Science & Medicine

TSR- Autumn 2015


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dots cml cropped  Is interferon safe for everyone?  It might depend on whether you’re JAK positive or negative according to findings in a new study by Dr. Richard Silver and his associates published in Modern Pathology  Silver and his associates compared before and after tissue samples of 12 myelofibrosis patients who had been on interferon therapy for periods ranging from 1 to 10 years.

They looked at bone marrow biopsies as well as clinical and lab data. They used standard measurements to determine clinical response and prognosis before and after treatment. The good news is interferon, pegylated or not, contributed to improved or stable clinical response for most (83%) of patients.

The surprising news:  Regardless of clinical results the prognosis for all JAK2 negative patients was worse following interferon therapy, one evolving to AML.

Before tossing out your pre-filled syringes, recognize this was a small study. The full mutational status of patients was not known and clearly more work has to be done on larger cohorts of patients to confirm or refute these very preliminary findings. Moreover, Dr. Richard Silver himself does not believe the sample size or analysis supports a position one way or another,

But isn’t it chilling to read: “JAK2V617F-negative patients were invariably associated with a worsening of the score or evolution to acute myeloid leukemia.”  Can’t just shrug it off.  These are findings in a long-term retrospective study undertaken by specialists in both interferon and hematopathology  published in a peer-reviewed journal.

“In conclusion,”  write the authors, ” …. Our results also highlight a possible role for JAK2 mutational status in the prognosis of interferon-treated myelofibrosis….”

After this study – and until further clarification is reported — it seems reckless for a patient to start interferon therapy without first determining JAK2 mutational status.  At the very least, it’s an indication for hematologists to determine  JAK2 status before prescribing interferon.

finger pointing small  Note: Dr. Silver is not the only one to believe it’s premature to act on these findings.  Dr. Srdan Verstovsk said “…to make treatment decisions or make projections for the future based on a study in 12 patients is not good.”

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dots cml cropped  Sunshine Act reveals “Dollars for Docs”….while drug companies remain in the shadows. Are our doctors, medical researchers and teaching institutions corrupted by money paid out by drug companies?   It’s a question the  Physician Payment Sunshine Act, part of the Patient Protection and Affordable Care Act, was created to address.Dollars for Docs Does the shower of gifts, imprinted office pens. consulting fees, meals, travel, lodging and multiple other perks traditionally rained down on the medical profession by the cash rich pharmaceutical industry influence medical judgment?

It’s a fair question.   The murky Sunshine Act response may be well-intentioned…but radically misses the target.

It’s a classic example of Ready. Fire. Aim!  A lot of smoke and noise… but not even close to the target.

Yes, there’s cash and valuable exchanges paid directly to doctors by drug companies.  

And Pro Publica’s Dollars for Docbrings the Sunshine Law’s findings to us.  You can type in your favorite doc’s name and up pops the total he or she was paid, the source of payment,  plus ranking at the pharma trough.  Problem is the abundance of trivial detail plus the lumping of payments  together in broad categories inevitably produces a foggy and distorted view of what’s going on.  Some of the allocation of payments — like third party editorial services — are chalked up to marketing and promotion while payments to institutions are often made in the principal investigator’s name. swelling the doctor’s apparent total receipts.

There’s no context, no distinction made between an MPN specialist consulting on clinical patient needs with a drug company product development team and a non-participating doctor on the consulting payroll .

No doubt there are some abuses and questionable activities like those  physician-featured road shows on the patient circuit in support of one drug or another. But overwhelmingly hematologists are not personally enriching themselves at patients’ expense.     Over many years of working with our doctors, it’s clear virtually all  are relatively underpaid, highly committed to patients and severely overworked.   Focusing on gross payments to them without needed context does a disservice to patients and doctors alike.

The bigger problem with this Sunshine Act debut is the distraction factor This focus on payments to physicians distracts us from addressing the cancer that has locked on to the American medical system, the unregulated, uninhibited greed of pharmaceutical companies expressed in extortionate drug pricing.

Even the little bit of Sunshine splashed on the drug companies in the current publication of data is useful in revealing Big Pharma’s  massive investment in non-research direct payments to institutions and physicians.    It just doesn’t go nearly far enough.  .

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“In 2014 the pharmaceutical industry spent over $250 million on lobbying and campaign contributions — far more than any other industry in America,” reports Senator Bernie Sanders.

And there is no Sunshine law requiring drug companies to disclose the real costs of research and development, the percent of those costs covered by taxpayers, the price of these same drugs in other countries, like Canada  where, on average, the identical drugs cost 40% less. Nor is there any law prohibiting drug companies from  preventing lower generic drug prices through acquisition and financial incentives.

The greed of this industry has produced swollen CEO compensation packages and high returns on stockholder investment. Yes we have $100,000 pills, the world’s most high tech diagnostic equipment, and extraordinary capabilities to extend human life. But when it comes to delivering basic health services, America is encumbered with the most expensive, least effective basic health care system of any developed nation. Beyond high deductibles and co-pays for drugs — Medicare/Medicaid patients have a 33% co-pay for a Tier 5 drug like Incyte’s Jakafi. — and the reluctance of insurance companies to approve high-priced drugs to contend with.

The most shameful statistic underscoring the deadly impact drug pricing has on American health comes from the U.S. government itself:  Nearly 1 in 10 American adults don’t take their medications as prescribed because they can’t afford to, according to the U.S. Centers for Disease Control and Prevention’s National Center for Health Statistics.

More on the Physician Payments Sunshine Act … from ASCO and the CMS. Center for Medicare and Medicaid 

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dots cml cropped  Remember her?  And all those fatigue surveys we filled out?

 She was the poster girl tiredfor the start of the Fatigue Project Back then all we had was the desire to know more, to get help.   The Mayo Clinic crew, headed up by Dr. Ruben Mesa and Dr. Robyn ScherberRobyn Emanuel with Ruben Mesa jumped into the breach. They took our desires and crude data and designed a series of powerful research instruments that have disclosed the face of MRF — MPN-Related Fatigue — and set us on a path to get some relief.

Posters of MRF findings were presented  at the last two ASH meetings and several important papers have appeared in scientific journals.  This work put MRF on the hematology map and called attention to fatigue…. the primary unaddressed symptom of MPNs.

Cancer is the latest peer-reviewed journal to approve publication of the results of Comprehensively Understanding Fatigue in the Myeloproliferative Neoplasms, (Scherber et al.)   Congratulations to all… including the nearly 1800 of us who participated in the on-line research.

divider horizon linedots cml cropped So now we know what Promedior was up to… You know that long delay between results of the PRM-151 Phase I clinical trial and the start of Phase 2? We were told it was about crunching data. About applying findings from the first trial that showed some positive clinical results. What we weren’t told was little Promedior was shopping for dollars…and a big partner. Last week they announced it. Giant Bristol-Myers Squibb is on board with a $125 million buy-in giving them the rights to acquire Promedior. The deal is structured so more than $1 billion can change hands in  milestone payments and outright acquisition of Promedior and its fibrosis reducing drug, PRM-151.  That drug  has been granted Fast Track designation in the U.S. and Orphan designation in the U.S. and Europe for the treatment of MF.

divider horizon linedots cml cropped   Such big fish, such a small pond.  With Bristol-Myers now joining Novartis,  Roche, Celgene,  Gilead and Janssen backing an MPN drug, five of the top 1o drug companies with combined sales in excess of $15 billion  are paddling around our little backwater  swimming hole.  Makes you wonder.  You know there are not enough of us to justify all that action, even with the bizarre and rapacious pricing currently in vogue.  Like former Incyte CEO Paul Friedman suggested way back when all this started, myelofibrosis is just a stop on the way to bigger things. That’s OK, we’ll take the research explosion any way we can get it.

divider horizon linedots cml cropped    The bi-annual CR&T  MPN  Patient Symposium is just around the corner, so if you haven’t already got your ticket time’s running out.  CRT 2015 posterNew wrinkle this year: The patient symposium includes the big international scientific weekend conference.  The line up reads like a Who’s Who of MPN science and medicine... Among the presenters are Drs. Robert Kralovics,  Ann Mullally, Jerry Spivak, Ruben Mesa, Srdan Verstovsek, Richard Silver, Andrew Schafer, and Jason Gotlib.  The full program is here.

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