Science & Medicine

The forgotten MPN Patient — Proliferative conferences, an editorial

MPN patient education conferences: Their surprising role in drug development from bench to market.

MPN patient conferences are fun.They’re a front row seat to medical history, a chance to fit faces to names, hook up with old friends, and learn a thing or two.

The major events are much more than that. They’ve become integral to the process of drug discovery, funding, testing, and marketing. They’re like the Super Bowl of MPNs….plenty of action on the field but a lot more happening during commercial breaks and color commentary.

In the January MPNforum Magazine News Briefs we report on a new MPN foundation organized to further patient education. Newly diagnosed patients — particularly the poor and those without easy access to the Internet — are surely in need of education. But they are not the target. MPN patient conferences by definition reach those who can afford it and have the computer access to learn about it. MPN patient conferences and foundations are only marginally involved in filling the real need for grass roots education.

MPN patient conferences do, however, fill an important space between the scientist’s workbench and the physicians’s prescription pad.

We need to reposition these events in our minds and recognize the role they play in the very high stakes game of bringing Jakafi to Sam…and then turn our attention back to the crushing need to provide basic MPN information to all.

MPN patient conferences are just not what they used to be. In a sense, they are much like a Trade Fair, a gathering of manufacturers, suppliers, contractors, sub-contractors, and customers. Principal investigators and their institutions are contracted by drug companies to undertake and report on clinical trials that might yield products for us, their customers.

Conference participants, seen in the context of these high stakes events, are available to drug companies at cut-rate prices. The $5,000 sponsorship contribution made by Incyte, for example, to one such event amounts to less than the cost of a single month’s Jakafi supply for one MF patient.

MPN patient education conferences weren’t like this in the beginning. Then it was just Harriet Gilbert reaching out to her MPN patients suffering these rare, under-publicized myeloproliferative diseases. Her e-mail list of patients became the basis for a conference to educate patients, bringing together hematologists and patients in a day long event. Some of that still exists within the patient support groups and in theUK.

Claire Harrison of Guys and St. Thomas’ who set up the non-profit MPD Voice ( ) said ” We have been running short evening forums for six years now all over theUK. There is a clear appetite for these and we have an increasing audience….In most meetings newly diagnosed patients have come and have always fed back how helpful they found the meeting and sense of community. Clearly people want to come and return repeatedly. We have never charged for these.”

That hasn’t been the American model. During the 15 years post-Harriet Gilbert, MPN patient conferences proliferated and morphed into something entirely different.

Now that we are in the full conference season — with no fewer than four separate foundations or non-profits hosting MPN patient education events — let’s ask ourselves, what is actually served by these events that bring together patients with (usually) famous physicians, to hear news of the latest drug approvals, scientific breakthroughs, and clinical trials?

As MPN patients, we are the consumers of these products, the drugs and services. And we are participants in clinical trials when we believe we have no other, better options and frequently — usually – derive little benefit from the trial and risk suffering further deterioration of health.

Patients don’t write prescriptions, discover genetic anomalies, and neither fund nor approve clinical trials. So why fuss about us? The answer may be found in the drug development and approval process.

When bench scientists discover a genetic anomaly, uncover the molecular structure of a proliferating cell, or decipher the biochemistry of communication and transcription that results in myeloproliferation, they may have uncovered a vulnerability to be exploited by drug developers.

The next steps require testing before turning that hypothesis into a drug.

Proof of the hypothesis is found in a controlled Phase III clinical trial – one where a matched cohort of patients is recruited, some get the drug, some don’t, and results are compared. We provide the pool of recruits to form that cohort.

Before that can happen, an earlier step is required. In phase I/II trials, the dosage and toxicity of a drug are explored by simply giving it to patients and escalating dosage until a clinical response or other intolerance is obtained. We are those patients. In rare cases, when a drug demonstrates sufficient efficacy with acceptable toxicity, it is given the green light for phase III clinical trial. If that trial is sucessful and an FDA seal of approval obtain, the drug is released to market.

There are a host of professional, semi-professional, and commercial events required to support the drug’s candidacy and entry into the distribution stream.

Scientific papers are submitted to journals and, in an occasionally flawed peer-review process, are published. Posters and abstracts are presented at annual meetings of prestigious societies and results discussed and debated. We’re still in the realm of science…. Not quite bench science, not pure laboratory science since we’re approaching applied science, that gray area where the whirr of the gene sequencer meets the ka-ching of the cash register.

The push-pull of marketing is charged with bringing the drug to a pharmacy near you. The push is provided by drug company-sponsored promotion of its drug and the pull by patients and their physicians demanding the product. We are the patients providing the pull.

Sometimes the whole process from bench to market works seamlessly. For years, we have watched, participated in, the unfurling of the JAK inhibitors, focusing lately on Ruxolitinib (Jakafi) , and are now happily seeing our friends and loved ones receiving the drug (at $7000/month) anticipating their miraculous recoveries, their splenomegaly reductions, the quality of life improvements we have seen reported and, in some cases, witnessed.

The MPN patient educational conference is in this arena. It is one significant area where word of discovery gets out, recruitment for clinical trial begins, patient drug demand is born.

It’s a limited arena, limited to those educated and affluent enough to afford the computer/internet hookup that advertises the event, the fees, the travel, lodging, time off work and all that. Still, 200 or so patients show up at each conference, meet each other happily, take notes, photos, and participate in discussions with doctors and other patients. Multiply those 200 by their network of on-line support groups and Facebook friends and you have a significant chunk of an MPN consumer group, the opinion leaders.

But is it an educational arena?

In Harriet Gilbert’s time, there was no broad-band internet, no Google, no international network of MPD patients. The JAK-2 mutation had not yet been discovered, the human genome had not yet been sequenced. 15 years ago, there was a real need for us and our hematologists to come together to learn and to share our knowledge with one another. That original need has morphed into a cottage industry with multiple purposes, the least of which appear to be education

The participation of principal clinical investigators representing the most renowned medical institutions is hardly necessary to educate patients. We have excellent YouTube videos. We have access to articles, both popular and scientific via Google, medical websites run by clinics, drug companies, foundations and the Federal government crammed with this information. And we have our own doctors and on-line patient forums where we daily exchange ideas and information.

Yes it is inspiring to have world class hematologists and researchers volunteering their time to answer questions in work sessions. Many of these men and women have devoted their lives to studying our diseases. But it’s nonetheless troubling when principal investigators present PowerPoint slides of their drug study findings — in carefully qualified terms — to unqualified patients who might well need those same drugs. It is doubly troubling when sponsoring drug companies sit in on the sidelines.

The American MPN Patient Conference is no longer an educational vehicle for patients, at least not primarily.

In the United States, the MPN Patient Conference today is a social and educational event, a sales fair for drug companies, a public platform for medical institutions, a recruitment tool for clinical trials, a subtle stock promotion event, and a PR and fundraising function for sponsors.

There is another model. The Living with MPDs conference held a few weeks ago inLondon, sponsored by MPD Voice, has elements of the original educational thrust of such gatherings. For one thing, fees were an affordable £10. And “it was timed so patients needn’t stay over night and was (located) adjacent to some of the main rail links inLondon,” says Harrison. Significantly, the event included strong patient-led participation in a scheduled session.

In the January issue of MPDforum Magazine two patients who attended that London event present radically different views of the day.

Available: December 15, on-line at

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