather it is to bring awareness to an ongoing crisis of inspection readiness that can’t be blamed solely on bad actors or isolated behavior. The grave consequences of receiving a 483 alone aren’t enough to provoke change. Inspection readiness is not improving, and perhaps it is even declining. Worst of all, this crisis of inspection readiness flies in the face of the digital transformation promised by eTMF and other eClinical technology. These 483s are a wasted opportunity and a brightly lit warning sign that the future of clinical operations is not turning out how we imagined.

But how do we move forward? I can’t say what’s right for everyone. But I know how I moved past my 483, and it wasn’t by wallowing in the fear of the next inspection.

The Solution: Metrics Clear the Path to TMF Success

Did you know that you only have 15 business days to respond to a 483? FDA encourages firms to respond to the 483 in writing, “with a corrective action plan and supporting documents within only 15 business days of the issuance”[2].

Three weeks isn’t much time to break down a 483 into its elements, identify a root cause, write a corrective action plan, implement changes, and document outcomes. Of course, many firms will ask for an extension to respond—which is fine. But from my point of view, however, the extension request itself must show serious due diligence and a concrete set of planned actions to return to compliance. A weak response letter regularly provokes more severe consequences from the agency.

 

When everything is going wrong all at once, it can be difficult to slow down enough to genuinely understand a problem. Providing perspective was the main service LMK’s TMF Consultants provided.

I first heard of LMK Clinical Research Consulting when I heard consultants were hired to write our TMF 483 CAPA just a few days after the 483 was received. Before meeting with these consultants, I suppose I expected a certain level of complexity. I thought, clearly, they must have the secret tools and strategies we don’t.

By the end of the first meeting, however, I was shocked.

LMK’s Consultants didn’t propose anything I hadn’t heard of before. They repeated the same language of timeliness, correctness, and completeness that I had heard at every eClinical conference I had ever attended. They identified the same shortcomings as the inspector had and assigned us to the same inspection readiness goals as the 483.

The main difference was the structure that LMK’s TMF Consultants intended to apply to our challenges. Every step forward towards writing the response letter and implementing the CAPA would be driven by the data: LMK had draft TMF plans ready to customize and implement, training resources already prepared that could be tailored to our processes, but most importantly LMK had clear TMF metrics. Now, instead of one insurmountable problem, we had a scalable system to measure incremental improvement. It was a clear path to TMF success, and the lifeline we desperately needed.

The Implementation: Meaningful Metrics Make the Difference

It’s often been said that you can’t improve what you don’t measure. I now wholeheartedly agree.

But what is a TMF metric? A TMF metric is a quantifiable measure that is used to track, compare, and assess performance or processes towards TMF health. Metrics are an essential part of the cycle of TMF quality control, that continually verifies the condition of the TMF against preordained expectations.

Before the 483, we didn’t really use any TMF metrics. Our TMF filing latency was simply too high for TMF metrics to be effective. After all who needs TMF metrics that are immediately obsolete? We were constantly working to catch the TMF up to trial milestones that had already occurred. In theory, our relatively simple TMF quality issues should have been easy to address in 15 days, but in practice they were symptomatic of a root cause stemming from poor TMF QC processes and lack of oversight.  We had good intentions, but even with the best of intentions, we were overwhelmed, overworked, and disorganized.

In the tense planning meetings that followed the inspection findings, it was easily demonstrated that our TMF quality issues had reached a breaking point long before our first TMF 483 was issued.

LMK’s TMF Consultants noted that in the months leading up to the 483 there was especially high turnover in the TMF group. This led to the loss of institutional knowledge and the hiring of inexperienced TMF professionals who were not supported to succeed. Beyond the high turnover, our cycle of TMF QC was broken. Certainly, we had a TMF plan and document index, but it was generic across all clinical trials. I know we felt we didn’t have the time, communication, or availability of experienced TMF professionals necessary to create and maintain a bespoke plan for each TMF. Of course, in hindsight, it should have been a priority.

Without a true TMF plan and index, our individual contributors did not know which documents were expected to be filed in their TMFs. They did their best and were always locating, collecting, and filing documents, but QC never went further than a basic document inventory. They were not empowered to take corrective action when problems were identified. There were no clear processes to fix mistakes. As a result, a small trickle of TMF quality issues grew into a deluge as TMF expectations became divorced from TMF reality.

The process of drafting the CAPA was trying. There were long days and late nights. Through them all, LMK’s TMF Consultants stressed that TMF metrics were our best (and only) means to turn things around in time. TMF metrics, they underscored, were a non-negotiable part of developing an acceptable provisional CAPA. They emphasized that the cornerstone of any CAPA isn’t the identification or correction of an issue—FDA has already identified the issue through the 483 and expects the noncompliance to cease immediately. What FDA truly desires to understand is the working of your process to prevent the issue from happening again and the data you plan to present to verify beyond doubt that your fix is working.

The new TMF metrics we created were based on the elements of TMF health that had all but slipped out of our control: timeliness, correctness, and completeness. These metrics, consistent with my early observations about LMK, were simple but robust. They were rooted in industry-standard objectives like low QC finding rates, high completeness percentage, and low document filing latency. What set LMK’s metrics apart, however, was the degree to which we were expected to understand the goals and supporting processes behind our metrics—even if that meant greatly customizing a ‘standard’ TMF metric to meet our unique needs.

I recall being especially challenged by one of LMK’s consultant’s imperative to directly link TMF performance metrics to staff allocation, performance appraisals, training, and hiring. I was caught off guard by the idea that TMF metrics could be linked with concrete business goals outside of the narrow scope of TMF operations. I wasn’t comfortable thinking about the TMF in such a multidisciplinary way.

The Outcome: Lead with Confidence, Not Fear

Of all the indirect costs of a 483, I believe the greatest cost is the most difficult to measure. Beyond the business process interruption, lost money, and wasted time, the fear a 483 produces is crushing to any team. Faced with inspection findings, there are no easy solutions. Relationships are strained, resignations are tendered, and confidence is shaken. The pursuit of excellence becomes replaced with the fear of failing to address the very real and urgent deficiencies identified by the Agency.

That’s why I’m so grateful for LMK’s TMF Consultants. They taught me, my team, and my old company to rely on data as an antidote to all the uncertainty.

As I began to rethink my narrow preconceptions of TMF metrics, I was aided by the metrics dashboard that LMK had implemented using our existing eTMF reporting features to track our progress. LMK’s consultants, my organization’s TMF managers, and our TMF associates met each day to develop our 15-day response. Those 15 business days flew by in an instant, but by the time we submitted our provisional CAPA, we had already shown real progress towards TMF health.

It was what happened in the months that followed, however, that astounded me. The metrics we thought would be our undoing allowed us to look beyond our immediate fear and liberated us to work together in ways we never had previously considered. With LMK’s metrics, our TMF specialists were freed from the fear of underperformance through clear performance standards that built up their confidence. Our CRAs were freed from the fear of looming backlogs by clear visibility into their sites’ TMF completeness. TMF leaders, like me, were able to deploy resources, or even demonstrate the need for hiring new faces, by citing TMF completeness, quality, and timeliness data as we sought executive approval. Many of the unnecessary administrative tasks we assigned ourselves fell away as our new metrics allowed us to focus on the work that truly advanced our shared goals. Our metrics, which redefined our definition of TMF health, had also transformed our ability to achieve TMF success.

While I moved on from that team years ago, I’m happy to report that our CAPA was successfully closed, and no Warning Letter or other negative proceedings were issued. From what I hear, those that stayed behind have had several successful inspections since then. I’ve been told many of the processes and tools in place today have their roots in the tools and templates LMK first shared with us those many years ago.

In my own professional journey, I’ve also retained many of LMK’s best practices and strategies for use with my new teams. I continue to be astonished by how a few simple TMF metrics implemented early on in a clinical trial’s lifecycle can shift the TMF from a stressful burden to a powerful prospective trial management tool.

This is not to say I don’t still face TMF troubles. Each passing year TMF expectations grow larger and inspection readiness becomes more challenging. But even after a particularly hard day, I still seek to mentor those struggling on my team using the lessons I learned from that trying time in my career.

I’m not shy about sharing the story of my 483 and the terror of hearing the wolf howling at the door, especially with inexperienced TMF professionals. The path to success in any industry requires finding (or rebuilding) the courage to attempt great new things. Sure, in the face of a serious setback like a 483, confidence can be in short supply, but its foundation lies just beneath the rubble. You can take a breath, focus on your core talents, and take stock of all the ways you’ve grown since your first day on the job.

Metrics, and the insights and knowledge they confer, are an oft overlooked superpower. They can break apart a mountain pebble by pebble or unwind the worst of 483s into discrete deliverables that are simple, manageable, and scalable. They are the cure for fear and uncertainty.  If you can measure your own performance, your own knowledge, and your own personal growth, there’s always a quantifiable path to believing in yourself again.

 

[1] FDA Form 483 Frequently Asked Questions | FDA

[2] What to Expect after an Inspection: 483s, Responses and Beyond – 12/14/2022 | FDA