Science & Medicine

Missing Seat at the Table

MPN patients not invited to the party.

– Zhenya Senyak

In this broken humpty dumpty world, there’s nothing quite so reassuring as a concerned doctor poking and prodding us in a determined effort to put us back together again. Norman Rockwell sateve That perception of the family doctor, that Norman Rockwell vision of reality, is long gone. Its lingering traditions are being crushed underfoot by the pressures of current medical practice.

We have seen the medical system regularly abused through sharp conflicts of interest, unnecessary tests and procedures, overprescribed meds, over-treated diseases all while overbooked doctors are forced to disregard and disrespect the essential intelligence and humanity of their patients in the interest of production. The result: A disconnect between physician and patient.

We have some responsibility to turn things around. Sometimes we just need to rock the boat.

Last week’s abstract from a Leukemia article underscores just how marginalized patients have become.  

24 international MPN specialists – and these are among the very best, some of our strongest allies and most valued physicians – assembledleukemia consensus cover to consider “trial design, patient selection and definition of relevant endpoints” of the  MPN clinical trial without patient input..

The International Working Group-MPN Research & Treatment and the European Leukemia NET experts published two consensus conclusions in its abstract.

To claim these are consensus-based recommendations on trial design, patient selection and relevant endpoints is a bit of stretch.  Without input from affected patients, it’s impossible to reach true consensus of affected parties.

Two of the group’s conclusions underscore how deep the divide is between us and our doctors when it comes to structuring the clinical trial process.

(1) “New drugs should be tested for preventing disease progression in MF patients with early disease in randomized studies…” and

(2)  There should be a format for clinical trials in MPNs “which facilitates communication (among) academic investigators, regulatory agencies and drug companies.”.

Let’s take a close look at those conclusions. 

We can call the first consensus recommendation “Try and See.” Basically,  give an early stage MF patient an investigative drug and see how it works over time — as opposed to “Watch and Wait.” 

There are strong arguments for early treatment of myelofibrosis …just as there are good arguments for Watch and Wait and minimal use of drugs beyond relief of symptoms.  While the objective of stopping MF in its tracks is laudable,  there is no therapy, however early applied, that can guarantee MF will be held in check.  Nor is there any MF therapy free of potentially dangerous side effects. 

But this isn’t an exclusively clinical or medical issue.  Entering a clinical trial is a human experience with physical, emotional, economic, social and spiritual components.

Designing the blueprint for the clinical trial system would be a good time to bring in early stage MF patients.  It would not be difficult to circulate proposed alternatives and  supporting evidence over the Internet and get patient input.

This is a good instance of the Great Divide, a steep and unbridged  chasm between doctors and patients.  Why recommend placing us in clinical trials of untested drugs at early disease stages without first assessing how we feel about taking that step, what our reservations are, what outcomes would make it a reasonable choice for us, what safeguards we would need?

Serious a lapse as “Try and See” is, it is the second conclusion that truly highlights the great structural divide between medical providers and patients.

Of course communication among investigators, regulators and drug companies  has to be facilitated. But isn’t there a chair missing at the table?  Is there no place for the primary stakeholder in clinical trials, the patient who will be presenting his or her body for medical experimentation?  Despite suffering and dying in clinical trials,  patients are traditionally excluded from collaborative input into work that will affect their lives and determine their fates. 

Drug companies, regulators and investigators already communicate with one another. It just has to get better, faster, more comprehensive across clinical trial sites.  But communications among drug companies. regulators and patients is essentially non-existent and between investigators and patients communications is only sporadic and dependent on personality and circumstance. Why not bridge the gap and include patients in the recommendations for facilitated communications? 

As we are herded and channeled through the medical treatment corral, we are made to understand how little our humanity counts. We become account and insurance numbers,  plotted points on a chart.  Our various complaints are given ICD numbers. Our fluids are taken, numbered, and recorded.  And we are given forms to fill out, prescriptions to follow, tests to report for, results to note, and bills to pay.

It’s all pretty straightforward.  Our doctors are caught up in the same dehumanizing system that corrupts relations between patient and doctor.

We can help by asserting our humanity, insisting, even to our good doctors, that we want a seat at the table. We want real MPN patients involved in discussions of treatment protocols.  We want to participate fully in our treatment.  And when the rules governing clinical trials are on the table, we want fellow patients sitting there who have been through the process.

The clinical trial system has to change.  For starters it might take something as simple as reshuffling the deck and dealing a hand to a new player who pulls a chair up to the table.



Take me back to the Contents

©, 2014. Unauthorized use and/or duplication of this material without express and written permission is prohibited. Excerpts and links may be used, provided that full and clear credit is given to with appropriate and specific direction to the original content.


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