ImetelChat

Again, no claim to any expertise here but since most questions are okay on ImetelChat, I have significant concerns about statements by some that only patients with MF that are R/R to Ruxolitinib (Jakafi/Jakavi) will likely be considered by health insurance companies for coverage of Imetelstat once it is approved out of the current CT.

Knowing, as MedTechBio has stated and again in his latest SA article, Jakafi is discontinued in more than 50% of patients with myelofibrosis within 3 years due to serious adverse events, loss of response, or disease progression. Yet here we are with MF patients having to endure possibly for years on average before they can treat the disease versus just dealing with the effects.

How can waiting several years before being insurance covered for Imetelstat be an intelligent health care strategy?

Yes, coverage would come in years hence with the successful completion of a frontline clinical trial, more special designations, another NDA, etc. Is that the one and only answer for most patients given typical FDA/EUA process?

If that is the case, we are here in this seemingly ironic situation of still keeping the curative powers of Imetelstat from thousands who could use the drug now even though it is widely agreed that the disease will continue to progress while being treated with Jakafi? Can it, and if so what will it take for this incredibly convoluted logic to be overcome with sanity and consideration for human lives ending before their time?

Not to mention, how would a new (frontline) MF patient feel about the fact that Imetelstat is on the market but likely not available to them under their insurance coverage for years? Or even a MF patient on Jakafi, who can detect the gradual slippery slope they on but not declared "medically R/R" yet but knowing Imetelstat can provide considerable relief and OS beyond their current treatment option but not until they are considerably more seriously ill? Can the system be any more ridiculously "rigged against" common logic and decency than this?

Yes, I do realize much has to happen yet for Imetelstat to just be available to R/R patients. I personally believe the successful availability of Imetelstat for all MF patients is just a matter of time. However, so many will not live that long given the complexity of showing efficacy, safety, etc. endlessly while many friends and family die in front of everyone.

Given the new information associated with the earnings announcement, the CT processes are moving along. So hopefully we can soon see Imetelstat available to R/R MF patients and covered by insurance plans.

But, given all the information available to the FDA, including information based on various mutation responses, is it unreasonable to hope for / expect that some forms of provisional approvals will be made for “all MF and MDS” patients coming out of the primary analyses completing in 2-3Q2018 while CTs continue on and new ones begin? This seems to be the “right” thing to be considered and actually done by the FDA and would give renewed hope to thousands waiting on death row for help.

And as most of us are investors in Geron as well, expanded coverage by insurance plans would be another boost to revenues, etc.

Hunt, I think it won't be hard for insurance companies to analyze saved hospital costs (fewer blood transfusions, longer life, less anemia, no JAK meds etc--don't know about impact on hospice/end of life effects--but you can be sure actuaries are at their calculators. What they will find it Imetelstat will be very cost effective compared with current treatments. They will be lining up to pay for it. bp

Thanks for the encouragement Biopear, I hope you are totally right about it!

From what I’ve read, the practice, called "off-label" prescribing, is entirely legal and very common. The FDA regulates drug approval, not drug prescribing.

However, it probably isn’t as easy as one might think, and I hope I am wrong, especially in areas of insurance coverage and various risks. With older people dealing with serious illnesses like cancer, seems to me they will likely need / want an advocate to help navigate.

In reading the articles I now realize another value of various research articles in helping support off-label drug use, is they actually might be required and help pave the way for a drug to be considered and insurance covered for an off-label use.

I’ve looked at several articles to help become knowledgeable in approaching the challenge but it could be more uphill than one might expect. Seems to me, anyone suffering from MF may want to have discussions with their doctor and advocate on how to navigate prior to the R/R approval. It also may not be something an oncology doctor wants to be encumbered with supporting.

Some excerpts and associated links follow:
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Guideline
Providers may request coverage of off-label usage. Supportive data substantiating usage must be submitted to the Plan (see pp. 3–4 for documentation requirements); the data must include:
1. A use supported by clinical research that appears in at least 2 randomized controlled trials on large patient groups (e.g., 300–3,000 or more depending upon the disease/medical condition studied) that definitively demonstrate safety and effectiveness. OR

2. If no evidence is available (per # 1), at least 2 trials with reasonably large patient samples (e.g., 20-300) showing consistent results of safety and efficacy. Greater consideration will be given to higher powered studies with levels of evidence in the following descending order: Randomized or nonrandomized controlled trials, prospective cohort studies, retrospective case control studies, cross sectional studies, surveillance studies, consecutive case series, singe case series. Studies submitted for Plan review must come from different centers and be published in national or international peer-reviewed journals (with editorial committees comprised of physicians). Note: Peer-reviewed medical literature includes scientific and medical publications. It does not include inhouse publications of pharmaceutical manufacturing companies or abstracts (including meeting abstracts). OR

3. A use that is an accepted standard of medical practice. “Are there published recommendations from specialty societies or in other authoritative evidence-based guidelines” (e.g., a state-of-the-art review article published in a recognized textbook or a reputable publication)?

Note: Acceptance by individual health care practitioners, or even a limited group of health care practitioners, does not typically indicate general acceptance by the medical community. Testimonials indicating such limited acceptance and limited case studies, distributed by sponsors with potential financial conflict of interest in the outcome, are not sufficient evidence of general acceptance by the medical community.

The broad range of available evidence must be considered and its quality must be evaluated before a conclusion is reached. After such evidence is received, the Plan will, with the appropriate guidance of specialty-specific consultants, as indicated, make a coverage determination for the off-label use of the drug or biological. The Plan may determine usage to be reasonable and necessary for the treatment of illness or injury if, on the basis of available or presented evidence, it is shown to be safe and effective and does not violate national (or in the case of Medicare members) local Medicare determinations and regulations. The approval will include, but is not limited to, diagnosis, dosage, route of administration, duration and frequency of administration and appropriate patient population.

https://www.emblemhealth.com/~/media/Fi ... proved.pdf

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The biggest problem is getting insurance plans to pay (reimburse) for off-label drug use. Many insurance companies will not pay for an expensive drug that’s used in a way that’s not listed in the approved drug label. They do this on the grounds that its use is “experimental” or “investigational.”
In cancer treatment, these issues have been largely addressed through 1993 federal legislation that requires insurance to cover medically appropriate cancer therapies. This law includes off-label uses if the treatment has been tested in careful research studies and written up in well-respected drug reference books or medical journals. In 2008, Medicare rules were changed to cover more off-label uses of cancer treatment drugs.

Still, the health insurance coverage laws and regulations are complex. If your doctor is thinking about off-label drug use, you and your doctor should carefully check your health plan’s coverage. If you are denied coverage, it might help if the doctor sends the insurer copies of peer-reviewed journal articles or other respected sources that support the off-label use.

Another problem is that off-label drug use often does not reflect “standard of care” treatment. This could raise concerns about the legal risk to the health care provider should a patient have an unwanted or bad outcome from the treatment.

One of the biggest problems related to widespread off-label use is the lack of information about how to best use the drug other than for what it was approved. The drug label is one of the most reliable and easy-to-find sources of information available to health professionals, caregivers, and patients. But the label can only contain the information that’s been approved by the FDA, and it does not cover off-label uses.
Here are some questions you might want to ask your doctor. Start by asking if all the drugs recommended for your cancer treatment are approved for the planned use. If any of the drugs are not, you can ask:
• Is there evidence to support the off-label use of this drug to treat my type of cancer?
• Is this off-label drug likely to work better than an approved drug?
• What are the risks and benefits of off-label treatment with this drug?
• Will my health insurance cover off-label treatment with this drug?
• If my treatment involves a combination of drugs and one of the drugs is being used off label, will my health insurance cover it?

https://www.cancer.org/treatment/treatm ... g-use.html

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Medicare Part D covers drugs prescribed for off-label use only if the drugs are identified as safe and effective for that use in one of three officially recognized drug compendia. "Compendia" are encyclopedias of drug chemicals, with information on dosage and usage. Congress has given Medicare the authority to revise the list of approved compendia as appropriate.[12] Unfortunately, the burden of proving if a particular Part D-covered drug has been included in the compendia falls to the Medicare beneficiary, and accessing these compendia can be more complicated than might be imagined.

http://www.medicareadvocacy.org/cma-rep ... -drug-use/

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A couple other links of interest to me were:
https://www.webmd.com/a-to-z-guides/fea ... -to-know#1
https://www.cancer.gov/about-cancer/tre ... /off-label

Hunt, I don't think this drug will be off label for long. Even in the current indications being studied the market is substantial. Re dealing with insurance companies--you can be sure J and J has an army of beaurocrats familiar with the process including a strong legal team. No insurance company wants the risk of law suits in the face of a drug with proven efficacy (we need to exceed some burden of proof). This is why right to try might have trouble in the absence of true careful clinical studies. Insurance companies just need to see patient benefit and lowered costs (fewer hospital days, transfusions, doctor visits etc--imagine getting rid of bone marrow transplant expense.) bp

Again I hope you are right or even close to right on this biopearl. I know there is great excitement building from an investors perspective, of which I am also, as well as the patients in need, so maybe I being a little cynical and jaded on the few experiences I’ve had, but the real world does have a way of taking some of the smile off sometimes. I believe that a level of compassion is noticeably missing in these processes, hopefully that can all change but not sure how or when.

Some of my experiences don’t give me any warm feelings about how and “when” this all comes together. Forget about the complexity of getting Imetelstat treatments, just on a very basic level, working with my wife’s many prescriptions on various autoimmune diseases, it has been virtually impossible in some cases to get past the insurance company if they have a different mind on what you should be able to do. Cost is probably the golden key but still a difficult process to overcome their “definition” of the SOC which is often based strictly on cost. I have not been very successful with appeals, etc. as there are many moving parts on input, from the doctors input (usually have to write the letter for the doctor’s to get that part taken care of), then there is the evidence for your case, issues around not being on the insurance company’s formulary, 2-3 weeks between rejection letters, etc., etc.

Also, based on my experiences in assisting my relative and his wife in his attempt to obtain Imetelstat (he was an R/R MF patient) once it became an orphan drug. One might think that being a drug with ODD, it would be approximately equivalent to a “right to try” drug, but it wasn’t at all. We found it nearly “impossible” for mere mortals like us to chart the path forward. Support organizations would / did not advocate, usually just pointed us to another group or JNJ. And of course, JNJ out-sourced the job of interfacing with the public on these requests and the maze was not something we could get resolved no matter how hard we had tried while the clock kept ticking. And yes the oncology doctor has to be quite involved and willing to assist to get this done, it wasn’t an automatic given the safety considerations. Any missing “cog” in the chain of teamwork required became effectively for us a stone wall.

Of course, an off-label prescription to an approved drug seems like it should be more accessible and if the math on cost is actually key and agreed on and the JNJ army suits up and takes the field, etc. this should all come together in time. I just know the MF disease isn’t going to wait on anything, it will just keep marching on.