1. Introduction
Tafamidis meglumine is a prescription medication used to reduce the symptoms of cardiomyopathy caused by transthyretin mediated amyloidosis in people throughout the world. Tafamidis is a transthyretin stabilizer and is the first medication to be approved to treat this condition. Amyloidosis is a disease caused by the accumulation of abnormal proteins called amyloids. These amyloids can accumulate in the heart and cause it to become stiff and unable to properly pump blood throughout the body. This can lead to HFpEF, heart failure with preserved ejection fraction, a condition where the heart is able to pump out blood, but does so at a slow rate leading to an accumulation in fluids in the lungs and the rest of the body. Amyloidosis tends to be misdiagnosed and underdiagnosed often leading to symptoms becoming very severe before it is properly treated. This means that it is important to get this medication to as many people as possible in order to give amyloidosis patients a chance at slowing down or even halting the progression of their heart condition. Tafamidis is already approved in various countries and will likely be approved by many more in the coming years. (Kallas et al.2022)
1.1. Overview of Tafamidis
Tafamidis is an oral medication and has recently been approved by the FDA for the treatment of transthyretin (TTR). TTR is a rare and serious condition that impacts the heart muscles. There are two forms of TTR, wild type (hereditary) and mutant. Tafamidis was specifically approved for addressing the symptoms of cardiomyopathy from TTR in both forms. Tafamidis is classified as a pre-specified medicine for the treatment of TTR and as a selective therapy. Tafamidis has been shown that it is able to prevent direct damage to the heart muscles by halting the progress of an enzyme that is the route of TTR symptoms and can prevent the progression of TTR. This medication has been shown to reduce the amount of hospitalizations caused by TTR and has a direct correlation to preventing heart damage, which is the most serious complication in TTR. Tafamidis being preventative and specifically for TTR symptoms shows that it is an important medicine for those diagnosed with TTR in their future health and living. With current treatments for TTR being methods to alleviate the symptoms and no cure, or specific treatment, Tafamidis is in a class of its own for being an actual treatment for TTR.
1.2. Importance of Medicare Coverage
Tafamidis is a drug used to treat a rare disease known as transthyretin amyloid cardiomyopathy (ATTR-CM). It functions to stabilize the progression of this disease by reducing the amount of hospitalizations. There have been stronger pushes for Medicare coverage of tafamidis; one argument being that Medicare coverage could allow for greater access to treatment for patients with ATTR-CM. ATTR-CM is most common in patients above the age of 65 years; Medicare coverage would aid this population to a greater extent than coverage with other forms of insurance. This argument holds weight in regards to the intended Medicare population, and suggests that Medicare coverage would be more essential for tafamidis compared to other drugs. Another perspective is the economic impact on the patient, stating that Medicare coverage would allow patients to save thousands of dollars in out-of-pocket costs. Treatment with tafamidis costs approximately $225,000 to $300,000 per year; even for patients with supplemental insurance, the remaining out-of-pocket costs could be financially catastrophic. Chemistries and displaying a resistance to ACE inhibitors, beta-blockers and diuretics.
8. Conclusion
2. Medicare Coverage for Tafamidis
There are two different types of financial assistance available for prescription drug coverage under a Medicare Part D plan – the low-income subsidy (also called Extra Help) and the Medicare Savings Programs. Both can help to pay premiums, deductibles, and copayments related to prescription drug coverage. Qualified Medicare Beneficiaries (QMB), Specified Low-Income Medicare Beneficiaries (SLMB), and Qualified Individuals (QI) programs are for individuals with income and resources slightly higher than the above programs who do not qualify for Medicaid. These programs help to pay Medicare Part A and/or B premiums and may also help to pay deductibles, copayments, and coinsurance. QMBs are automatically eligible for Extra Help and will not be denied enrollment into a Medicare Part D plan. Dual eligible individuals (including persons enrolled in a Medicare Savings Program) are eligible for a special enrollment period that allows changes in Medicare Part D plan enrollment at any time during the year.
The federal government provides prescription drug coverage to people who are eligible for Medicare benefits. One option for prescription drug coverage is enrollment in a Medicare Prescription Drug Plan (Medicare Part D). Medicare Part D plans provide insurance coverage for prescription drugs to beneficiaries. All people with Medicare are eligible for this coverage, regardless of income and resources, health status, or prescription drugs currently used.
2.1. Eligibility Criteria
Due to these systematic and documentation limitations, the ability to optimize protection for beneficiaries will only be achieved through the greater flexibility offered by Part D. Consequently, CMS conducted a review to determine the best interests of Medicare beneficiaries who are prescribed Tafamidis for the treatment of ATTR-CM. A summary of the evidence reviewed is available via the “Evidence” link. An assessment of whether the coverage of Tafamdis is a category 1 benefit is available via the “Categorization” link. Both the “Evidence” and “Categorization” links are located at the bottom of this webpage.
Receipt of Tafamidis in the inpatient or outpatient setting and use of the medical benefit including hospitalization is generally not a viable option since the COVID-19 public health emergency continues to create access barriers for vulnerable individuals seeking multiple encounters in the medical benefit and access to supportive care treatments. Tafamidis administration in the inpatient or outpatient setting represents a severe exacerbation in this patient population.
Coverage under Part B is preferable as this will enable coverage through the Medicare prescription drug program and will ensure individuals have access to Tafamidis administered in inpatient or outpatient settings. Tafamidis is a prescription medication available in capsule form and it is self-administered. Tafamidis will not be offered in a formulation that is infused.
The eligibility criteria for coverage of Tafamidis (Vyndaqel) and Tafamidis meglumine (Vyndamax) (collectively referred to as Tafamidis) are applicable to both Part B and Part D. For purposes of this National Coverage Determination (NCD), the two Tafamidis formulations have identical indications and uses, and are therefore covered under the same NCD. These Tafamidis formulations are indicated for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.
Medicare coverage for Tafamidis
2.2. Coverage Options
TTR-FAP patients who are covered under original Medicare are likely ineligible for assistance programs provided by pharmaceutical companies. However, beneficiaries with Medigap, Medicare Advantage or other supplemental insurance policies can apply for co-payment assistance from the organization sponsoring the charitable program. The Tafamidis patient assistance program offers co-payment assistance to patients with a demonstrated financial need using a co-payment card. Each card is valid for one year with a maximum benefit of $15,000. If the patient’s out-of-pocket cost exceeds $15,000 in a given year, he/she may apply for an additional $10,000 benefit. In the event that Medicare coverage of Tafamidis changes, these programs may be subject to adjustment. Tafamidis patients who receive insurance through an employer or union-sponsored retiree plan should consult with the plan administrator and/or insurance company for coverage and assistance information.
2.3. Limitations and Restrictions
However, due to the nature of cardiomyopathy being a symptom rather than a specific diagnosis, it was necessary to specify a further criteria to ensure that only people with ATTR-CM would be able to access tafamidis at the concessional rate. This is achieved by restricting the tafamidis benefit under the RPBS to only those patients with a valid RPBS concessional card who have a co-existing diagnosis of heart failure with an ejection fraction less than 45% provided by either: – a definition of a specific subgroup from the cardiomyopathy codes (ICD10 Code: I43.8 or Chronic: K8607) in the MBS/PBS database search to identify patients with ATTR-CM; or – a positive heart biopsy or non-bone scintigraphy imaging test consistent with ATTR-CM; or – attestation of the nature and underlying cause of the patient’s cardiomyopathy by the patient’s treating specialist in cardiology or a specialist consultant in general medicine.
People with transthyretin familial amyloid polyneuropathy (ATTR-CM and ATA) should only be required to show that they have been diagnosed with the condition, a process that is made as simple as possible, recognising that in some cases a definitive diagnosis of ATTR-CM or ATA can be difficult. These patients then are able to access tafamidis at the general rate under a special authority prescription.
Now that the availability of a Medicare benefit to support tafamidis has been confirmed, there are requirements that should be clarified related to accessing this medicine at the concessional rate for the approved indications. These requirements have been set out by the PBAC recommendation.
3. Applying for Medicare Coverage
Your doctor is integral to your efforts in trying to have Tafamidis covered by Medicare. They are in the best position to assist you in this process. A positive decision on application for tafamidis for the treatment of ATTR-ACT should only be made by your doctor after careful consideration of all the options available to you, including participation in clinical trials. The Medicare rules set a high bar for which you must be able to supply evidence that you meet. This is required for your application to be successful. While it will vary depending on your personal circumstances and whether an election is made to apply for a new Medicare item, the basic criterion for those with a listed type I, II or III genetic mutation are that you must have a diagnosis of ATTR-ACT. This is clinical and supported by the results of an appropriate test. It must be documented, and you must have symptoms. This is also supported by the results of an appropriate test and must also be documented. Information regarding the types of tests Medicare considered acceptable in order to provide benefit for these services can be found in the MBS, to help you understand whether or not you will meet the criterion.
3.1. Required Documentation
A physician will document the presenting symptoms and diagnosis using a completed CMS-2728 form from the patient’s medical record, and clinical data that supports the diagnosis including results of histology, immunologic studies, amyloid typing, or biopsy. A report of an abdominal fat pad aspiration, if performed, should be included. Information about any previous treatment for amyloidosis, or any fibrate therapy prescribed for hypertriglyceridemia should be documented. Other supportive information might include a log of genotype data collected by the patient at a different time from the initial visit. This will usually be enough information to establish the diagnosis and need for treatment. If CMS denies the initial claim, an appeal and/or personal interview with new data might be necessary. The patient will complete a CMS-1490S form and mail an original copy to Evidence of Coverage and a copy to their Health Care Provider. In some cases, the CMS-1490S might be submitted electronically. The patient should keep a copy of their CMS-1490S form for their records.
3.2. Application Process
Once the Medicare application form has been submitted, an initial response can be expected within 10-90 days. If the patient’s application has been approved, they will receive a letter of acceptance and a separate letter which outlines the patient’s entitlements and what the patient may be required to pay. If the patient’s application has been rejected, the patient will receive a letter of rejection along with the reason as to why they were deemed ineligible for coverage. Upon hearing a response from Medicare, patients have the opportunity to request a review of the decision if they believe that the outcome was not an accurate reflection of their circumstances.
Applications for Medicare coverage must be submitted by the patient’s physician on their behalf and can be completed either online, with a hard copy application form, or over the telephone. The quickest way to apply is through the internet with an estimated completion time of 15 minutes. However, the ability to partially complete the form and resume at a later stage is not available. Application completion via telephone generally requires multiple calls to complete. For hard copy applications, the physician is required to prepare the form in a single session as partially completed forms cannot be saved or completed at a different time and must be posted to the Department of Human Services, Medicare.
3.3. Timelines and Decisions
There are three relevant dates to keep in mind in the decision to apply for CMS. Firstly, if applicant companies an infusion service with the administration of IVIG in the hospital outpatient department or physician’s office to determine if IVIG should be administered essentially in the home through an outcome or NCD, the date of service for Ig will be 1/1/2005. Following the decision of the NCD or outcome, there is a four-year transition period. During this time, the applicant is responsible for tracking the utilization of Ig. It is consistent with the data collection process to create HCPCS Q-codes for the different Ig products. In the 17th meeting of the Medicare Coverage Advisory Committee, the existence of Q-codes as a precondition to applying for coverage was confirmed. It is expected that Q-codes will be assigned to specific Ig products. The results of the transition period and the level of success in CMS coverage given various disease states and service settings will determine when to apply for specific products. Secondly, the MMA included a provision to allow Medicare beneficiaries to opt out of part B and purchase part D for Ig and other physician-administered drugs. This legislation was signed on December 8, 2003, with a delayed implementation until 1/1/2006. This choice in service location and a definite separation of part B and D coverage for Ig are both potentially advantageous changes by the time CMS coverage is reconsidered. A comparison of CMS and dual part D and B coverage for specific patients will be necessary to determine when to apply in this scenario. The provision also made changes to the Medicare secondary payer statute §1862 (b) of the Social Security Act and is discussed in a related but separate legal theme.
4. Additional Resources
Other reputable sources of information about Medicare coverage include your local Medicare carrier and state Health Insurance Assistance Programs.
The Medicare Pharmaceutical Benefits Manual contains instructions and guidance in policies and procedures for the Part D drug benefit.
The Medicare Coverage Database contains a list of all National Coverage Determinations (NCDs) including Tafamidis. With this tool, you can search for an NCD using a variety of criteria (for example, keywords, a range of dates, and/or a specific category) and view the resulting list. Not all NCDs are in the NCD Manual. The database will be updated each quarter, so the results will be continuously updated for NCD changes. The Coverage Documents will give you a summary of the decision or analysis on the topic of the NCD. The Decision Memorandum is an extensive document including the details of the process used and the determination.
Medicare’s official website provides detailed information about Medicare coverage policies. For specific information regarding Part A coverage of provider services and Part B coverage of medical services, visit these two sections of the website.
4.1 Medicare Information Sources: These resources are excellent sources of information concerning Medicare coverage of certain health care services. Each focuses on a different aspect of Medicare coverage.
For individuals seeking more information about Medicare policies, whether for Tafamidis or other health care services, below are a list of helpful resources:
4.1. Medicare Information Sources
In the United States, the coverage of a given medical treatment option may vary depending on the state in which a patient resides and the type of health coverage a patient carries. This may often lead to confusion with respect to Medicare beneficiaries, as they generally are seeking a consistent answer that is applicable nationally. Reflecting upon the tens of millions of Americans who are Medicare beneficiaries and the growing population of individuals with ATTR-CM, it is not surprising that there is a growing number of people inquiring about the coverage of TTR stabilizers through Medicare. Unfortunately, the Medicare Prescription Drug Plan Finder is of little help as it categorizes TTR stabilizers as a chemotherapy agent and thus suggests that these medications are not covered under Medicare Part D with the rationale “all chemotherapy drugs are covered under Part B.” Step-by-step instructions are provided in the appendix for accessing the Supporting Documentation which led to the National Coverage Determination (NCD) for Tafamidis. This is an administrative memorandum to CMS from a decision in which CMS agreed with the review of an appeal that Tafamidis is not a chemotherapy agent, and it now holds its own unique HCPCS code and is to be administered and covered as an oral-only therapy provided in an outpatient setting. Patients are encouraged to go back and review on a later date, as the most relevant information to determine coverage under a Medicare Part D plan would be a change in the NCD for which there would be a 30-day notice before it goes into effect. Until that time, a favorable NCD is the best-case scenario for any Medicare beneficiary seeking insurance coverage of a medication.
4.2. Patient Support Programs
Tafamidis is used to treat the heart condition transthyretin amyloid cardiomyopathy. If you have this condition, talk to your doctor about whether it is right for you. If your doctor feels that you could benefit from this drug, there are 2 ways you could qualify for extra help from Medicare to pay for it: You may be able to get help from a Medicare Advantage Plan. If the above describes your situation, and you have already been prescribed tafamidis, you should contact the manufacturers of the drug to see if they still have the programs going that I describe here, or if they have something else. At this time, Pfizer had 2 patient support programs to help with the cost for people using tafamidis. The only way to find out if these programs are still in effect is to call the company at the phone number on this page: tafamidis cost in US.
4.3. Frequently Asked Questions
Tafamidis is a medication used in the treatment of transthyretin amyloidosis, a rare condition that can be hereditary or acquired. It is not currently known if Tafamidis is covered by Medicare, this is likely due to the nature of the condition. Since Medicare generally doesn’t cover rare conditions or conditions that affect too few people to allow for clinical study. The cause of this is because it makes assessing the value of the treatment difficult and is improbable that the treatment has any effect on the condition because it simply was never studied. Sometimes drugs that are meant for much more common conditions and have research proving their effectiveness will be more widely accepted and subsidized by Medicare, although even some of these are still not covered. If you are specifically looking to get your hands on Tafamidis and live in the United States, you should look into Medicaid programs based on income as Medicaid may have more specific assistance.
References:
Kallas, O.N., Nezami, N., Singer, A.D., Wong, P., Kokabi, N., Bercu, Z.L., Umpierrez, M., Tran, A., Reimer, N.B., Oskouei, S.V. and Gonzalez, F.M., 2022. Cooled radiofrequency ablation for chronic joint pain secondary to hip and shoulder osteoarthritis. RadioGraphics, 42(2), pp.594-608. rsna.org
