[HN Gopher] Why are clinical trials so expensive? Tales from the...
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       Why are clinical trials so expensive? Tales from the beast's belly
        
       Author : wawayanda
       Score  : 156 points
       Date   : 2022-11-10 12:00 UTC (11 hours ago)
        
 (HTM) web link (milkyeggs.com)
 (TXT) w3m dump (milkyeggs.com)
        
       | orzig wrote:
       | This is incredibly important if you want to understand
       | healthcare. Every little thing is expensive in a way that is hard
       | to imagine before you get into the field.
        
       | seidleroni wrote:
       | As someone working at a company that has run several clinical
       | trials, this really rings true, especially the segment on
       | language translations.
        
       | CharlieDigital wrote:
       | I worked as Director of Engineering at a company for 10 years
       | that built a SaaS for clinical trial document management.
       | 
       | There is a weird perversion of incentives in the industry driven
       | under the guise of "quality" and "safety" that from a software
       | engineer's perspective, looked pretty bad to me because of how
       | manual and error prone the process is.
       | 
       | The problem is that the people who have the knowledge aren't
       | incentived to make it more efficient or cost effective; the
       | purpose of many of these folks is to simply extract rent from the
       | system and blame regulations and "patient safety" concerns.
       | 
       | At an MES startup, one of the industry stalwarts (starts with V)
       | was more or less using mafia tactics to force the startup to use
       | their software or they would voice no confidence with pharma
       | companies where big V was already entrenched.
       | 
       | One of my passion projects would be to build an open-core
       | clinical trial document management system with a focus on
       | automation and templatization of common processes and workflows.
       | It's insane how much waste this system has.
        
       | protomyth wrote:
       | I worked on one in the 90's that was $50,000 a day for three
       | years. The drug company, assuming the trial was successful, had
       | about 4 years to make all its money back before the patent
       | expired. Testing blood, urine, and stool for multiple things is
       | expensive.
       | 
       | Intellectually, I can understand the need for control groups, but
       | I still think it's immoral. When you stare at a spreadsheet and
       | see 70% of the control group is dead because some random number
       | generator sorted them there like hell's own sorting hat, and the
       | 98% of the people getting the drug are alive, you have no
       | business talking about statistics. That graph will haunt me til
       | my dying day.
        
         | Slaminerag wrote:
         | It's possible to stop a trial because the new drug is much more
         | effective than the old drug. It's also possible to stop a trial
         | because the new drug's significantly worse than the old. We've
         | done both. Also, I hope that no one ever tells you that you
         | have to do a drug schema change because "too many kids are
         | dying."
        
         | s1artibartfast wrote:
         | You didn't kill 70% of the control group, you saved 98% of the
         | experimental group.
         | 
         | Also, with the appropriate trial design, you can stop the
         | control and transfer patients once you see these big
         | differences. Same thing should happen if you see the opposite,
         | e.g. killing 70% of your experimental group.
        
           | disgruntledphd2 wrote:
           | Yeah if it's a potentially life saving treatment you really
           | need that design that allows peeking at the results.
        
           | protomyth wrote:
           | Everyone knew the results of "standard treatment", that's why
           | they were researching the drug. There were years and years of
           | statistics.
           | 
           | We killed 70% of the control group. Doomed by a random number
           | generator.
        
             | bigfudge wrote:
             | If that was the case, the study actually was unethical.
             | 
             | Some designs do include interim analyses and stopping
             | rules.
             | 
             | However in my view the real scandal is we are still using
             | NHST for clinical trials. We should be continuously
             | updating a sensible prior for the effect size and approving
             | the drug/stopping the trial when we have sufficient
             | evidence one way the other.
             | 
             | As it is, the results of many underpowered trials are
             | essentially thrown away because p > .05, which is stupid.
             | This says nothing about the balance of evidence for the
             | efficacy of an intervention... only the inertia and
             | innumeracy of many clinicians preserves the tradition.
        
             | s1artibartfast wrote:
             | Not trying to invalidate your experience, but I think it is
             | interesting and disagree.
             | 
             | I suspect there are some people with different philosophies
             | that that sit better with this sorta thing.
             | 
             | It seems like you feel guilt not only for the harm you
             | cause, but the harm you fail to prevent. Do you apply this
             | logic to other parts of your life?
             | 
             | How do you feel about the trolley problem, were you have to
             | kill some to save others?
             | 
             | How do you feel about the moral imperative of doctors to do
             | no harm versus a utilitarian approach of maximize lives
             | saved?
        
               | protomyth wrote:
               | I don't feel guilt, I still feel rage. I didn't determine
               | any of the parameters or rules of the trial so I have no
               | guilt on me. I just observed that the control group is
               | dead because of some belief that they provided value when
               | we already knew exactly what was going to happen to them.
               | 
               | If you can prevent harm to others, then not doing so is
               | just being an a$$. The trolley problem is just counting
               | souls and really doesn't happen much in real life. The
               | true problem is thinking the only choices are us/them
               | versus everyone. I'm not a doctor so I have no idea how
               | their ethics applies to their professional decisions.
        
               | dekhn wrote:
               | Control groups exist because they get us closer to global
               | optimality.
               | 
               | We can't eliminate suffering and dying of untreated
               | people but it's generally considered ethical to eliminate
               | suffering and dying of many while withholding that
               | treatment from a subgroup of the study ("allowing them to
               | die" by _not taking action_ , rather than the "causing
               | them to die" by _taking action_ ).
               | 
               | as long as that selection is done randomly and "fairly" I
               | think it's an entirely acceptable risk. There have been
               | trials that were truly badly run, and people died and
               | suffered more than necessary, due to mistakes (often
               | amateur ones) in the protocol. I'm more concerned about
               | those types of deaths.
        
               | s1artibartfast wrote:
               | Thanks for clarifying your position
        
             | funklute wrote:
             | > Everyone knew
             | 
             | If that was truly the case, then - as the parent hinted -
             | it was the trial design that was the problem, and not the
             | practice of having control groups.
             | 
             | It is pretty common to stop trials early when it is obvious
             | that the treatment works. Conversely, it is easy to think
             | that "yea of course the new treatment works" despite the
             | evidence not being there. The need for robust analysis must
             | also be respected.
        
               | protomyth wrote:
               | It was my understanding that every study has a control
               | group getting the standard care. I didn't see a study
               | without such a control group.
        
               | anthuswilliams wrote:
               | No, in fact it is quite common to do "open label"
               | clinical studies in which patients know what drug they
               | are getting and no one is given the current standard of
               | care. This is especially common in cancer, where the
               | standard of care is so poor, and in rare diseases, where
               | the patient population isn't large enough to admit such a
               | comprehensive study.
               | 
               | It is harder to have the same statistical confidence of
               | efficacy and safety in such studies, but clinical
               | researchers try to address those issues by varying e.g.
               | dosage quantities, time in between doses, etc.
               | 
               | Source: place I work is currently doing studies of this
               | nature, and in general such studies seem to be well-
               | understood and accepted by the FDA.
        
               | s1artibartfast wrote:
               | Randomization is independent from blinding. Open label
               | studies can be either randomized or not, and controlled
               | or not.
               | 
               | The FDA will usually push for blinding if possible
               | (sometimes it is not). They will also usually push for a
               | randomization zed control with standard of care. They way
               | the FDA views it (and I agree), is that control patients
               | are not harmed because they are still getting the same
               | care they would outside of the trial.
               | 
               | It is usually unethical to have a treatment free arm.
               | However, this has its own problem, where if you keep
               | using equivalence comparisons, the end of the chain might
               | not actually be any better
        
               | funklute wrote:
               | Yes, exactly. Control groups are incredibly important for
               | ensuring good quality of clinical studies. It's a
               | technique that solves multiple "calibration" problems,
               | including:
               | 
               | - being able to draw causal conclusions
               | 
               | - being able to adjust against placebo effects
               | 
               | A lot of clever people have tried to come up with ways of
               | doing away with control groups. But ultimately, the best
               | we can achieve is to stop early, as soon as the trial has
               | a clear outcome. I do perhaps think this has become more
               | common in recent times though, so perhaps the study you
               | were involved in was at a time when early stopping wasn't
               | really "the done thing".
               | 
               | But it still beats "studies" done 100 years ago, when you
               | might give someone a cough mixture, see that they
               | improved (if they died, let's just ignore that), and
               | conclude that it was the cough mixture that did it!
        
               | robocat wrote:
               | > the best we can achieve is to stop early, as soon as
               | the trial has a clear outcome
               | 
               | That must be really hard: if you wait for 95% confidence,
               | you are selecting for 5% noise. If you repeatedly re-
               | measure for 95% confidence, you strongly select for
               | random noise.
               | 
               | Not many medical advancements provide such an anomalously
               | strong signal (98% survival versus 30% survival).
        
               | funklute wrote:
               | > If you repeatedly re-measure for 95% confidence, you
               | strongly select for random noise.
               | 
               | You can't use standard methods for early stopping - as
               | you rightly point out, you get gibberish if you naively
               | keep peeking at a growing data set. Instead, you have to
               | use statistical methods that explicitly adjust for the
               | repeated sampling in early stopping trials. This does
               | make early stopping more complicated to analyse than a
               | trial with a pre-determined duration.
        
               | tsbischof wrote:
               | For such a large effect it is quite possible to implement
               | an adaptive trial design with unbalanced arms and interim
               | analysis for efficacy. But you have to ask for this, the
               | FDA will not necessarily suggest it directly.
        
           | Khelavaster wrote:
           | Peek-and-do-more is compatible with double-blindness, as long
           | as the peeker-and-decider is different from the dispenser and
           | patient.
        
       | photochemsyn wrote:
       | Interesting article, but the lack of mention of 'patent' or
       | 'intellectual property' sort of stands out. There may be many
       | out-of-patent drugs that are effective treatments for various
       | conditions, but typically clinical trials for alternative uses of
       | out-of-patent drugs are not pursued as anyone could then
       | manufacture and sell them.
       | 
       | This is not new - it dates back to the origins of the modern for-
       | profit pharmaceutical industry, such as with Bayer's discovery of
       | the anti-bacterial action of sulfanilimide (patented 1909) in the
       | early 1930s (by which time patent had expired). Bayer made a
       | derivative of sulfanilimide, i.e. Prontosil (patented in 1932)
       | and tried to convince the world that this was the more effective
       | product. In reality, it just broke down to sulfanilimide once
       | ingested, that being the active species.
       | 
       | As far as clinical trials in the USA, there have been numerous
       | scandals regarding lack of consent, failure to halt trials
       | immediately on indication of severe side effects, etc. Major
       | stories about this came out in 2005, 2008, etc. Then there's the
       | world of unregulated third-world clincal trial testing, and
       | actual falsification of clinical trial data by contractors hired
       | to run trials for various Big Pharma outfits, such as this from
       | 2022.
       | 
       | https://endpts.com/clinical-trial-coordinators-sentenced-to-...
       | 
       | > "Tellus, which has worked with big-name pharma sponsors in the
       | past like Pfizer, Gilead, Takeda, Boehringer Ingelheim and Eli
       | Lilly, initially came under scrutiny for two trials related to
       | opioid dependency, two for irritable bowel syndrome, and one for
       | diabetes."
       | 
       | That's just organized crime 101: hire a contractor to do the
       | dirty work, keep your own hands clean, claim ignorance, act
       | shocked, promise reforms, etc.
       | 
       | Best solution I've heard of is a move to open-source (patent-
       | free) drug discovery with federally financed clinical trials.
       | Since almost all new drugs are discovered at universities with
       | federal funds (see remdecivir, mRNA vaccines, etc.), before being
       | exclusively licensed to private interests, the quickest way to
       | that is to revoke Bayh-Dole and state that universities must non-
       | exclusively license their discoveries to any parties for a flat
       | fee.
        
       | pfdietz wrote:
       | One thing not mentioned: people are paid to participate in
       | clinical trials. For example, in a vaccine clinical trial I know
       | about, participants are paid over $1000 (in dribs and drabs over
       | the course of the trial, as a retention mechanism.)
       | 
       | With tens of thousands of participants, this cost adds up.
        
         | idealmedtech wrote:
         | Patient compensation is intentionally limited to prevent
         | economic incentives from interfering with informed consent; pay
         | people too much and they may participate in research that they
         | would have passed on. Also, speaking from experience, patient
         | compensation is a very small amount (5% or less) of overall
         | cost. The vast majority goes to clinical staff costs, room
         | costs, hospital overhead, labs etc.
        
           | pfdietz wrote:
           | That's not a vaccine trial though, right? I'm figuring those
           | need more participants and have more retention problems, as
           | well as less risk of informed consent problems.
        
             | ebiester wrote:
             | I mean, the CONFIRM trial
             | (https://www.research.va.gov/currents/1117-Largest-ever-VA-
             | cl...) had 50,000 patients. (That's not a pharmaceutical
             | sponsor-led clinical trial, but it is a clinical trial.)
             | 
             | But yes, the largest phase 3 trials ever are generally
             | vaccines. https://www.evaluate.com/vantage/articles/news/sn
             | ippets/its-...
        
           | boole1854 wrote:
           | > Patient compensation is intentionally limited to prevent
           | economic incentives from interfering with informed consent;
           | pay people too much and they may participate in research that
           | they would have passed on.
           | 
           | I'm not sure I understand the logic here. How does agreement
           | suddenly become less informed or less consensual because the
           | participant gets paid more? Sure, if they get paid more, they
           | might be willing to accept more risk or do something more
           | unpleasant. But that seems fair, not immoral. Why would it be
           | more fair to say "yes, perhaps this trial is risky, but we
           | only want participants who don't demand to be compensated
           | much for the risk they take"? If anything, maybe _that_
           | approach leads to less informed consent by filtering out the
           | people who actually understand the risk and would have only
           | agreed to participant in exchange for more compensation!
           | 
           | Should we also be morally offended any time an employer
           | raises wages in order to attract more workers?
        
             | knodi123 wrote:
             | > How does agreement suddenly become less informed or less
             | consensual because the participant gets paid more
             | 
             | Why shouldn't college professors sleep with their students?
             | How does the relationship become less informed or less
             | consensual? The answer is power.
             | 
             | Money is power, and too large of a power imbalance corrupts
             | a relationship. Money is pressure, and too much pressure is
             | coercive.
        
               | JamesianP wrote:
               | Professors historically were prohibited from
               | relationships with students because it creates the
               | appearance (and high likelihood) of favoritism. And it
               | probably pisses parents off a lot because the age
               | difference seems creepy.
               | 
               | "Power" is a relatively new argument. These days the
               | student has the power to utterly destroy the professor.
               | The professor has at best the charismatic "power"
               | equivalent to any number of smooth pickup artists
               | occupying college dorms, of getting a temporary good
               | time.
        
               | s1artibartfast wrote:
               | I think it's still a fair question. The money is intended
               | to be an incentive. If the trial is ethical and has a
               | positive risk benefit ratio, there is no harm from
               | coercion.
        
               | knodi123 wrote:
               | Okay, but what if a person sincerely does not want to
               | participate in a medical trial? But then when he sees the
               | dollar figure, he thinks "well, shit, I hate doing this,
               | but I can't afford to turn that much money down."
               | 
               | Same reason we don't allow the purchase of kidneys, and
               | the same reason we think it's unethical for a millionaire
               | with a tophat and a monocle to offer two homeless guys a
               | wad of money to the winner if they fight each other.
               | Sure, it's an adult making a decision of their own free
               | will- but it's obviously unethical.
        
           | pinky1417 wrote:
           | That's true. Good Clinical Practice guidelines say that
           | Institutional Review Boards must evaluate compensation is not
           | too high otherwise it constitutes "undue influence".
           | 
           | ...but I think that's a terrible idea. I understand that, to
           | come up with a far-fetched example, high compensation could
           | result in some weird edge cases where, for example, a Phase I
           | trial with healthy volunteers and a non-preventive drug
           | candidate (e.g. an antibiotic rather than a COVID vaccine),
           | but you're confident that drug is going to kill 99% of the
           | volunteers so you offer $10 million to each volunteer. A
           | totally informed volunteer might view this as something akin
           | to a life insurance policy that pays out to the family even
           | upon suicide. I don't personally see an ethical problem with
           | that so long as volunteers are _really_ well-informed, but I
           | could see how others and the law would be uncomfortable with
           | the ethics of such a situation.
           | 
           | But aside from that far-fetched situation, it seems harmful
           | to limit compensation... especially if it's true that patient
           | compensation is a small part of overall cost. Perhaps if
           | every trial doubled compensation to 10% of today's overall
           | costs, absolute overall costs would _decrease_ because less
           | effort has to go into patient recruitment.
           | 
           | I do see one real-world issue with high compensation, but it
           | has nothing to do with ethics. It has to do with statistical
           | interpretation. If you start dangling too much money to
           | participate in a trial, you may change the kind of people who
           | participate in the trial. These individuals might not be
           | representative of the actual users of the drug. The problem
           | is similar to running a drug trial in the US versus Japan.
           | The US has a pretty diverse population with respect to
           | genetics. Japan, in contrast, is almost entirely ethnic
           | Japanese. If you were to somehow succeed in using Japanese
           | trial data to market an acne cream in the US, the same drug
           | that cleared up Japanese faces might result in Venezuelans
           | having their noses fall off!
           | 
           | However, I'm cautiously optimistic that higher compensation
           | could make trials _more_ representative. In my mind, I could
           | see how African-Americans, who tend to participate in trials
           | at a lower rate than their Caucasian fellow humans, would, if
           | only for the shameful history of the Tuskegee Syphilis Study,
           | require greater compensation to take the risk of
           | participating in a trial.
        
             | idealmedtech wrote:
             | As you said, at the end of the day it's an ethical
             | guideline that must be upheld, more than something based in
             | economics. We do compensate participants as well as we're
             | allowed within these guidelines!
             | 
             | I agree with your points, but compensation must be approved
             | by the independent institutional review board, who is
             | accountable to national ethical review boards and thus acts
             | very conservatively.
        
             | JamesianP wrote:
             | I would think the worst of such edge cases should be
             | prohibited by law anyway. A set of more subjects is
             | presumably a superset of a set with less subjects. So any
             | issues can be addressed by screening them better. I suppose
             | there could be a greater problem with fraud as people claim
             | to have issues when they don't.
             | 
             | It just seems unfair to subjects to me. Not to mention
             | paternalistic. Every other actor in the process gets
             | enriched based on (limitless it seems) market rates for
             | their contribution, except for the subjects who take on all
             | the risk.
        
       | fuckyah wrote:
        
       | robocat wrote:
       | Call me cynical, but this could apply in most any industry:
       | Suppose that one tries to push back on inefficiency in
       | generalsomeone naive but well-intentioned, like myself, who is
       | tired of the Long March of interminable meetings. In fact, I
       | expressed my concerns about the proliferation of unproductive
       | meetings in the clinical department to the C-suite executives
       | (with whom I remain on good terms!) and, together, we carefully
       | planned a series of reforms to company culture designed to reduce
       | meeting burden. We hoped to implement a soft cap on the number of
       | meeting participants and the length of a meeting, to normalize
       | efficient behavior such as leaving meetings midway if you no
       | longer need to participate in the remainder, starting meetings on
       | time without 15 minutes of small talk, and a mandated review of
       | all recurring meetings with the intention of pruning overall
       | meeting load by >50%.            Ironically, on the morning that
       | we planned on roll out these reforms, we received urgent and
       | vehement protests from a member of middle management who was
       | notorious for obsessively overscheduling meetings with huge
       | participant lists just for the sake of "inclusion" and
       | "engagement." Because this particular manager was currently
       | playing a crucial role in managing the rollout of several
       | clinical trials at the same time, executive management judged
       | that the risk of alienating him was too high, and to this day I
       | believe these reforms remain unimplemented (although I hear he
       | has since left the company, so perhaps my slides will eventually
       | see the light of day).
        
       | manv1 wrote:
       | FYI, the RFP process is basically the same in other industries.
        
         | apohn wrote:
         | I came here to post the same thing.
         | 
         | I used to be in enterprise software sales and consulting and
         | have been involved for RFIs/RFPs across multiple industries. I
         | don't know a single person who thought the process was sensible
         | or efficient. Unless you have an person whose job is dedicated
         | to responding to the questions, it's basically just a game of
         | passing a hot coal around until you have something you can
         | submit.
        
       | NDizzle wrote:
       | One point in there - data management. It varies in cost because
       | on the upper end, that includes support for data change requests
       | for the life of the trial. Zillions of people involved, human
       | error, need paper trails and sign offs to fix most things.
        
         | bmj wrote:
         | This is usually downstream of the CROs, or, at least contracted
         | out. What's most interesting to me about the post is that the
         | author doesn't mention any efficiencies gained by moving to
         | electronic data collection. But, I also understand why it isn't
         | mentioned -- I work for an EDC/eCOA provider, and our biggest
         | competitor for new business isn't our competitors in the space,
         | it is paper-based data collection.
        
       | olieidel wrote:
       | This is so, so interesting. Thanks so much for posting this!
       | 
       | The author already touched upon a few hypotheses why the industry
       | has arrived at this state. There's one I'd like to highlight: The
       | author, now having gone through this experience, would actually
       | be in a perfect position to found their own CRO and implement all
       | their efficiency learnings there. However, they probably look
       | back upon this experience and think it was super painful and
       | would prefer to do fun stuff like developing software instead.
       | 
       | That, for me, is the main reason. There are smart, structured,
       | pragmatic people who got acquainted with this industry, but they
       | choose to leave it.
       | 
       | And that's sad!
       | 
       | So the solution would be to find a group of smart, structured,
       | pragmatic people with a high tolerance for bureaucratic pain and
       | give them the resources to make this industry more efficient.
       | 
       | And here's my plug: That's exactly what we're doing at
       | OpenRegulatory [1], albeit for medical device software and not
       | for clinical trials. Among other things, we've published all our
       | document templates for free (also on GitHub), host a free Slack
       | community for companies to help each other out, and offer a
       | compliance SaaS with transparent and fair pricing. It's still a
       | long way, but it's a start :)
       | 
       | [1] https://openregulatory.com
        
         | BeefySwain wrote:
         | Your company is very interesting. I briefly looked over the
         | website, but couldn't tell what country(ies?) you operate in?
         | My assumption is the regulatory requirements of each country
         | are dramatically different, so seeing the pricing in not $
         | makes me think it's unlikely you offer services in the US, for
         | instance.
        
           | jtwaleson wrote:
           | They operate mostly in Germany, but the Slack community
           | around it is broader, I think 90% is EU based though.
        
       | refurb wrote:
       | I worked in clinical development and the rough estimate of phase
       | 3 clinical trial costs (this is a few years back) was $15,000 per
       | patient per year.
       | 
       | Depending on the trial design it can be less (less frequent
       | follow up, less invasive testing) or a lot more (say doing
       | multiple MRIs or other expensive imaging).
       | 
       | It's kind of mind blowing when you think about the really big
       | trials for medications like cholesterol. Trials are often 30,000
       | or more patients, in two trials for 2-3 years.
       | 
       | You're roughly talking about $2B or more just for phase 3
       | (ignoring all the earlier trials and preclinical work).
        
         | s1artibartfast wrote:
         | Another huge cost is comparator products. Some trials budgets I
         | have seen spent 100-200+ million purchasing the drugs they are
         | trying to outperform. This is for trials with <1000 patients.
        
       | idealmedtech wrote:
       | There's a reason why we ended up putting our trials together
       | essentially by trial and error! You learn a lot by writing your
       | own clinical protocol, interacting with the FDA etc. It's an
       | intimidating process at the outset, but if you can save $200k on
       | a first-in-man study by not contracting a CRO, that's gonna
       | extend your runway by a lot!
        
         | seidleroni wrote:
         | A fair point, but with the FDA responses taking up to 30 days
         | you burn a lot of runway.
        
           | ska wrote:
           | They have a 180 day review commitment. A 30 day delay on
           | their side would be unusual and burn a big chunk of that
           | clock.
        
           | s1artibartfast wrote:
           | That's why you have several Type B or Type C meetings during
           | development to work out the kinks before you submit a
           | protocol.
        
         | vibrio wrote:
         | I don't completely disagree with your point, but for one's
         | clinical regulatory strategy, perhaps "trial and error" isn't
         | the best branding to appeal to investigators, patients,
         | regulators or investors.
        
           | idealmedtech wrote:
           | Perhaps I was being too self deprecating! We worked closely
           | with the FDA through multiple QSub meetings to make sure it
           | was up to snuff, and did a lot of legwork on the backend to
           | make sure we understood what they were asking for. 3 or 4
           | revisions later, we had an approval!
        
           | s1artibartfast wrote:
           | Are you under the impression that that's how they branded
           | things?
        
         | adultSwim wrote:
         | If you are worried about $200k, do you have enough money to
         | bring your product to market?
        
           | idealmedtech wrote:
           | When we started putting the trial together, we were still
           | self funded. In medical device, to get to series A you need
           | human data, so by doing the trial protocol ourselves, we
           | avoid the chicken and egg problem of needing money in order
           | to raise money. Once you get human data, your valuation
           | skyrockets.
        
       | SubiculumCode wrote:
       | Nicely written piece highlighting the value of in-house expertise
       | and capabilities, providing a lesson that translates very well
       | imo to why government projects should have more in house
       | expertise and not contract everything to the private sector.
        
       | napier wrote:
       | Best explanation I've read online. Most attempts at describing
       | why boil down to the circular "clinical trials are very expensive
       | because the process of getting a new drug approved is lengthy,
       | complex and expensive".
        
       | Melissa_bime wrote:
       | I totally understand the frustrations experienced by the author
       | of this post. I am a nurse and my exposure to the Clinical Trial
       | Industry came from a more bottoms up entry point. Its mind
       | boggling to the the total costs CRO's charge for the services
       | compared to the eventual experience for the staff and patients
       | taking part in these studies. CRO's are in dire need of
       | disruption. I also agree about the incestuous industry hence the
       | very slow progress being observed. It's hard to show any progress
       | when it's the same people moving around the same companies and
       | implementing ideas that are somewhat outdated. Also, from a cost
       | perspective, i think the future is also coming to a place where
       | Clinical trials get outsourced outside the US. Sites and PI's
       | outside the US should be empowered to run this clinical trials as
       | they provide a more cost effective solution with regards to
       | operational costs and also access to an ever growing number of
       | patients, reducing clinical trials timelines that lead to
       | increased costs. I understand the need for CRO's to maintain
       | quality, and they can do this by building out the right
       | relationships and partnerships with overseas contractors, but i
       | believe in the long run, this is the solution. I believe this so
       | much that i launched my own CRO company. Infiuss Health. The goal
       | of this company is to unlock the potential for studies to the run
       | by CROs and smaller life science companies outside the US. We are
       | starting with Africa. As soon as we can unlock this population
       | that has traditionally just been taboo to life science companies,
       | imagine the lengths we could go with with access other
       | geographies.
        
       | invitrom wrote:
       | I am eagerly waiting for the clinical trial on BC007 for Long
       | Covid. As a chronically ill patient the slow pace of research is
       | almost unbearable.
        
       | mandmandam wrote:
       | This all sounds so familiar.
       | 
       | How many industries could these sentences descrive, stripped of
       | context?
       | 
       | Or more easily, are there _any_ industries where this is _not_
       | the norm?
       | 
       | > an industry which is severely lacking in human capital and
       | fully captured by bureaucratic tendencies
       | 
       | > going through this process is an extended exercise in sheer
       | absurdity.
       | 
       | > meaningless bloat
       | 
       | > a farcical "data-driven" scoring process
       | 
       | > there was an intermediary clinical organization we attempted to
       | work with which was so obviously attempting to grift money from
       | our coffers, the entire relationship escalated into the level of
       | back-and-forth legal threats
       | 
       | > One cannot help but get the impression that this is an industry
       | which is, at best, severely lacking in anything that could be
       | called "human capital" and, in reality, simply deeply unserious
       | 
       | > despite the very large amount of boilerplate content that every
       | single actor in this ecosystem has spent thousands of hours of
       | manpower developing, there seem to have been very few actual
       | gains made in simplifying common tasks or processes!
       | 
       | ... Et cetera.
       | 
       | To me, this sounds a lot like processes in education, teaching,
       | energy, environmental protection, agriculture, research, arms,
       | media, law, IT, etc.
       | 
       | I don't believe we can afford to remain this stupid, however much
       | money the inefficiencies are making for those at the top.
        
         | knodi123 wrote:
         | > Or more easily, are there any industries where this is not
         | the norm?
         | 
         | porn?
         | 
         | Or in general, any business with highly mobile
         | customers/employees and narrow profit margins.
        
       | turnsout wrote:
       | Great summary! And yet, for all their problems, CROs are
       | sometimes still the fastest alternative. We did a clinical trial
       | for a software platform a few years ago, and hoped to partner
       | with a prestigious hospital system to run the trial.
       | 
       | The hospital partner was 100% on board--our protocol just had to
       | pass their internal review board (IRB). IRB is a black box, and
       | they could only give us a vague estimate of how long this would
       | take. We had a huge team, and every week we sat around waiting
       | cost about $200k.
       | 
       | Finally the client couldn't take the uncertainty and burn rate
       | anymore, and they reached out to a CRO they had used in the past.
       | The CRO's IRB had a fixed timeline (2 weeks?), and we were
       | onboarding patients a short time later.
        
         | MichaelZuo wrote:
         | Why couldn't the team be tasked on something else while
         | waiting?
        
         | 1auralynn wrote:
         | Yeah the lesson with getting IRB approval if you want it done
         | in less than 6 months is to always use an external IRB (if
         | possible). Institutional IRBs are sloooooowwww.
        
         | BurningFrog wrote:
         | Soap box: IRBs are one of many things deteriorating modern
         | science, and in great need of reform.
        
       | getsloped wrote:
       | The logistical inefficiencies mentioned in this article are mind
       | boggling when compared to what we've come to expect in different
       | industries. We all know healthcare is always a little behind the
       | curve, but with clinical trials, we're talking decades.
       | 
       | For example: most clinical studies have no way to track samples
       | going from sites to labs beyond a hope, prayer, and constellation
       | of unconnected spreadsheets. The status quo is to write a 50 page
       | lab manual and include a wall of text and bullets outlining a
       | list of multiple stakeholders that need to be emailed a tracking
       | number each time a sample is shipped. Not to mention there are
       | days you really shouldn't ship samples. Labs have no idea when
       | samples are going to show up. Seriously, samples just show up,
       | they open the box expecting a piece of paper to identify the
       | samples.
       | 
       | An incredible amount of perfectly good, usable samples get LOST
       | or DESTROYED due to the fact that there's no way to track any of
       | these samples or coordinate stakeholders. We're talking critical
       | samples, like blood from kids with cancer. It's absolutely
       | ridiculous.
       | 
       | Compare this sob story to the fact that I know in _realtime_
       | where all the dumb shit I bought on Amazon is. The cognitive
       | dissonance here is beyond comparison!
       | 
       | + + +
       | 
       | About 8 years ago my brother and I were given a window into this
       | problem. Being Amazon FBA sellers ourselves, we thought, "hey now
       | that can't be too hard to fix" and thus began the wild journey of
       | Slope [0]. What we learned working on this problem is that
       | clinical research sites literally get crapped on left and right
       | with ridiculous contracts & protocols written by people who don't
       | understand the clinical setting or logistics in general. With
       | each research site running 30-50 clinical studies and each of
       | those studies having its own catalog of kits, shippers, and other
       | inventory, it's no wonder why clinical research is so expensive!
       | And this is just supply chain folks.
       | 
       | Slope started by making an inventory and sample management
       | software to address the needs of sites and have worked backwards
       | into selling this platform to Sponsors and CROs. While I have
       | your attention, we're hiring a head of engineering now and will
       | have a JD up for a product manager next week [1]. Thanks for
       | reading!
       | 
       | [0] https://www.slope.io/ [1] https://www.slope.io/jobs/head-of-
       | engineering
        
         | trap_goes_hot wrote:
         | People complain about industries with bureaucracy but nobody
         | wants to take the hit and actually work in those industries.
         | They want magically for their pay/benefits/culture incentives
         | to align.
        
           | ativzzz wrote:
           | Exactly - like the OP you replied to - Slope are trying to
           | hire a Head of engineering for a salary that competent
           | mid/senior level engineers should be easily getting. It's no
           | tech industry, and the work is less fun
        
       | xiphias2 wrote:
       | While there are lots of new biotech tools, most of the illnesses
       | left to treat are caused by aging, when the human system falls
       | apart from the interaction of old parts, therefore super hard to
       | treat.
       | 
       | At this point I gave up waiting for a medical solution for my
       | chronic illness, and have a better hope that I can live 15-20
       | years with it and rejuvenate my body after that time period.
        
       | wins32767 wrote:
       | The author doesn't really understand the details of the
       | operations of clinical trials, though this is a good overview of
       | the kinds of outcomes that show up. I worked on operations
       | software for clinical trials for about a decade, and there are
       | (mostly necessary) regulatory drivers a large fraction of the
       | complexity he complains about. The first order effects of the
       | regulations are pretty straightforward, but when you get into
       | second, third, and fourth order impacts, you start seeing some of
       | the outcomes he's describing.
        
       | [deleted]
        
       | AlbertCory wrote:
       | I have to admit I found this tl;dr. That's my bad, because it's
       | an important article.
       | 
       | All the bureaucratese in the RFPs and heavy-duty lawyering just
       | add, unnecessarily and hugely, to the cost. At bottom, though,
       | you have:
       | 
       | 1) A lot of human beings who have to be recruited and paid to be
       | subjects
       | 
       | 2) Medical institutions that have to administer the drugs or run
       | the devices
       | 
       | 3) A sufficient amount of elapsed time for effects and side
       | effects to manifest
       | 
       | 4) A reporting system that's trustworthy
       | 
       | So at best you could cut (puts finger to the wind) _half_ the
       | cost? Surely not 90%.
        
       | pinky1417 wrote:
       | I worked at a small-to-mid-size clinical research organization
       | for a hot minute. That company had its own set of - let's say
       | "unique" - problems*, but the author touched on many of the
       | issues I saw in my short experience at a CRO. I was in an odd
       | (for me), pseudo-managerial, HR-type role at the company (a role
       | in which I'll be the first to admit I wasn't good at) so perhaps
       | I can offer some additional perspective.
       | 
       | But first, I want to give some background on an important part of
       | the CRO world: the "clinical research associate" or "CRA". CRAs
       | are are the CRO's meat-and-potatoes, boots-on-the-ground, core,
       | etc. after contracts have been signed. A CRA is the person who
       | actually goes to clinical trial "sites" (e.g. a doctor's office,
       | a hospital, or, even a dedicated facility for clinical trial
       | participants). The CRA goes to each trial site, talks to the doc,
       | makes sure participant recruitment is going well, checks/writes
       | documentation, and follows up as the trial goes on. In other
       | words, the performance of the CRAs strongly & directly affects
       | the performance of the clinical trial.
       | 
       | Anyway, my top issues with clinical trials today are:
       | 
       | (1) Poor patient recruitment - this is a major problem. There is,
       | of course, lots of variability between trials and CROs with
       | respect to recruitment, but it should be telling that "trial
       | rescue" is often done (at least where I worked) as a result of
       | the incumbent CRO's inability to recruit enough patients.
       | 
       | (2) (Related) Limiting pay to study participants: although it's
       | up to the institutional review board's interpretation (IRB, an
       | ostensibly independent entity that's supposed to sign off on the
       | trial's ethics), good clinical practice (GCP) guidelines states
       | that paying participants too much would be "undue influence". I
       | have no idea how this plays out in practice, but I think it's a
       | bad guideline. Most of the other parts of GCP are good things -
       | stuff like informed consent and not coercing subjects - but this
       | one is (again, in my opinion) probably raising trial costs
       | through recruitment delays or total non-recruitment, even
       | accounting for the extra cost of paying participants more.
       | 
       | (3) Clinical Research Associate (CRA) training & hiring: this
       | isn't unique to the CRO industry but it seems like a bigger
       | problem than in other industries. I've seen US median salary
       | ranges from $70,000 to $120,00 from publicly available sources
       | but my time in the industry rarely demonstrated numbers that
       | high. Although CRAs don't treat patients directly, it is a role
       | that requires medical knowledge, attention to detailed paperwork,
       | and good communication skills. There seemed to be a lot of
       | cutting corners on training & hiring CRAs - e.g. hiring highly
       | experienced CRAs from India and putting them into entry-level
       | roles in the US on the cheap. I don't mean to say there aren't
       | great experienced CRAs from India who are also great when dropped
       | into an entry-level CRA role but doing so as a matter of
       | unofficial policy is a bad idea: India's drug industry has a
       | different regulatory environment (although there is significant
       | cooperation between FDA and Indian manufacturing facilities) and,
       | perhaps more importantly, there will be a wide variation in
       | communication skills simply because english is a second language
       | (or, in the case of India in particular, "Indian english" has
       | taken on a life of its own).
       | 
       | (4) Over-promising, under-delivering - true of any contractor
       | even outside the CRA industry. Even when working on a fixed price
       | basis, there are perpetual "change orders" and arguments over
       | change orders.
       | 
       | Side-note: if you want to learn more about the process by which
       | drugs come to market, I strongly recommend _New Drugs_ by
       | Lawrence T. Friedhoff. It 's short and the HN crowd won't have
       | any problem understanding. Amazon link:
       | https://www.amazon.com/New-Drugs-Insiders-Scientists-Investo...
       | 
       | * "Unique" problems in the sense that they were personality-
       | based. I won't go into specifics nor do I wish to speak
       | negatively of my prior employer who, to their credit, took a
       | chance on me that just happened to not work out. But I will give
       | some general advice: if, during a job interview or in early
       | conversations, senior management insults his/her own family _and_
       | dumpsters their own employees, you 're going to have a bad time.
        
         | esel2k wrote:
         | I would add to your list of problems: Decades of easy-money
         | milk-the-cow style companies (Often CROs) with very outdated
         | systems with folks sittings in cheap countries to do some data
         | change requests and manual tests. Truely horrible and outdated
         | tech, mixed with bad salaries, high turnover and bad
         | management. I used to work for a company were because of
         | timezone issues a wrong date was entered, CRA requested
         | correction (edit manually, sign, scan) and wrong assumptions
         | etc we had to change 7 times the whole thing and send CRA
         | onsite and recorrect the mess. In short: Inefficient mess.
         | 
         | I left that industry quickly and can say I that others are
         | better. Lab tracking is way better with diagnostic samples with
         | private labs or when mixed with good logistics partners. It is
         | not everywhere as bad as this.
         | 
         | TLDR: add outdated tech, greed and bad management
        
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