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Trends That Matter for Partial Orphan Drugs

April 8, 2021

A new study published in Health Affairs found that spending in the orphan drug category is overwhelmingly concentrated on so-called partial orphan drugs, which have both orphan and nonorphan indications. The study affirms growing concerns across the health care industry that drugmakers are misusing the orphan drug designation and introducing unwarranted cost into the drug channel, AIS Health reported.

The Orphan Drug Act of 1983 covers diseases that affect fewer than 200,000 people in the U.S., plus diseases that affect more than 200,000 people but are so expensive to treat that companies developing and marketing such therapies are not expected to recover their costs. With the designation, the FDA grants drugmakers expanded intellectual property and commercial rights intended to offset these steep costs.

A new study published in Health Affairs found that spending in the orphan drug category is overwhelmingly concentrated on so-called partial orphan drugs, which have both orphan and nonorphan indications. The study affirms growing concerns across the health care industry that drugmakers are misusing the orphan drug designation and introducing unwarranted cost into the drug channel, AIS Health reported.

The Orphan Drug Act of 1983 covers diseases that affect fewer than 200,000 people in the U.S., plus diseases that affect more than 200,000 people but are so expensive to treat that companies developing and marketing such therapies are not expected to recover their costs. With the designation, the FDA grants drugmakers expanded intellectual property and commercial rights intended to offset these steep costs.

The new paper found that 70.7% of spending on drugs designated as orphan drugs went “to nonorphan indications,” and noted that in 2017, 25% of U.S. prescription drug spending was for orphan drugs.

Ge Bai, Ph.D., an associate professor at Johns Hopkins University’s Carey Business School and Bloomberg School of Public Health, asserts that the orphan drug designation is being abused by drug companies.

“They [orphan drugs] treat a small number of patients, but at the same time they can be used to treat much more common diseases. So they [drugmakers] can still charge a high price and take the tax credit, while at the same time enjoying their impressive commercial success,” Bai says.

Bai adds that drugmakers have become adept at coordinating outside pressure to convince the FDA to expand orphan drug designations beyond their intended purpose.

Trends That Matter for New Heart Failure Drugs

March 25, 2021

Treatment for heart failure still relies significantly on tried-and-true generic drugs, but new brand-name entrants — including Novartis’ Entresto (sacubitril/valsartan) and Amgen’s Corlanor (ivabradine) — are important additions to prescribers’ clinical arsenals against the high-mortality condition, industry insiders tell AIS Health.

“Generic heart failure drugs, including beta blockers, ACE inhibitors, and ARBs [angiotension receptor blockers] have historically been used and continue to be the backbone of therapy,” says April Kunze, Pharm.D., senior director of clinical program development for Prime Therapeutics. “However, in the past few years, additional treatment options have become available. Entresto is now recommended as a first-line treatment option in patients with an ejection fraction <= 40%."

Treatment for heart failure still relies significantly on tried-and-true generic drugs, but new brand-name entrants — including Novartis’ Entresto (sacubitril/valsartan) and Amgen’s Corlanor (ivabradine) — are important additions to prescribers’ clinical arsenals against the high-mortality condition, industry insiders tell AIS Health.

“Generic heart failure drugs, including beta blockers, ACE inhibitors, and ARBs [angiotension receptor blockers] have historically been used and continue to be the backbone of therapy,” says April Kunze, Pharm.D., senior director of clinical program development for Prime Therapeutics. “However, in the past few years, additional treatment options have become available. Entresto is now recommended as a first-line treatment option in patients with an ejection fraction <= 40%.”

Novartis said Feb. 16 that Entresto won an expanded indication from the FDA to reduce the risk of cardiovascular death and hospitalization in adult patients with chronic heart failure. “Benefits are most clearly evident in patients with left ventricular ejection fraction (LVEF) below normal,” the drugmaker said.

Prime Therapeutics currently prefers Entresto on formulary, and the PBM recommends that plans remove prior authorizations for Entresto in order to encourage its use, Kunze says.

Mesfin Tegenu, CEO and chairman of RxParadigm, says that Entresto, which has an average retail price of around $600 per month, typically is placed on formularies as a preferred brand drug. Meanwhile, Amgen’s Corlanor can be beneficial in reducing heart failure-associated hospitalization for patients with symptomatic (NYHA Class II-III) stable chronic heart failure with a left ventricular ejection fraction of less than or equal to 35% who are receiving a maximal tolerated targeted dose of a beta blocker and in sinus rhythm with a heart rate of 70 beats per minute or greater at rest, Tegenu says.

The graphic below show how key chronic heart failure medications are covered among commercial health plans, health exchange programs, Medicare and Medicaid programs under the pharmacy benefit.

Trends That Matter for New Prostate Cancer Treatment

March 11, 2021

People being treated for advanced prostate cancer now have a new oral option. On Dec. 18, the FDA approved Myovant Sciences GmbH’s Orgovyx (relugolix), a gonadotropin-releasing hormone (GnRH) receptor antagonist, to treat adults with advanced prostate cancer, AIS Health reported. Dosing for the drug is a loading dose of 360 mg on the first day of treatment and then 120 mg of the tablet once daily.

Treatment of advanced prostate cancer usually involves androgen deprivation therapy (ADT), and the current standard of care is luteinizing hormone-releasing hormone (LHRH) receptor agonists, such as leuprolide acetate, which is administered by a health care provider as an injection or implant. It is available as AbbVie Inc.’s Lupron Depot and Eligard from Tolmar Pharmaceuticals, Inc. to treat prostate cancer.

People being treated for advanced prostate cancer now have a new oral option. On Dec. 18, the FDA approved Myovant Sciences GmbH’s Orgovyx (relugolix), a gonadotropin-releasing hormone (GnRH) receptor antagonist, to treat adults with advanced prostate cancer, AIS Health reported. Dosing for the drug is a loading dose of 360 mg on the first day of treatment and then 120 mg of the tablet once daily.

Treatment of advanced prostate cancer usually involves androgen deprivation therapy (ADT), and the current standard of care is luteinizing hormone-releasing hormone (LHRH) receptor agonists, such as leuprolide acetate, which is administered by a health care provider as an injection or implant. It is available as AbbVie Inc.’s Lupron Depot and Eligard from Tolmar Pharmaceuticals, Inc. to treat prostate cancer.

For the Managed Care Oncology Index: Q3 2020, between Aug. 26, 2020, and Sept. 30, 2020, Zitter surveyed 50 commercial payers about their anticipated management of Orgovyx within six months of availability. Those with 89% of covered lives said they were likely to manage the drug to label.

During the same time frame, Zitter surveyed 100 oncologists, and 77% said they were likely to prescribe Orgovyx, with 62% saying they were likely to prescribe the new drug over leuprolide acetate. Almost half of respondents indicated they are likely to transition patients on leuprolide acetate to Orgovyx.

Winston Wong, Pharm.D., president of W-Squared Group, says that he expects Orgovyx to have “significant uptake for several reasons. Even though it is taken daily, it is easier for the patient to be treated when compared to the patient having to travel to the physician office to receive their injection.”

He says he expects prescriptions to drop for Lupron and Eligard. “Possibly the remaining niche for either of these injectable drugs would be for the traditional Medicare patient who does not have a prescription plan through Part D or a Medicare Advantage plan.”

Trends That Matter for Rx Benefits in Medicaid

February 25, 2021

Beginning in April, California and New York will join a growing list of states that have opted to carve out prescription drug benefits from their Medicaid contracts with insurers, wagering that the state can do a better job at negotiating drug prices with manufacturers than managed care organizations and their contracted PBMs, AIS Health reported.

“I think the pharmacy benefit overall will be something that states are looking at in order to find savings in some way — whether through carveouts or through another policy — just because there are limited levers that the state is going to be able to pull to save money,” says Rachel Dolan, a senior policy analyst with the Kaiser Family Foundation’s Program on Medicaid and the Uninsured.

Beginning in April, California and New York will join a growing list of states that have opted to carve out prescription drug benefits from their Medicaid contracts with insurers, wagering that the state can do a better job at negotiating drug prices with manufacturers than managed care organizations and their contracted PBMs, AIS Health reported.

“I think the pharmacy benefit overall will be something that states are looking at in order to find savings in some way — whether through carveouts or through another policy — just because there are limited levers that the state is going to be able to pull to save money,” says Rachel Dolan, a senior policy analyst with the Kaiser Family Foundation’s Program on Medicaid and the Uninsured.

Other states that have carved out their drug benefits from managed care contracts include Missouri, North Dakota, Tennessee, West Virginia and Wisconsin, and Nevada plans to carve out the prescription drug benefit in fiscal year 2023 when its MCO contracts are renewed.

One catalyst for the carveouts may be the increasing scrutiny on spread pricing, which occurs when PBMs charge payers more for prescription drugs than the amount they reimburse pharmacies and retain the difference.

“It also allows the state more control over their formulary and their payment policy, and it can also help them potentially negotiate more supplemental rebates with manufacturers if basically all the drugs are in one big pool covered by the state,” Dolan adds.

For Medicaid MCOs, there are a host of important considerations when states opt to carve out their pharmacy benefits, notes Brian Anderson, a principal with Milliman. For example, such a move will generally hinder MCOs’ ability to influence prescribing practices, which “have a profound impact on drug utilization and drug mix,” he says. Pharmacy networks are another key issue, as MCOs often use closed networks but some states have any-willing-provider regulations for pharmacy participation in networks.

Trends That Matter for Prevention of HAE Attacks

February 11, 2021

When the FDA approved BioCryst Pharmaceuticals, Inc.’s Orladeyo (berotralstat) in December 2020, the drug became the first oral treatment for prophylaxis to prevent hereditary angioedema (HAE) attacks, AIS Health reported. According to Zitter Insights, payers with nearly three-quarters of covered lives plan to manage it at parity to other prophylactic treatments.

The FDA approved the first drug to treat HAE, Shire plc’s Cinryze (C1 esterase inhibitor [human]), on Oct. 10, 2008. Since then, the FDA has approved eight drugs to treat HAE: half for acute attacks and half for prophylaxis.

When the FDA approved BioCryst Pharmaceuticals, Inc.’s Orladeyo (berotralstat) in December 2020, the drug became the first oral treatment for prophylaxis to prevent hereditary angioedema (HAE) attacks, AIS Health reported. According to Zitter Insights, payers with nearly three-quarters of covered lives plan to manage it at parity to other prophylactic treatments.

The FDA approved the first drug to treat HAE, Shire plc’s Cinryze (C1 esterase inhibitor [human]), on Oct. 10, 2008. Since then, the FDA has approved eight drugs to treat HAE: half for acute attacks and half for prophylaxis.

Orladeyo will compete against Cinryze, Haegarda (C1 esterase inhibitor [human]) and Takhzyro (lanadelumab-flyo) to prevent HAE attacks.

For the Managed Care Biologics and Injectables Index: Q1 2020, between Feb. 25, 2020, and April 1, 2020, Zitter surveyed 51 commercial payers with 138.1 million covered lives about their anticipated management of Orladeyo. Payers covering 93% of lives said they likely would manage it to label and not allow off-label use.

Among 46 commercial payers with 130.3 million covered lives surveyed during the same time period, payers with 74% of covered lives said they were likely to manage Orladeyo at parity to the prophylactic therapies. And even though it is the only oral HAE drug, payers with 80% of lives said they were unlikely to manage it separately from the other drugs based on route of administration.

Among providers who would transition current patients to Orladeyo, the most likely prophylactic agent to transfer from was Cinryze, cited by 42%; followed by Haegarda, at 25%; and Takhzyro at 8%.

Trends That Matter for Epilepsy Medications’ Market Access

January 28, 2021

Pharmaceutical treatment for different types of epilepsy generally still relies on tried-and-true generics, despite recent efforts by drug manufacturers to introduce new branded medications into the mix, PBM insiders tell AIS Health.

Xcopri (cenobamate tablets), manufactured by SK Biopharmaceuticals Co., Ltd.’s subsidiary SK Life Science, Inc., launched in May for the treatment of partial-onset seizures. However, many plans haven’t jumped to add Xcopri to their formularies, says Mesfin Tegenu, R.Ph., president of PerformRx.

Pharmaceutical treatment for different types of epilepsy generally still relies on tried-and-true generics, despite recent efforts by drug manufacturers to introduce new branded medications into the mix, PBM insiders tell AIS Health.

Xcopri (cenobamate tablets), manufactured by SK Biopharmaceuticals Co., Ltd.’s subsidiary SK Life Science, Inc., launched in May for the treatment of partial-onset seizures. However, many plans haven’t jumped to add Xcopri to their formularies, says Mesfin Tegenu, R.Ph., president of PerformRx.

“Some plans have opted to take a cautious approach and leave the medication as non-formulary to start,” Tegenu says. “It is difficult to tell the impact of this new drug launch on the treatment of epilepsy. However, Xcopri trials demonstrated high efficacy in partial onset seizures and refractory epilepsy, lending it a strong clinical profile. One could reasonably suspect a high impact on the epilepsy treatment paradigm.”

In most cases, though, generics are the first-line treatments for many forms of the disorder, according to Tegenu. Many of the drugs used to treat epilepsy are covered without restriction by plans.

Prime Therapeutics LLC treats Xcopri as a non-preferred brand, says April Kunze, senior director of clinical formulary development and trend management strategy for the PBM. Premera Blue Cross’ Medicare Advantage formularies, Cigna Corp.’s national preferred formulary and HealthPartners’ commercial formularies impose quantity limits on Xcopri, according to their plan documents.

“Treatment is based on the type of epilepsy diagnosed, and labeled and off-label indications of the individual products. There is a fair amount of overlap as many drugs share multiple indications. However, many newer agents are narrowly indicated,” Tegenu says.

The graphic below show how epilepsy medications are covered among commercial health plans, health exchange programs, Medicare and Medicaid programs under the pharmacy benefit.