#drugprices : The High Cost of Quality of Life and How You Can Effect Change #socialmedia #activism

If you suffer from multiple sclerosis (MS), unless you have lived in a cave since 1993 when Betaseron, the first FDA approved disease modifying drug (DMD) came on the market, you are well aware that the cost of these drugs is astronomical. It seems morally wrong to put a outrageous price tag on a substance–capable of improving quality of life for those who suffer from this disease–only because people are desperate to get it.

Drug companies, when asked to defend their pricing strategies, quickly point out that the cost of research and development (R&D) is exorbitant, and only a small percentage of drugs ever win FDA approval after decades-long trials. If a drug wins that distinction it must pay for not only its own R&D costs, but for those that failed as well.

But there's much more that factors into how these drugs are priced.


I had the honor of speaking with one of the country's foremost experts on the topic of drug pricing, Dr. Kenneth Kaitin, a professor at Tufts University School of Medicine in Cambridge, Mass. We spoke for nearly thirty minutes on the topic of drug prices, how they are achieved, and the emerging power of social media to effect change.

He spoke to me about value, competition, patent life, and more. But he also spoke at great length about how patient groups, physician groups, and individuals are increasingly effecting change, enabled by the power of social media.

The interview was part of the research I did for the two part series of articles I wrote for Healthline.com:

Part 1: Should Multiple Sclerosis Drugs Cost $62,000 per Year?
Part 2: Voices For Change: How You Can Impact MS Drug Pricing


In short, the pharmaceutical industry is one that operates like any other. They depend on their cash cows–in this case the MS drugs–to be the stable, revenue-producing portion of their portfolio that allows them to invest in riskier, cutting-edge research that often yields astounding breakthroughs in medicine. Like it or not, it's the system we have.

To be certain, all drug companies now offer generous patient assistance programs, so the majority of those who want them have access to the DMDs.

If you need to be on a DMD but think they are cost prohibitive, be sure to ask your neurologist about the programs available to assist in covering the cost.

After writing the series I felt uplifted, empowered, and motivated to use my online presence to try and inspire others. There are so many people suffering from MS out there who are sitting at home feeling powerless, frustrated and desperate about their situation. They feel hopeless to change the course of their disease, or they can't afford health care to try.

In this age of social media, however, activism is only a keyboard away. If you want to make a difference, start by joining the National Multiple Sclerosis Society, encourage those at a local support group to do the same. Get involved in speaking out. Blog your hopes and dreams for the future of healthcare, send your congressperson or senator an email about your situation and your desire for affordable medicines and healthcare. Take a stance and use your voice.

Every little bit helps. I'm going to use my voice to educate others on personal empowerment and how to effect change.

Now go do something positive and never forget: One person CAN make a difference.

New Hope for ALS Patients - A Study with Gilenya

I think the scariest words a person could ever hear on this earth would be "You have ALS." I cannot begin to imagine the terror that must grip a person as their body stops responding to the brain's commands. To some extent, having multiple sclerosis, I have felt the fright of limbs that won't move or cannot feel.

Over the course of my disease I have had to use canes, a walker and even a wheelchair for brief periods of time. But, with a relapsing-remitting diagnosis, I have always had the luxury of making a nearly full recovery once the disease activity subsided between relapses.

Not so with ALS patients. Their disease marches on, unrelenting, unforgiving, robbing them of one thing after another. Taking away voluntary movement and confining the person to a prison within a body they can no longer control. The worst part is that there is no treatment to slow the slaughter of neurons.

I have always felt a strong compassion and sadness for those unfortunate enough to be diagnosed with ALS, feeling "there but for the grace of God go I." So when news of a possible treatment for the disease in Phase 2a clinical trials hit the wire this morning I could't wait to share it. The fact that it is my pride and joy, Gilenya, (TDI-132 aka: fingolimod, Gilenya™) that might make a difference in the lives of these patients leaves me smiling ear to ear.

http://www.prnewswire.com/news-releases/fda-approves-clinical-trial-of-tdi-132-gilenya-in-als-patients-190659561.html

It's my wish that amazing things are discovered and Gilenya can help lots of ALS patients live longer lives. More importantly, I wish them Quality of Life, because without that, living becomes just a prolonged wait for death.

Best of luck and Godspeed to all who enter the trial and to those who await the results.

A formal listing of the clinical trial can be found here:
http://www.als.net/ALS-Research/166/ClinicalTrials/

Information on why Gilenya might be helpful for ALS (pdf):
http://www.als.net/docs/uploads/Why_might_Gilenya_be_helpful_for_ALS.pdf

Out with the Old, In with the New

As most of you who have been following along already know, I'm a Gilenya Guide, or patient speaker for Novartis. I travel around the country and share my story with groups of multiple sclerosis patients to raise awareness of the disease and treatments available. I have agreed not to blog those experiences in detail, but I do like to share dates/places where I'll be speaking.

Out with the old

Last year was a great year for patient advocacy! I spoke around the country at Novartis events as well as non-Novartis events, advocating for those with MS as well as ePatients, clinical trial patients, and just patients in general.

It was a great start to what I hope will become an even bigger crusade in 2013.

The Disruptive Innovations To Advance Clinical Trials Conference  - September 2012

My Part @

The Disruptive Innovations To Advance Clinical Trials Conference  - September 2012

In with the New

I have several events scheduled already this year. As a Gilenya Guide, I will be speaking on the following dates:

This Saturday 1/12/13 in Tallahassee, FL:

Saturday, January 12, 2013
2:00 PM ET
Presenters:
Megan Barrett, ARNP, BSN
Jeri B
Lafayette Park Center
501 Ingleside Ave, Tallahasse, FL 32303

Tuesday, 1/15/13 in Glenview, IL outside Chicago:

Tuesday, January 15, 2013
6:30 PM CT
Presenters:
Catherine Meyer, RN, MSCN
Jeri B
Lalo's
1432 Waukegan Road, Glenview, IL 60025

In Atlanta on my birthday, 1/31/13

Thursday, January 31, 2013
6:30 PM ET
Presenters:
Robert Gilbert, MD
Jeri B
Brio Tuscan Grille Buckhead
2964 Peachtree Road NW, Atlanta, GA 30305

and I have 2 non-Novartis events coming in February and March:

One is in San Francisco on February 26-28, 2013:

eClinical Pharma Decision Making

The other is in Philadelphia, PA on March 20-22, 2013:

C.R.O.W.N.

2nd Annual Clinical Research Operations & Worldwide Networking

I can't wait to get this year going! It's going to be a great year for patients everywhere. I can feel it.

The Disruptive Innovations Event in London, as Seen From My Living Room

I was born into a generation that can remember when telephones had curly cords and rotary dials and the only color they came is was black because that's what color telephones were. We also got immense satisfaction from being able to slam the handset into its cradle, putting a physical exclamation point to the end of our conversation if the situation warranted it. When you dialed "0" you got a real live person. My Great Aunt Mary used to be a switchboard operator back when you had to ask them to dial the person you'd like to speak with. We always said that's where the stereotypical operator's voice with that nasally sound came from that you hear in old time movies because that's how she spoke. When she said my sister Lorraine's name it came out "Lo-ray-un".

So fast forward a half century. Things have changed. Phones no longer tether us to a wall or a counter or a desk. In fact, you can go into the middle of the nowhere with no person in sight for miles and still talk to someone because your phone is in your pocket. Always within reach.

Not only can you TALK to someone, but you can SEE them, too. If you plan on calling in sick to work you better look the part now and be phoning in your jammies from your death bed because it's not enough to sound sick from your beach chair.

Technology.

You can now address a roomful of people in London while sitting in your living room in Florida. I know because that's exactly what I did last week.

After my interview with Craig Lipset of Pfizer (see this post for how we met) at the Disruptive Innovations conference in Boston this past September, I did some networking and met many amazing and influential people. By way of Pablo Graiver , from www.trialreach.com/, I met Maneesh Juneja from www.health2con.com who reached out to me.

Because of my patient perspective on how clinical trials are conducted – and my willingness to share it – I've got something to say that folks in the pharma arena apparently consider worth hearing. It seems that while many of these folks have dedicated their very lives to research, most have not ever come face to face with an actual volunteer subject nor listened to them expound on how a trial might be better designed from the patient perspective.

I am a focus group of one.

So there I was, up on the big screen with my 10 foot tall head sharing the story of my clinical trial (the TRANSFORMS trial conducted by Novartis for Gilenya, the first ever pill form of treatment for multiple sclerosis). I told them how it came to pass that I found the trial, and then how I could find nothing on the internet describing a clinical trial from a patient perspective.

So I blogged it here. (There's no link because literally, it was this blog. Right here.)

I decided that if I couldn't find the info I was looking for, then I was going to document my journey and let everyone know I did it. And if I can do it, anyone can because we all know by now that I am the biggest hypochondriac that ever lived.

I ended my talk in London (while sitting at my computer with no shoes on my feet, curling my toes into the carpet) with a sort of laundry list of things I'd like to see change in the world of research. At least those things which effect the end user experience.

I figured if I was going to be talking about how things needed to change, the least I could do was give some examples of exactly what I meant by that.

First off, that Informed Consent document (that is almost as thick as my whole set of 1960's World Book Encyclopedia that's all swelled up from having gone through a flood) needs some companion booklet to go with it. Sort of a chaser to help smooth out that bare whiskey dose of reality (and a whole lot of possible negative outcomes) that comes with it.

The informed consent document has to outline the purpose of the trial, the known side effects of the medication (everything they ever found out during earlier, even unrelated, trials), whatever adverse events might be predicted along with a very clinical description of the tests that will be performed to monitor the safety and efficacy of the drug along the way. The positive, upside of what they HOPE will happen seems like a byline or afterthought. Possibly a footnote¹.

I propose a user friendly, brochure type of Guide to the Clinical Trial that tells you, in terms normal people can understand, what to expect. A brief outline of the goal of the study, along with pictures of the equipment they will use to test you, and pointers about making the tests go more smoothly (like drinking lots of water before a blood draw). Just something like a Chamber of Commerce version of "Welcome to the Trial".

It might sound silly but I insist it would have been a comforting thing to read after Googling the guts out of all the stuff I deciphered from the Informed Consent.

I also told them that the people who enter the trial, unless they are the healthy control subjects who are in it for the profit, are usually at their wit's end. Looking for relief from their illness and hanging whatever shred of hope that remains on this new drug I can't get anywhere else yet. And their biggest hope is that they aren't wasting their time taking a placebo. They are at their most vulnerable and need to be treated as the humans they are. With respect and  compassion.

I won't go into everything I suggested. I'll save that for another day. Let's just say I was well received and felt so honored to be asked to participate in this Health 2.0 event.

I still can't get over how I have tripped and fallen head first into the role of ePatient Advocate. I hope I can continue to represent for ePatients, clinical trial patients, plain old patients, and humans everywhere.

A big thank you to Maneesh and www.health2con.com for giving me a platform from which to share my message.

I'm usually so excited about the advancements in technology and how rapidly they are coming out with new gadgets like top quality webcams, but this time I would have loved for my talk to have necessitated a trip to England. Ah, the down side of Skype.


¹ Just an example, not a bona fide footnote.