My Dad – The Drug Maker

I am pleased to introduce to you, one of our MLC, LLC team members, Lawrence Yarmaloff!  Larry is a veteran of 23 years in the clinical research industry.  As a consultant for the past 13 years, he provides quality assurance and clinical operations management support to small-midsize pharmaceutical and medical device companies.  He holds professional certifications from the Society of Quality Assurance and the Association of Clinical Research Professionals.

Go for it Larry!

“My dad makes drugs,” explained my 10-year-old son when chatting with his buddies about their fathers’ professions.

I guess that my job as a Good Clinical Practice Quality Assurance and Clinical Research Operations Consultant is not easy for a fifth grader to articulate.

At least he understands that I play a role in the drug development process.  As a younger lad, he equated the profundity of my job with the number of points he marked on his wall map of places to which I have traveled.

Some of his notable inquiries, my attempts at responses, and some extra credit facts:

“What exactly do you do when you travel?”

I explained that I need to check that doctors and drug companies “follow the rules” when studying new drugs.

Good Clinical Practice (GCP), developed and adopted by the International Conference on Harmonisation (www.ich.org/), delineates a set of international standards under which clinical research is to be conducted.  The FDA has long established regulations for the investigations of new drugs, which invoke the principles of GCP.  For stimulating bed time reading, download 21 CFR Parts 50, 54, 56, 312, and 812 and ICH-E6 to your tablet.

The Quality Assurance audits that I perform during my excursions across the US and abroad provide just one of the “planned and systematic actions that are established (by ICH and FDA) to ensure that a trial is performed and the data are generated, documented, and reported in compliance (with GCP) and applicable regulatory requirements.”

I said to my son, which I hope resonated with him, that to do these things well I need to read “a lot.”  A lot of records, documents, and procedures to assess if the information collected from studies are reported correctly and completely.  Just like what parents expect with their kids, we expect that people that do research tell the truth about what happened during studies, good, bad, or otherwise, and how they did or could improve their “research behavior”.

“How do you make sure drugs work?”

I explained to him that we need to use the same type of approach that he uses in science class.

GCP requires that we implement scientifically driven protocols that “describe the objectives, design, methodology, statistical considerations, and organization of a trial.”  I told him that we conduct a lot of studies to understand what drugs do in the body, to determine if a product helps to make sick people feel better, and to make sure that drugs do not hurt people or make them sicker.

“How long does it take to make new drugs?”

I told him that it takes years.  “But how many years?” he queried.  I provided the standard “it depends” response.

An October 2013 report in the Journal of the American Medical Association asserted that the average approval time for clinical drug testing and approval is 7.5 years and 5.1 that for “expedited drugs “(i.e., for unmet medical needs).  FDA commissioner, Dr. Margaret Hamburg, indicated last month (at the Pennsylvania Bio’s Life Sciences Future Conference) that since taking office in 2009, the FDA has made great progress in accelerating the drug approval process, and admitted that more needs to be done to continue this trend.

I did not delve with my son into the industry buzz about risk mitigation, streamlining clinical trials, and encouragement of industry/regulatory agency collaborations as modalities to accelerate the marketing approval of new drugs.  Nor did I dissect for him prevalent positions that less can be more, less can just be less, and that industry and regulatory reforms cannot abrogate our ethical obligations to protect consumers.  I will save those discussions for at least the eighth grade.

“Does it cost a lot of money to make new drugs?”

I told my son that it can cost millions of dollars.  “But how much money?” he asked.  Yet again, I provided the standard “it depends” response.

The November 2014 edition of the Boston Business Journal cited a recent study by the Tufts Center for the Study of Drug Development that the cost of new drug development is approaching $2.6 billion.  The study also asserted that the cost has increased 145% since 2003.

Why so much?  The Tufts study attributes higher R&D costs and a low ratio of drug candidates to approved drugs (about 8.5:1).

When I have the discussion about risk mitigations with my son, I can also pontificate about the politics and special interests that can influence the economics of drug development.   This is assuming that the interests of early teenagehood do not promote apathy of what his dad does to pay the mortgage.

“Why don’t you bring back gifts for us anymore when you travel?”

That’s easy.  “Because I am saving for the car that I will buy you when you turn 17.”  Yeah right…

• Rules are the rules – FDA regulations are law, and FDA expects that ICH guidelines are followed. Be honest about any deviations and non-compliances when they happen, and develop and document a corrective and preventative action plan to prevent future problems.
• The old adage is true – if it’s not documented, it did not happen. QA auditors and FDA inspectors will assess documentation based on ALCOA principles (attributable, legible, contemporaneous, original, and accurate).
• Don’t set the precedent of buying gifts for your kids when you travel.