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Back to index of statements Mark McClellan M.D., Re: Draft Formulary Review Criteria Dear Administrator McClellan: The Alzheimer's Association appreciates the opportunity to comment on the proposed Centers for Medicare and Medicaid Services (CMS) Strategy for Affordable Access to Comprehensive Drug Coverage for the Medicare prescription drug benefit in the Medicare Prescription, Drug, Improvement and Modernization Act of 2003 (MMA). The Alzheimer's Association is the premier source of information and support for the 4.5 million Americans with Alzheimer's disease. Through its national network of chapters, it offers a broad range of programs and services for people with the disease, their families, and caregivers and represents their interests on Alzheimer-related issues before federal, state, and local government and with health and long term care providers. The largest private funder of Alzheimer research, the Association has committed nearly $165 million toward research into the causes, treatment, prevention, and cure of Alzheimer's disease. General Comment The Alzheimer’s Association supported the MMA because it will provide significant relief to 4.5 million Americans who have Alzheimer’s disease. For the first time, Medicare beneficiaries will receive coverage for outpatient prescription drugs. Our comments and concerns reflect our objective to ensure access to medications needed by Alzheimer’s the compassion to care, the leadership to conquer beneficiaries. Access to effective medical treatment must be paramount to CMS during the revision process. We encourage CMS to consider the impact of these guidelines on the most vulnerable Medicare beneficiaries, including the chronically ill, the dual eligibles and the residents in nursing facilities. While we have several recommendations that we believe are critically necessary to build on your proposed review process, we are generally supportive of the approach that CMS proposes to take with respect to reviewing Part D plan benefit structures and formularies. We are pleased to see that CMS recognizes the need to review Part D plan formularies to ensure that they do not discriminate or substantially discourage enrollment by certain groups of Medicare beneficiaries. We support the identification of three areas of focus: pharmacy and therapeutics (P&T) committees, formulary lists, and benefit management tools as among the most critical areas for your review. We appreciate that CMS is viewing the statutory requirement of two drugs per class as a floor, and not an absolute standard. We are encouraged that CMS recognizes the need to look at each plan’s use of cost management tools. We are particularly pleased that CMS intends to show deference to widely accepted clinical practice guidelines. We primarily focus our comments on the recommendations of evidence-based treatment guidelines that are utilized by clinicians who treat individuals with Alzheimer’s disease. Access to New FDA-Approved Pharmaceuticals We are pleased that CMS acknowledges the need for plans to consider providing coverage for new chemical entities, although the draft guidance is insufficient. Given the recent increase in research and development of drugs for Alzheimer’s disease that would benefit the Medicare population, this process should be expedited and require automatic inclusion of drugs within 30 days of FDA approval. The draft guidance states that a best practice is to require P&T Committees to review each new chemical entity within 90 days of its release onto the market, or a clinical justification must be provided if this timeframe is not met. We believe that this length of time is too long, and does not take into consideration unique characteristics of the Medicare population. Many Medicare beneficiaries have life-threatening or chronic conditions for which there is no effective treatment. Given the lengthy process to obtain FDA approval of these agents, plans are, or should be, aware of the pending approval of new pharmaceuticals and can plan accordingly. For example, in January 2004, the FDA approved the first drug (memantine) that was proven to be safe and effective for individuals with moderate to severe Alzheimer’s disease. By February 2004, the Veteran’s Administration, one of the largest health care providers in the country, published its Criteria for Use of Memantine and began providing immediate access to this newly approved pharmaceutical. When new chemical agents are approved, Medicare beneficiaries need immediate access to these new treatments, and plans should be required to include coverage of them within 30 days of FDA approval. Ensuring that P&T Committees function effectively We concur that the P&T Committee is vital and that P&T operations should ensure that the benefit is clinically sound and nondiscriminatory. We do, however, have some concerns with the guidance regarding P&T structure. Specifically, the requirements listed do not adequately ensure that determinations will meet the desired goal of being clinically sound, unbiased, and nondiscriminatory. P&T Requirement: The draft guidance, as written, seems to anticipate that all plans will have formularies and thus require a P&T committee under the MMA. However, while unlikely, it is possible that a plan might not have a formulary. All Part D plans—with or without formularies—should be required to have a P&T Committee to conduct best practice reviews, evaluate physician prescribing, and assure the interests of enrollees are protected. Therefore, we recommend that the guidance state that all plans should have a P&T Committee that meets the outlined membership and conflict of interest requirements and has responsibilities including (but not limited to) evaluating beneficiary drug use patterns, compliance reviews, establishing and monitoring drug screen processes to reduce adverse events. Further, the guidance should outline the goals and objectives of the P&T Committee to include a charge for P&T committees to “ensure that the interests of enrollees, taking into account the unique needs and co-morbidities commonly associated with aging populations and people with disabilities served by Medicare, are protected by all formulary and benefit design decisions made by the Part D plan” and that P&T decisions be made with the goal of ensuring “adequate access for the most clinically efficacious drugs in the preferred tier for all classes of covered drugs.” Composition of P&T Committee: The elderly and individuals with disabilities respond very differently to medications from the general population. Elderly patients are particularly vulnerable to drug-related problems and have a high rate of drug-drug interactions. This is because of the frequency with which they take multiple medications, as well as age related changes in pharmacokinetics and frequent compliance issues (Merck Manual of Geriatrics). Because these plans will be serving the elderly and individuals with disabilities, we urge that the guidance incorporate P&T committee membership requirements that will better ensure that the unique needs of the Medicare population are adequately addressed:
Conflict of Interest: The guidance requires that one pharmacist and one physician on the P&T Committee be independent and free of conflict with respect to the sponsor and plan. As an advocacy organization, we believe it is important that the P&T Committee is unbiased. However, we are concerned that restricting members to individuals with no conflicts of interest would limit the knowledge base of the committee. We believe that CMS should emphasize disclosure by committee members and that such disclosure should be public. Once a year disclosure on paper is not effective and could be meaningless. In-person disclosure at every meeting is practicable, straightforward, and current and therefore, can protect enrollees from hidden conflicts. This procedure is similar to the current practice at the Medicare Coverage Advisory Committee meetings and will promote unbiased review. Meeting Administration. We recommend additional administrative provisions to ensure that P&T Committee decisions are timely and unbiased. Given the challenges of establishing this new benefit, we support a requirement for monthly P & T Committee meetings at least for the first two years of the program in order to address implementation issues and concerns that require prompt resolution. In addition, there may be times when decisions are required between meetings. The guidance should require each P&T Committee to have procedures for determinations and decision-making between scheduled sessions. We agree with CMS’ recommendation that all P & T Committee decisions must have written documentation. Appropriate use of benefit management tools We strongly support CMS’ statement in the draft guidance that they will review plans’ use of drug utilization review tools and techniques to assure appropriate access to medically necessary therapies in a timely manner. We are concerned that the cost containment strategies employed by some of the plans may encourage the use of older or less effective drugs that often result in increased side effects and adverse reactions in older, frailer beneficiaries, such as those with Alzheimer’s disease. With impaired memory and judgment, individuals with Alzheimer’s disease may not be able to recognize, understand or report early signs of an adverse reaction until it results in an acute episode. Due to the increased age, lower metabolism and co-existing conditions of this population, physicians require a wider range and greater flexibility of therapeutic agents when treating their patients. Ninety-five percent of Medicare beneficiaries with dementia have one or more other chronic conditions, including congestive heart failure (28%), diabetes (21%), chronic obstructive pulmonary disease (17%) and coronary heart disease (30%). Sixty to ninety percent of Alzheimer’s individuals suffer from neuropsychiatric symptoms including agitation, depression, apathy, anxiety, delusions, hallucinations, paranoia and sleep impairment. These patients are taking a combination of medications to appropriately manage these conditions. It is a delicate balance that requires access to specific medications to enhance the care to the beneficiaries. Many formulary restrictions and cost-sharing requirements could have significant and disproportionate adverse impacts on individuals with Alzheimer’s disease. Given the high prevalence of neuropsychiatric symptoms and co-existing conditions, these beneficiaries often rely heavily on multiple medications to treat their conditions and are likely to be more sensitive to and less tolerant of many medications. To ensure appropriate access to necessary prescription drugs, CMS should provide meaningful beneficiary safeguards for vulnerable populations, including those with Alzheimer’s disease. Recommendations : We strongly recommend that CMS prohibit plans from engaging in therapeutic substitution without the consent of the treating provider. We also believe that plans should be prohibited from having restrictions on the frequency of dispensing, the maximum daily dosage, or the number of prescriptions filled. Prohibiting such limits would be consistent with comments from Dr. Mark McClellan during his confirmation hearing in the Senate Finance Committee when he stated that, “beneficiaries who elect to enroll in this new open-ended drug benefit will have no limits on the number of prescriptions filled, no limits on the maximum daily dosage, and no limits on the frequency of dispensing of a drug.” We are also concerned about the application of prior authorization and urge CMS to look at what has and has not worked in the Medicaid program as a guideline for setting standards for prior authorization programs ( e.g. , use of “dispense as written” provisions, time frame for determinations, access to therapy in emergency situations). We urge that, when prior authorization is imposed, if the prior authorization process has not been completed within 24 hours of the time that a prescription was first presented at a pharmacy , plans be required to dispense a temporary supply of the prescribed drug pending the completion of the prior authorization process. The final rule should also provide for exigent circumstances when an emergency temporary supply of a prescription drug must be dispensed immediately, without allowing for a 24-hour prior authorization period. In assessing plans’ use of benefit management tools, the draft guidance states that CMS will compare the use of these tools (e.g., prior authorization, step therapy, generic substitution) in the prescription drug plans (PDP) and Medicare Advantage plans to the use of these techniques in existing plans (private sector, Medicaid, FEHBP) to ensure non-discrimination. Most state Medicaid programs have highly tailored their use of prior authorization and fail-first policies. It is believed that most drugs dispensed by most Medicaid programs do not require prior authorization. Further, classes of drugs for the treatment of mental illness and drugs with a narrow therapeutic index are commonly excluded from prior authorization. States also employ fail-first policies very selectively. Indeed, a review of state use of fail-first policies suggests that this policy tool is generally used only with drug classes for which there is strong evidence of clinical equivalence, and states generally do not use fail-first policies broadly across large numbers of drug classes. Given the risk for increased side effects and adverse reactions, we oppose the use of these cost containment tools for individuals with Alzheimer’s disease. Treatment Guidelines for Management and Treatment of Alzheimer’s Disease In the draft guidance, CMS requests recommendations of evidence-based treatment guidelines. Our response to this request includes discussions of four types of medications that are essential for the management of cognitive and noncognitive symptoms of Alzheimer’s disease: cholinesterase inhibitors, memantine, antipsychotics, and antidepressants. We also address two other significant, medication-related issues in the management of Alzheimer’s disease. It is important for CMS to consider that treatment and management of Alzheimer’s is a dynamic field and new research is frequently being published. Consequently, CMS should review the plan formularies on a regular basis to assure that they include new, more effective medications and take into account the most recent findings on dosage and side effects. The formularies must be responsive to the needs of Alzheimer’s beneficiaries to assure that Medicare beneficiaries have access to the most effective and proven treatments available. Alzheimer’s disease is not a “one size fits all” condition. Each individual has different manifestations of particular symptoms. Providing symptom relief for an individual requires an understanding of the specific symptoms of the individual and creativity in devising the treatment plan. Treatment must be dynamic and flexible. As previously noted, treatment plans are often developed by trial and error to acquire the correct combination of medications. In 2001, the American Academy of Neurology (AAN) published clinical guidelines to address pharmacologic and nonpharmacologic treatments for dementia management. These practice recommendations, Practice parameter: Management of dementia (an evidence based review): Report of the Quality Standards Subcommittee of the American Academy of Neurology , were published in Neurology 2001; 56:1154-1166 . These guidelines provide an excellent framework for considering formulary issues for people with dementia. We recommend that CMS follow the AAN recommendations; however, the practice parameter was published in 2001. Since then, FDA has approved one new type of medication for Alzheimer’s, and other important findings have emerged about the other types of medications discussed below. These changes in a 3-year period highlight the importance of ongoing review of formularies that should be modified or updated as new research or new drugs become available. We suggest recent review articles that incorporate relevant findings since 2001. Cholinesterase Inhibitors There are four FDA approved cholinesterase inhibitors: tacrine, donepezil, rivastigmine and galantamine. The AAN practice parameter notes that “significant treatment effects have been demonstrated with several different cholinesterase inhibitors,” and recommends that cholinesterase inhibitors should be considered standard practice in patients with mild to moderate Alzheimer’s disease. At the time of the publication, there had been no head-to- head comparison of the cholinesterase inhibitors. The primary differences among the four FDA approved drugs are side effects and ease of dosing. Two review articles published in 2003, as well as guidelines published by the Veteran’s Administration (VA), support the AAN recommendations but provide updated specific dosing information and data for each drug. These three documents differ from the AAN guidelines in their recommendation of only restricted use of tacrine because of research and clinical findings of significant adverse side effects, especially hepatotoxicity risk. The VA guidelines and the two publications can be found at: Veteran’s Administration - Cholinesterase Inhibitor Therapy for Alzheimer’s Disease: Criteria for Use. May 2003, Updated March 2004 . http://www.vapbm.org or http://vaww.pbm.med.va.gov. Kawas, C., Clinical Practice: Early Alzheimer’s Disease. N. Engl J Med 349:11, September 11, 2003. Grossberg, G. and Desai A., Management of Alzheimer’s Disease. Journal of Gerontology: Medical Sciences 2003, Vol 58A., No. 4, 331-353. In addition, the Agency for Healthcare Research and Quality (AHRQ) had a comprehensive evidence report prepared by McMaster University which was published in April 2004. This evidence report, Pharmacological Treatment of Dementia. Evidence Report/Technology Assessment No. 97 , http://www.ahrq.gov/clinic/tp/dempharmtp.htm provides a discussion of all the pharmacological research published through 2003 and may be a helpful resource document for CMS. Memantine Memantine, an NMDA receptor antagonist, was approved by the FDA for use in moderate to severe Alzheimer’s disease in January 2004. The VA published its Criteria for use of Memantine (Namenda) , found at http://vapbm.org or http://vaww.pbm.med.va.gov soon after the FDA approval. The VA recommends the use of memantine alone and in combination therapy with cholinesterase inhibitors. Due to its recent approval, use of memantine is not extensively discussed in the AAN guidelines or the Kawas and Grossberg publications. Antipsychotics It is well documented that there is a high prevalence (60-90%) of neuropsychiatric symptoms in individuals with dementia. These symptoms include agitation, depression, apathy, anxiety, delusions, hallucinations, paranoia and sleep impairment and have serious adverse consequences on the patients. Appropriate treatment of these symptoms provides substantial benefits to these individuals, such as the improvement of function and the reduction of disruptive behavior. The AAN guidelines recommend as standard practice the use of antipsychotics to treat agitation or psychosis in patients with dementia. The AAN subcommittee suggests that atypical agents may be better tolerated compared to traditional agents. The American Psychiatric Association’s, Practice Guideline for the Treatment of Patients with Alzheimer’s Disease and Other Dementias of Late Life , published in 1997, recommends specific treatments intended to decrease psychotic symptoms and agitation, screaming, combativeness or violence in this vulnerable population. The APA guideline stresses the importance of selecting a pharmaceutical intervention that is specific to the target symptom in order to optimize treatment and to monitor the effect of that treatment. The Kawas and Grossberg articles provide updated research and dosing information for the use of antipsychotics. The AHRQ evidence report also includes a review of studies on this class of medications. A recent publication on antipsychotic treatments reviews the scientific literature available as of summer 2003 on five atypical antipsychotic medications for patients with dementia. See, Katz, I. Optimizing Atypical Antipsychotic Treatment Strategies in the Elderly , JAGS, 52:S272-S277, 2004. While Dr. Katz’s review is quite valuable, he stresses the importance of the on-going research supported by NIMH, Clinical Antipsychotic Trials of Intervention Effectiveness (CATIE), which is comparing the effectiveness and safety of conventional and atypical antipsychotics in the treatment of older adults with Alzheimer’s disease. Information about this five-year, multi-site trial can be obtained online at http://www.catie.unc.edu. Antidepressants Depression and depressive symptoms are common in individuals with Alzheimer’s disease, occurring in 25 to 50 percent of the Alzheimer’s population. Treatment of these symptoms can be effective in the individual and caregiver. The AAN guideline suggests that SSRIs, MAO-B inhibitors and selected tricylics should be considered in the treatment of individuals with dementia, with side effect profiles guiding the choice of agent. The APA guideline is consistent with the AAN recommendation and provides detailed information on the side effects of various antidepressant agents. Both the ANA and the APA suggest that SSRIs may offer better tolerability than other antidepressants. The Kawas and Grossberg articles provide updated data and dosing information for use of antidepressants to improve depression and depressive symptoms in persons with dementia. A review of the treatment guidelines for antipsychotics and antidepressants for individuals with Alzheimer’s disease will corroborate that physicians need significant flexibility to address the specific needs of the individual. The research has shown that the drugs within a class have different effectiveness and side effects and are not interchangeable. Thus, formularies should be required to include more than two drugs in each class in order to meet the special needs of this vulnerable population. Other Medication-Related Issues The Alzheimer’s Association believes it is important for PDPs and Medicare Advantage plans to address the issue of pain in persons with Alzheimer’s disease. Pain management is a problem for many people with dementia because of the lack of recognition of pain symptoms and the inability of the person to effectively communicate or report pain. There are two publications that discuss common pain behaviors in cognitively impaired older persons and recommend appropriate pharmacologic treatment for this population: American Geriatrics Society Panel on Persistent Pain in Older Persons: The Management of Persistent Pain in Older Persons , JAGA 50:S205-S224, 2002. Horgas, A, McLennon S., Floetke, A., Pain Management in Persons with Dementia , Alzheimer’s Care Quarterly 2003: 4(4): 297-311. Finally, the AAN guidelines and the Kawas and Grossberg articles stress the importance of a thorough evaluation of drugs which may cause confusion, excess disability or anticholinergic effects in the Alzheimer’s population. The VA’s Drug Class Review: Cholinesterase Inhibitors and Criteria for Use , referred to above, include discussions of safety concerns of drug interactions of various agents. Both documents include tables that show potential anticholinergic activity and drug interactions of selected agents. Conclusion The Alzheimer’s Association appreciates the enormous effort of CMS to implement these provisions of the Medicare Modernization Act. We recognize the challenges that this entails, including the difficulty of balancing the interests of the various stakeholders with the need for protection of vulnerable Medicare beneficiaries. We hope that our comments will assist CMS in achieving the proper balance. In summary, we recommend that CMS:
We continue to believe that each Alzheimer’s beneficiary is unique and that the intervention selected be unique to the needs of the individual and be of benefit to the individual, the family and the caregivers. The Alzheimer’s Association is ready to work with you, and to assist in identifying appropriate clinical experts, to assure access to medically necessary drugs for beneficiaries with dementia. Please feel free to contact Leslie B. Fried, Director of the Association’s Medicare Advocacy Project, (202) 662-8684, to further discuss these matters. Sincerely, Leslie B. Fried 
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