BannerFans.com
« »
Sep
16

FDA gives Terminally ill Population the Shaft: the Sordid History of Iplex Access and Lou Gehrig’s Disease

By J. Edward Nelson
New York Times - ALS patient receives firsst injection of iplex in March 2009

New York Times - ALS patient receives firsst injection of iplex in March 2009

 

In a society which subsidizes the cost of prescription drugs for the rest of the world by being charged substantially more for pharmaceuticals THAN ANY OTHER COUNTRY, you would think that terminally ill patients in America suffering through debilitating diseases which have no cure, would be afforded the unfettered option of being prescribed any therapeutic approved by the Food and Drug Administration (FDA), which they and their doctors mutually agreed upon.

I mean just think about it, if it’s a virtual certainty that a particular patient population is going to die in the short term, and the FDA has already subjected a drug through all three phases of a clinical trial, finding it to be both safe and well tolerated within another patient population, then why deny this dying population access to that drug and the one thing which understandably dominates their collective consciousness? Hope.

Unfortunately, this nightmarish scenario is currently being endured by American patients living with Amyotrophic Lateral Sclerosis (ALS), more commonly referred to as Lou Gehrig’s disease in the U.S. after the hall of fame baseball player who died from it in 1941.

 

ALS is a neuro-degenerative disorder (ie, progressively disrupts the brain’s ability to communicate with voluntary muscle groups) that has no cure, which wreaks havoc on a person’s ability to do things which most people take for granted such as: walking, talking, swallowing, eating, breathing, taking a shower or simply rolling over in bed. ALS patients rapidly progress toward complete paralysis and death from respiratory failure within 3 – 5 years from diagnosis.

The only drug specifically approved for ALS is called Rilutek, which does not slow disease progression or help patients with ALS (referred to as “PALS”) regain their severely compromised motor function.  But simply extends life for up to three months.

Ninety additional days of COMPLETE PARALYSIS may sound a bit more like torture to some rather than anything remotely therapeutic. Sanofi-Aventis, the manufacturer of this drug, has a more “pragmatic” view of Rilutek as it represents $150 million in global revenues per year.

“Iplex” Buzz

Over the last few years, a drug called Iplex has produced a significant buzz within certain segments of the ALS community. It was approved by the FDA for children with short growth stature in December 2005, but was prescribed off-label in 2006 and 2007 to PALS in the United States with promising “anecdotal” results.

Rumors that these U.S. PALS, in a short period of time, were able to improve from eating pureed foods to normal adult meals, gain weight, increase breathing capacity, as well as regain some of their hand and foot motor function – UNHEARD OF THERAPEUTICALLY IN ALS PATIENTS – motivated PALS in Italy to successfully petition their government, through the Italian legal system, for access to Iplex.

From January 2007 till the present, as many as 100 Italian PALS have received Iplex from its manufacturer, Insmed Inc., based in Richmond, VA, through an expanded access program also referred to as “compassionate use.”

Access Denied

In a country where 4.4 billion prescriptions are written annually at a cost of over $1 trillion, PALS in the U.S. are being denied access to Iplex which is currently on the FDA’s list of approved drugs. Given the ultra-liberal government attitude toward pharmaceuticals as constituting the standard bearer for treating illnesses in America, the brief history of Iplex availability and U.S. PALS is quite perplexing to say the least.

A settlement agreement between Insmed, Tercica and Genentech regarding patent infringement litigation – Tercica, acquired by the Ipsen Group in 2008 for $404 million, manufactures a similar drug called “Increlex” approved by the FDA in August of 2005 (3 months prior to Iplex) – resulted in Insmed discontinuing the marketing of Iplex commercially as of March 2007.

Once their supply of Iplex was exhausted, those PALS in the U.S. who had been taking the drug off-label “reportedly” reverted back to their pre-Iplex conditions within 3 – 4 weeks.

The late Ben Byer, ALS patient and director of the award winning documentary film, “Indestructible,” commented on being forced off of Iplex and subsequently taking Increlex as being the equivalent to, “trying to get drunk on cough syrup when there’s a case of bourbon locked in the closet.”

Although Insmed continued to provide Italian PALS with Iplex through the expanded access program, doctors in the U.S. could no longer prescribe it off-label even though its status as an approved drug HAD NOT CHANGED.  The settlement agreement prevented Iplex from being commercially marketed in the European Union, the U.S. and Canada SOLELY for those indications which Increlex was being marketed, ie, short growth stature, which did not include contra-indications (off-label uses) such as ALS.

In the United States, drug manufacturers cannot actively market off-label indications to the public.  But they can promote them to doctors even if they have pending new drug applications FOR THE VERY SAME INDICATIONS, according to FDA guidelines. (Some within the medical community view the regulatory agency’s role in this regard as being too lenient).

When juxtaposed with the fact that 65 million prescriptions are written annually in the U.S. for thousands of drugs which ARE NOT approved by the FDA, as the result of a “regulatory black-hole” which the agency has been well aware of for decades, it is quite the proverbial “slap in the face” to PALS and their doctors in the U.S. to be denied off-label access to a therapeutic actually approved by the FDA.

Efforts by PALS, such as a demonstration in Washington D.C., resulted (some believe) in an amendment to the settlement agreement giving Insmed the right to supply Iplex worldwide to ALS patients in expanded access programs, royalty-free. Many PALS were excited by this development but in retrospect, it more closely resembles a “red herring” because the original settlement agreement DID NOT LIMIT INSMED FROM SUPPLYING IPLEX TO ALS PATIENTS, nor could it.

No company currently holds a patent for insulin-like growth factor-1, the active ingredient in both Iplex and Increlex, for the ALS indication.

PALS were then instructed to apply to the FDA for compassionate use of Iplex with the assistance of their doctors as if it were an investigational drug, and not a drug which had already survived the rigors of the approval process through the agency.

Initially, all requests were denied even though the FDA has a policy to approve VIRTUALLY ALL COMPASSIONATE USE REQUESTS submitted by terminally ill patients, supported by both the treating physician and the drug manufacturer.

The FDA then announced, out of the blue, on March 10, 2009, that only requests received by March 6th would be approved. I personally remember that date because on March 2nd, I met with my ALS doctor and he declined to assist me in applying for Iplex.  His rationale being that he did not think it would slow disease progression, even though he had prescribed Rilutek a year prior. I reminded him of that and he readily admitted that Rilutek didn’t slow disease progression either, and he regularly prescribed drugs off-label.

Suffice it to say, that was the last time I saw him.

Twenty applications were received by the retroactively imposed deadline, although two of the twenty applicants had died prior to the announcement. The FDA also announced that Insmed would conduct a Phase II Iplex clinical trial for PALS selected through a lottery system later in the year, which Insmed postponed indefinitely in July. Insmed also sold the facility it used to manufacture Iplex along with its follow-on biologics platform to Merck for a reported $130 million.

This chain of events has resulted in extremely limited access to Iplex for ALS patients for the foreseeable future, numbering in the dozens who have been afforded access out of a worldwide ALS population of approximately 300 thousand.  Which for many PALS will likely translate into their being denied the option to even try this particular drug, as the result of their shortened life expectancy.

Even though there is no definitive clinical evidence to support the claim that Iplex reverses or even slows the symptoms which characterize an ALS diagnosis – only anecdotal evidence largely associated with the American PALS who were the first to take Iplex off-label – American PALS SHOULD NEVER HAVE BEEN DENIED ACCESS TO IPLEX, and the explanation as to why is fairly simple.

The restrictions on the sale of Iplex in the U.S. were outlined within a legal document, during the coarse of a civil proceeding, crafted by agents of Insmed and two other corporate interests (Tercica and Genentech), not the FDA or any other regulatory agency in the U.S. 

The dispute between the three parties was a “civil” matter, NOT AN ISSUE OF REGULATION.

If any of the three had consciously or unconsciously broken the terms of the agreement, possible remedies might have included seeking monetary restitution or a cease and desist order through THE COURTS. Otherwise, Tercica and Genentech, who initiated the patent infringement lawsuit, would have sought a resolution directly from the organization that caused the dispute to begin with, THE FDA.

Insmed was never ordered to stop manufacturing Iplex, or supplying it to Italian PALS.  Nor was the company prevented from being reimbursed millions of dollars by the Italian Ministry of Health through the expanded access program.  So why would the FDA prevent off-label prescriptions, or force American PALS to apply for compassionate use of Iplex, when 1 in 5 prescriptions written by doctors in the United States, or 880 million, ARE OFF-LABEL?

Given the “pro-drug” culture in the Washington D.C., with even unapproved prescription drugs being advertised in The Physician’s Desk Reference, and minimal (if any) ramifications for these blatant violations of federal law, the actions of the FDA with regard to Iplex simply does not follow suit with government policy toward the pharmaceutical industry since the early 1980’s, ie, DE-REGULATION.

Government Interference

In my opinion, the Iplex-ALS saga is a prime example of government intervention within decisions which should be made between doctor and patient. Particularly when the FDA had already fulfilled its due diligence by giving the drug a “clean bill of health.” It is tantamount to government deciding how doctors practice medicine. This is precisely the type of heavy handed governance which many Americans fear will escalate if government controlled health care coverage is substantially expanded.

I also believe that the case can be made for American PALS, family, friends and organizations having caused serious injury to PALS throughout the rest of the world by not being more vigilant in gaining access to Iplex, because fortunately or unfortunately, other countries with corresponding patient populations are directly effected by precedents of policy set in the United States concerning access to pharmaceuticals.  As well as therapeutic innovations which stem from off-label use of prescription drugs.

When terminally ill populations in America get the “shaft,” the consequences can literally reach around the globe. When “hope” is taken off of the table through limiting avenues of medical innovation, the will to PERSIST within certain patient populations is sorely tested.

 

  • Share/Bookmark

If you enjoyed this post, make sure you subscribe to my RSS feed!

Blog Traffic Exchange Related Websites
  • FDA Approves Continuation of ALS Stem Cell Trial Emory University researchers have received approval from the Food and Drug Administration (FDA) to advance to the next phase of a landmark trial to treat patients with Amyotrophic Lateral Sclerosis (ALS) using human neural stem cells. The Phase I trial, currently underway exclusively at Emory University, is designed to assess......
  • Reblog this post [with Zemanta]PluristemBegins Enrollment in U.S. With Unmatched Placenta-Derived Stem Cell Product PLX-PAD in a Phase I Clinical Trial for Treatment of Peripheral Artery Disease Pluristem Therapeutics Inc. today announced that it has begun enrolling patients in the U.S. for a Phase I clinical trial with its unmatched placenta-derived stem cell product, PLX-PAD, the Company`s leading product candidate for the treatment of critical limb ischemia (CLI), the end-stage of peripheral artery disease (PAD). Enrollment began......
  • the placebo effectThe Power of The Placebo Effect - How Can 35-45% of People Cure Themselves? How Powerful is the Placebo Effect? One of the most confounding things for modern, medical science to explain is called the ‘placebo effect’. A placebo is a sugar pill administered to a test patient who believes it’s an actual drug. This power behind concept has been used by doctors for......
  • Chart of the Day - Nonfarm Payroll Jobs Lost to Outsourcing & Productivity Improvements Since 1940s WWIIMiddle Class Job Erosion & Mind Blowing Job Loss Statistics Gee, anyone else see a long developing trend, followed by a step off the precipice here? Image via Chart Of The Day When people begin to wonder why the rest of the world is catching up to the U.S, and why our middle class is shrinking, you must remember that......
  • Blogs are not about dirty laundry; they\'re about proper positive public relations.Corporate Blogging Precautions Whenever you decide to get into corporate blogging, you should be aware that there are some precautions you will need to take. Here are some guidelines to help you avoid disaster and create a very successful blog. 1. Never air your company’s dirty laundry. If you are frustrated about a......
Categories : Business, Health, Politics

Comments

  1. Get Ripped says:

    Incredible work on the blog! :) I wish blogging was easy as getting ripped was for me! :(

  2. J. Edward Nelson says:

    Well, you could blog about ”getting ripped.”

Leave a Reply