Patient advocacy groups representing millions of patients are speaking out against a plan in Massachusetts that would limit patients’ access to life-saving treatments, therapies and medications prescribed by their doctor.
In September, health officials in Massachusetts submitted a formal request to amend Section 1115 Demonstration Project with the Centers for Medicare & Medicaid Services. The request, if granted by federal authorities, could cause abrupt changes and serious disruptions in treatments for patients enrolled in MassHealth, the state’s Medicaid program
“This proposal is bad for patients,” said Terry Wilcox, executive director at Patients Rising, a national patient advocacy group that fights for improved health care access. “MassHealth’s Section 1115 demonstration waiver would limit patients’ ability to access the life-saving treatments that are prescribed by their doctor.”
Dozens of patient advocacy groups and non-profit organizations, including the Jett Foundation, National MS Society, Epilepsy Foundation, Leukemia & Lymphoma Society, National Organization for Rare Disorders, Hemophilia Federation of America, and American Heart Association, are fighting the proposal, which they say, could have devastating effects for patients living with serious, chronic and rare diseases.
Closed Formulary: Barrier for Patients with Rare Diseases
Of particular concern to patients, a provision that would impose a closed formulary for medications, which would leave patients without access to the treatments prescribed by their doctor. Instead, patients could only gain access to treatments that have been approved by a government official based on a host of factors, including cost. Patient advocates say that policy jeopardizes the health of patients, especially with rare and hard-to-treat conditions,
“The provisions within the proposed Amendment to the MassHealth Section 1115 Demonstration would create a barrier to access for patients hoping to gain access to those future therapies,” explains the Jett Foundation, which fights for patients living with Duchenne muscular dystrophy, a rare muscle disease that primarily affects young boys. “This waiver if granted will dramatically hinder the ability of Duchenne patients and medical experts to execute care plans that could improve the health and quality of life of Duchenne patients.”
That view is reinforced by Patients Rising and the National Organization for Rare Disorders, which is concerned that the Section 1115 demonstration amendment could have a “devastating impact on the health and well-being of rare disease patients.”
“Every patient should have access to the right treatment prescribed by their doctor,” Patients Rising wrote in a letter of opposition to the Section 1115 demonstration amendment. “Medical professionals are in the best position to decide which treatment will work best for their individual patient – based on numerous factors, including the patient’s medical history, potential to suffer side effects, treatment resistance and severity of the disease or condition.”
Privileges Certain Types of Medical Care, Discriminates Against Patients with Chronic Diseases
117 million Americans live with a chronic disease or condition, and advocates warn that their treatments could be disrupted by the proposed changes. The American Diabetes Association, which “is strongly opposed to the commonwealth’s proposal to implement a closed formulary for the MassHealth program,” explains that every patient with diabetes requires a personalized treatment plan.
“Diabetes care should be patient-centered, and requires a close working relationship between the patient and clinicians involved in treatment,” the organization wrote in opposition to the Section 1115 Demonstration Amendment request. “We are particularly concerned that important types of medications for people with diabetes could be left out if the commonwealth chooses to cover only one medication per therapeutic class.”
In effect, the proposal privileges some types of medical care over others. If a patient with heart disease was rushed to the hospital for life-saving surgery, MassHealth would, rightly, cover the procedure. Paradoxically, MassHealth’s proposal would make it more difficult for the same patient to access medications that could reduce their risk of a heart attack.
Even seemingly small changes in medications can have major health consequences for patients. Mental Health America points out that patients living with mental health challenges can spend years finding the right medication in the right dosage. Treatment restrictions, such as those proposed with the Section 1115 Demonstration Amendment, can deliver a permanent setback to these patients.
Section 1115 Demonstration Amendment Could Increase Health Care Costs
The Section 1115 Demonstration Amendment is likely to increase overall health care spending over the long term. Access to innovative treatments reduces overall health care costs. According AIDS Action Committee of Massachusetts, access to HIV medications has contributed to a 67% viral suppression rate, a nearly 50% drop in new infections and an estimated $1.8 billion in lifetime HIV treatment costs.
The single-minded pursuit of cost-cutting is counterproductive. A 2016 study found that “a $1 increase in prescription drug spending reduces non-drug expenditure by approximately 20 cents.” By denying access to the right treatment, a patient’s condition can worsen, resulting in more expensive procedures and more time in the hospital.
At a time when technology is providing new opportunities to tailor medicines to the unique needs of every patient, this proposal is a step backwards – returning to the days of one-size, fits-all policies and inflexible formularies.
Check out what leading patient advocacy organizations are saying about Massachusetts Section 1115 Demonstration Amendment Request.
American Heart Association/ American Stroke Association: “Prescription drugs are a lifeline for people with chronic diseases”
As a key health care cost driver, we understand the need to manage prescription drug costs. However, we are concerned that more limited specialty pharmacy networks and a closed formulary, as proposed in the 1115 Waiver Amendment, would impose unnecessary barriers to needed medications. Unlike many of the other proposed changes, these changes apply to all
MassHealth members, including people with disabilities, children, and seniors. Prescription drugs are a lifeline for people with chronic diseases especially those with cardiovascular disease.
Mental Health America: “Cost-based access restrictions are not appropriate for psychotropic medications”
With our years of experience in state and federal policy, we find that cost-based restrictions on access to medication injure individuals and end up not being an effective cost-saving strategy.
Cost-based access restrictions are not appropriate for psychotropic medications because each molecule is unique and each individual responds differently. The process of finding a medication that an individual responds to with tolerable side-effects can take months or even years. This is true not only between molecules, but between formulations as well, such that generic formulations have different therapeutic effects than their name-brand counterparts. On this backdrop, cost-based restrictions, and especially restrictions that only guarantee access to a single drug in a therapeutic class, are not appropriate.
Empirical research on cost-based restrictions to psychotropic medication access bears out this claim states have not saved money, and in some cases even paid more, and hospitalizations increased for the individuals affected. Each instance of crisis will hurt the individuals, their families, and their communities throughout Massachusetts, without providing benefit to the taxpayers.
Hemophilia Federation of America: MassHealth Section 1115 Demonstration Amendment Request would put patient health at risk
Closed formulary… we strongly urge MassHealth, as it considers a possible move to a closed formulary, to adhere to the longstanding practice (widely followed across state Medicaid programs) of carving out hemophilia therapies from the standard drug utilization review/preferred drug list (PDL) process. Limiting product options for individuals with bleeding disorders via PDLs or otherwise would put patient health at risk and could actually result in higher overall medical costs with respect to this patient population.
…limiting product options for patients with bleeding disorders is neither an effective nor a therapeutically appropriate way to manage this class of patients. Clotting factors vary in a number of important respects, including half-life and immunogenicity, and as such are not therapeutically equivalent or interchangeable. No generic clotting factor exists. Patient bleeding patterns and responses to different clotting factors vary widely.
Recognizing this diversity of clotting factor products, the Medical and Scientific Advisory Council (MASAC) of the National Hemophilia Foundation (NHF) has stated that product selection for bleeding disorder patients “require[s] a complex decision making process” between a patient and his or her physician: “it is critical that the bleeding disorder community has access to a diverse range of therapies and that prescriptions for specific clotting factor concentrates are respected and reimbursed.” Because the selection of the medically-optimal product for each patient is so individualized and so important, MASAC urges third-party payers to cover whichever factor product is prescribed by the patient’s treating physician rather than resorting to a PDL or formulary approach.
By contrast, a one-size-fits-all approach (for example, requiring patients, for non-medical reasons, to switch from a prescribed product to a different, POL-listed product) can undermine adherence to therapy, weaken the doctor-patient relationship, and worsen patient outcomes in both the short and long term — while also raising payer costs due to additional doctors’ visits, hospitalization, and/or extra required factor usage. Closing off access to certain factor products for Medicaid patients could thus end up costing more for state programs and impairing patient care.
Harvard Law School Center for Health Law and Policy Innovation: MassHealth Section 1115 Demonstration Amendment Request “concerning for continued access to HIV and HCV medications”
MassHealth proposes to establish a closed formulary with preferred and covered drugs across the entire program. Currently, MassHealth is required to cover any drug for which the manufacturer participates in the federal Medicaid rebate program. This requirement ensures that patients have access to the highest standard of care available and allows physicians to prescribe the course of treatment they and their patients believe is most appropriate. A closed formulary would restrict the drugs MassHealth covers, with as few as one drug available per therapeutic class. Unlike several of the changes proposed elsewhere in this 1115 Waiver Amendment Request, this would apply to all MassHealth members, including people living with disabilities, children, and seniors. Prescription drugs are a lifeline for people living with chronic and complex conditions, and further restrictions on access to medications will only serve as a barrier to obtaining the treatment regimens that are most appropriate for these individuals.
This proposal is particularly concerning for continued access to HIV and HCV medications. Physicians choose which drugs to prescribe their HIV and HCV patients based on a wide range of factors, including co-occurring illnesses, medical history, and previous treatment tolerance. It is important to note that HIV and HCV drug regimens are not interchangeable. HIV and HCV are complex diseases and treatment options must take into account several individualized medical factors as well as concerns regarding a patient’s medication adherence. Before initiating treatment, physicians must consider drug interactions, coexisting conditions, and side effect profiles. Therefore, it is important that doctors are able to provide treatment based on patients’ needs, not on availability in MassHealth.
Implementing an exceptions process to a closed formulary through which an individual can attempt to access coverage for a drug not on the formulary would also fall far short of ensuring that people living with HIV or HCV and their providers can access the appropriate treatment regimen. This is true because of the uncompensated cost to providers of going through the exceptions process, because this coverage is not guaranteed, and because the process of obtaining this coverage is often opaque.
Massachusetts American Cancer Society Cancer Action Network: “Could hinder cancer patients’ access to medically necessary prescription drugs”
We are concerned with the Department’s request to adopt a closed drug formula for Mass Health, as cancer patients are often appropriately prescribed off-label use drugs to treat their disease, a practice that is especially common with rarer cancers like pediatric cancer. Congress has long recognized the need for off-label use of drugs in oncology.
ACSCAN is concerned that the proposed policy could hinder cancer patients’ access to medically necessary prescription drugs. We note that the waiver cites to the Medicare Part D program as justification for permitting a closed formulary. However, we would suggest that this comparison falls short. The Medicare Part D program covers outpatient prescription drugs and it is not clear from the proposal whether the waiver seeks to impose a closed formulary for all prescription drugs or would only apply to outpatient prescription drugs. In addition, while the Medicare Part D program does allow plan sponsors to create a formulary with at least two drugs per therapeutic class, it also requires sponsors to cover all or substantially all drugs in six classes and categories of prescription drugs including anticonvulsants, antidepressants, antineoplastics, antipsychotics, antitretrovirals, and immunosuppressants (the so-call “protected classes”). In fact, the Medicare Part D manual clearly states that “CMS instituted this policy because it was necessary to ensure that Medicare beneficiaries reliant upon these drugs would not be substantially discouraged from enrolling in certain Part D plans, as well as to mitigate the risks and complications associated with an interruption of therapy for these vulnerable populations.” It does not appear that similar protections are contemplated under the waiver.
In addition, it should be noted that while Medicare Part D maintains a formulary system, Medicare Part B, which covers physician-administered prescription drugs including many forms of chemotherapy, are not subject to a formulary of any kind. Again, it is unclear from the text of the waiver whether physician-administered drugs would also be subject to the proposed formulary.
National Alliance of Mental Illness of Massachusetts: “Restrictive medication formulary threatens to harm people with mental health conditions”
NAMI Massachusetts opposes the Commonwealth’s proposal to adopt a restricted psychiatric medication formulary limited to at least one drug in each therapeutic class. For most people with mental health conditions, medication is a critical component of treatment and promotes stability and recovery.
Medications used to treat mental health conditions are not interchangeable, even in the same therapeutic class. The effectiveness of psychiatric medications is highly individualized and can vary substantially from person to person.vm Many psychotropic medications have strong side-effects, and tolerance of these effects is highly individual and variable.1x Overtime, people often require dosage adjustments, additional medication or a switch to more effective medication.x People with mental health conditions and their treating providers should be given the opportunity to work together to determine the most clinically effective, appropriate medication.
Restricting access to psychiatric medications is likely to create negative consequences for individuals with mental health conditions. A study by the American Psychiatric Association found that Medicaid recipients who had difficulty accessing medications were 3.6 times more likely to experience significant adverse events, including emergency room visits, repeat hospitalizations, incarceration and homelessness.
The Commonwealth’s proposal to adopt a restrictive medication formulary threatens to harm people with mental health conditions. Any realized cost-savings achieved up front will be undermined by higher costs incurred through disruptions in care, which may cause people’s conditions to worsen. Providing access to effective medications, as prescribed in close consultation with a mental health provider, helps people recover and become self-sufficient. Restricting access to psychiatric medications will ultimately shift costs to expensive hospital and crisis care, homeless services and the criminal justice system.
Depression and Bipolar Support Alliance: MassHealth Section 1115 Demonstration Amendment Request “would severely limit access to medications that are commonly used to treat mental health conditions”
The proposed changes would severely limit access to medications that are commonly used to treat mental health conditions and create serious challenges for people who have these mental health disorders. When it comes to the treatment of mental health conditions, the clinical management of real world patients often involves “trial and error.” Treating mental health conditions is not like treating pneumonia, where an oral antibiotic medicine is started and (in almost all instances) the illness goes away and the medication is stopped several weeks later. In contrast, the treatment of mental health conditions is almost always “trial and error,” and in many instances requires long-term treatment. In fact, the available published evidence suggests that only 25 percent to 33 percent of people who have a mental disorder experience a complete clinical response to the first two to three medications, even when prescribed in the presence of ongoing psychotherapy. Not allowing patients to be treated with access to all FDA-approved treatments for their condition is both cruel as well as costly.
National Council for Behavioral Health: “Restricting access to medications may have serious unintended consequences”
The National Council for Behavioral Health is a national, non-profit association of over 2,900 behavioral health provider organizations. Our members serve over 10 million adults, children and families with mental health and addiction disorders. A clear majority of their clients depend on Medicaid for their mental health care.
Restricting access to medications may have serious unintended consequences to both individuals’ health and to overall healthcare costs. Without appropriate access to the most effective and well-tolerated medications, persons with mental illness may experience instability-and at a high personal and fiscal cost: increased risk of hospitalization and emergency room visits, loss of employment, homelessness, and, too frequently, incarceration.
People with serious mental illness, especially those who depend on Medicaid, need access to qualified professionals and a full range of medications to make recovery possible. By restricting low-income patients’ access to psychiatric medications, preferred drug lists can limit recovery.
AIDS Action Committee of Massachusetts: MassHealth Section 1115 Demonstration Amendment Request “decrease access to high quality treatment for many PLWH”
Massachusetts currently leads the nation in our HIV care and prevention, in large part because of MassHealth policies. Our expansion of access to HIV medications has meant our population has achieved a 67% viral suppression rate, significantly higher than the national average. And because those that are virally suppressed are un-infectious to others, we have seen a nearly 50% decrease in new infections since we expanded MassHealth eligibility for those living with HIV in 2001. That remarkable progress was recently published as a model for the country, specifically citing our MassHealth access as critical to our success. The reduction in new infections has not only saved lives, it has saved the state an estimated $1.8 billion in avoided lifetime HIV treatment costs. Our success to date has even allowed us to envision our ultimate goal, which is getting to zero new infections, death, and HIV-related stigma.
First, creating a closed drug formulary under MassHealth would decrease access to high quality treatment for many PLWH. Currently, MassHealth covers all drugs from manufacturers that participate in the federal Medicaid rebate program. The closed formulary would restrict the drugs that MassHealth covers. This is especially concerning for PLWH. The current system allows providers to choose the best medication for each patient based on individual experiences with side effects and resistance profiles. Each individual HIV patient may react in unique ways to antiretroviral medications, and a patient and provider may change medications several times in order to find the ideal treatment regimen. A complicating factor is polypharmacy and drug-drug interactions. Many PLWH have comorbidities and take multiple medications. This is especially true of PLWH age 50 and older, who comprise more than half of PLWH in Massachusetts.
It is difficult to see how a formulary could take into account the complex HIV treatment guidelines. According to federal “Guidelines for the Use of Antiretroviral Agents in HIV-1- lnfected Adults and Adolescents,” a nearly 300 page document published by the U.S. Dept. of Health & Human Services Panel on Antiretroviral Guidelines for Adults and Adolescents, there are no fewer than six medication combinations recommended for patients who are first initiating treatment, and an additional table of options for patients based on other complications and comorbidities. There are separate guidelines for patients who have already had experience with HIV treatment but who may have developed various levels of resistance, and other subpopulations that have patticular considerations. These guidelines were revised twice in 2016 alone.
American Diabetes Association: “Strongly opposed… deep concerns” with MassHealth Section 1115 Demonstration Amendment Request
Because no single diabetes treatment regimen is appropriate for all people with diabetes, providers and patients should have access to a broad array of medications and supplies to develop an effective treatment modality. The Association is strongly opposed to the commonwealth’s proposal to implement a closed formulary for the MassHealth program. We are particularly concerned that important types of medications for people with diabetes could be left out if the commonwealth chooses to cover only one medication per therapeutic class.
Diabetes care should be patient-centered, and requires a close working relationship between the patient and clinicians involved in treatment. Developing an individual’s diabetes management plan should take into account the patient’s age, cognitive abilities, school/work schedule and conditions, health beliefs, support systems, eating patterns, physical activity, social situation, financial concerns, cultural factors , literacy and numeracy skills, diabetes complications, comorbidities, health priorities, other medical conditions, preferences for care, and life expectancy. As it relates to medication therapy, the Association’s Standards of Medical Care in Diabetes- 2017 identifies multiple factors which must be considered by a clinician when determining the appropriate medications for an individual with diabetes, including efficacy, risk of hypoglycemia, impact on weight, side effects, and cost. Implementing a closed formulary will severely hinder prescribers’ and MassHealth enrollees’ ability to choose the most appropriate diabetes medication(s) for the individual.
Should the commonwealth move forward with developing a closed formulary, the Association has deep concerns with the factors the state will consider when determining which drugs to exclude. Specifically, in the Amendment Request, the commonwealth requests flexibility to “exclude drugs with limited or inadequate clinical efficacy from its primary formulary.” Factors the commonwealth will consider when determining which drugs have limited or inadequate clinical efficacy include drugs for which “[o]nly surrogate endpoints have been reported.” For diabetes medications, efficacy is determined based on reduction of blood glucose levels, as measured by HbAlc. According to the Food and Drug Administration, HbAlc is the “primary endpoint of choice, albeit a surrogate.” So the “primary” endpoint used to assess the efficacy of diabetes medications is actually a surrogate endpoint. A policy to exclude from the MassHealth formulary drugs approved based solely on surrogate endpoints would negatively impact the availability of diabetes medications.
Epilepsy Foundation: “Epilepsy medications are not interchangeable and treatment of epilepsy is highly individualized”
…closed formularies could mean that Medicaid will not cover a medication that a physician prescribes for a patient. Limiting access to certain medications is dangerous to patients, especially individuals living with epilepsy, and it leads to higher overall health care costs. Epilepsy medications are not interchangeable and treatment of epilepsy is highly individualized. Epilepsy medications are lifesaving for individuals with epilepsy, and they help avoid unnecessary hospitalizations and ensure individuals can live well with epilepsy.
For the majority of people living with epilepsy, epilepsy medications are the most common and most cost-effective treatment for controlling and/or reducing seizures, and they must have meaningful and timely access to physician-directed care. There is no “one size fits all” treatment option for epilepsy, and the response to medications can be different for each person. Selection of the appropriate medication to prevent seizures is determined by a number of variables, including type of seizure, seizure frequency, age, gender, and other health conditions, and it requires careful evaluation and monitoring by physicians and their patients. A treating physician is in the best position to make the judgment about which medication is most appropriate.
American Academy of Dermatology Association: “Proposal will significantly impede access to medicine”
On behalf of the nearly 13,500 U.S.-based members of the American Academy of Dermatology Association (Academy), we are writing to express our opposition to Massachusetts’ proposed amendment to the state’s 1115 Medicaid waiver. This proposal will significantly impede access to medicine for those enrolled in MassHealth.
When a dermatologist sees a patient, the dermatologist evaluates a number of the patient’s individual characteristics to determine which drug is best for that patient. A formulary with only one drug per class is a one-size-fits-all solution that will lead to barriers or delays to necessary care. As a result of this policy change, several commonly used topical dermatologic drugs will likely be excluded from the MassHealth formulary. Therefore, this policy change risks both harming the patient and saddling them with undue economic burden.
Additionally, we are concerned that the proposal will increase the number of utilization management tools available for MassHealth formularies. Physicians and patients already cite experiencing difficulties with the appeals process and by enabling the use of additional utilization management tools, such as prior authorization, step therapy and tiering of formularies, patients will see increased delays in accessing their prescription drugs. If a patient with a chronic condition is stable on a drug and loses access due to utilization management tools, it can cause a flaring of the disease and diminish the effects of the very same treatment in the future. This loss of access can also contribute to increased medical costs when the condition is not stabilized with prescription drugs.
The Leukemia & Lymphoma Society: “Inappropriate to exclude a particular therapy” in Section 1115 Demonstration Amendment Request
LLS believes that both proposals could bring harm to patients, in particular those living with complex medical needs.
On a related note, LLS must take exception to language on page 9 of the Demonstration Amendment Request stating that “Many drugs coming to market through the FDA’s accelerated approval pathway have not yet proven their efficacy on primary endpoints in clinical trials.” It’s troubling that the Commonwealth seems to be implying that the FDA is approving therapies of such questionable value that a state must take matters into its own hands by establishing secondary review processes.
LLS believes it is inappropriate to exclude a particular therapy from the MassHealth formulary purely on the grounds that such therapy received accelerated approval. While LLS certainly appreciates the need for MassHealth resources to be spent on benefits and services of high value, adopting this as a blanket approach will no doubt prevent or delay some cancer patients from accessing medically appropriate-and potentially life-saving-therapies.
Further, LLS is alarmed at the prospect of a secondary, state-level review process; if adopted in multiple states, this process would result in dramatic variation in access to new cancer therapies across the country. Given the small population of cancer patients relying on these new medications and the importance of timely adherence to treatment regimen, LLS believes the creation of a closed formulary, without a clear and robust exception process would be harmful to blood cancer patients.
The Jett Foundation: “Barrier to hope for Duchenne boys”
Certain provisions within the Proposed Amendment to the MassHealth Section 1115 Demonstration would, if enacted, greatly inhibit the ability of Massachusetts residents with Duchenne to gain access to potentially life-saving therapies that could improve their quality of life… This waiver if granted will dramatically hinder the ability of Duchenne patients and medical experts to execute care plans that could improve the health and quality of life of Duchenne patients.
Given that Duchenne is such a rare and devastating disorder where the majority of the population lives without a disease modifying therapy, FDA will likely continue to use accelerated approval in an effort to treat the entire Duchenne population with drugs that are safe and appear to be efficacious. The provisions within the proposed Amendment to the MassHealth Section 1115 Demonstration would create a barrier to access for patients hoping to gain access to those future therapies.
Importantly, at Jett Foundation we speak for the patients; not the pharmaceutical or the health insurance industry, and it is the Duchenne patients residing in Massachusetts who will truly suffer dire consequences of the proposed Amendment to the MassHealth Section 1115 Demonstration. It is not the role of EOHHS, or any other payer to make decisions on whether patients can access potentially life-saving FDA approved treatments treatments, it is the role the of the FDA, expert clinicians, and the patients and families to weigh the benefits and risks of trying an FDA approved treatment.
National Organization for Rare Disorders: Section 1115 Demonstration Amendment Request “devastating impact on the health and well-being of rare disease patients”
The enactment of these changes would have a devastating impact on the health and well-being of rare disease patients. NORD has seen firsthand how “commercial-style” formulary restrictions overrule the prescribing decisions of physicians thus resulting in patients being unable to access the medicines best suited to treat their condition.
As a result, such restrictions inhibit quality care by causing lapses in medication adherence and delays in use of medicines that provide an enhanced clinical benefit. Over time, this will not only result in poorer health outcomes for MassHealth beneficiaries but raise health care costs for the state.
The National MS Society opposes a closed prescription drug formulary and maintains that all FDA-approved MS DMTs should be made available in health plan drug formularies. Studies show that early and ongoing treatment with a DMT is the best way to modify the course of the disease, prevent the accumulation of disability and protect the brain from damage due to MS.
The complexity and uncertainty of MS makes identifying an effective treatment following diagnosis and staying on that treatment essential. Physicians’ clinical judgement in the treatment decision process must be upheld in the best interest of the patient’s well-being. It is also recommended that MS treatments not be changed unless a medical reason necessitates a switch. Patients switching medications should only occur due to a sub-optimal treatment response, intolerable side effects, and inadequate adherence to the treatment regimen.