Product Release

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.

Radar On Market Access: Orphan Drug Act Has Been Overused, A New Study Shows

March 18, 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.

“We have so many patient advocacy groups — they are representing rare disease patients. And many of those advocacy groups are heavily sponsored by drug manufacturers,” Bai says. “You cannot just single out the drugs that are being most abused — because even those drugs do have orphan drug features….It’s very hard to disentangle.”

Similarly, Avalere Health’s Lilian Buch and David Kowalsky observed during a Feb. 26 podcast that the orphan drug advocacy work that manufacturers coordinate with patients does serve an important purpose and can improve patient outcomes on an individual basis. Kowalsky is director of the consulting firm’s commercialization and regulatory strategy practice, while Buch is an associate principal in the same practice.

Buch added that, for some rare disease patients with acute symptoms, drug manufacturers sometimes offer the kind of support services more often provided by plans or providers.

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.”

Radar On Market Access: FDA’s Breyanzi Approval May Not Change NHL Management

March 9, 2021

With the FDA’s approval of Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) last month, there are now three chimeric antigen receptor T cell (CAR-T) therapies to treat a certain type of non-Hodgkin’s lymphoma (NHL). A Zitter Insights poll shows that payers do not anticipate its approval as having much of an impact on their management of the space, AIS Health reported.

On Feb. 5, the FDA approved Breyanzi for the treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma and follicular lymphoma grade 3B.

With the FDA’s approval of Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) last month, there are now three chimeric antigen receptor T cell (CAR-T) therapies to treat a certain type of non-Hodgkin’s lymphoma (NHL). A Zitter Insights poll shows that payers do not anticipate its approval as having much of an impact on their management of the space, AIS Health reported.

On Feb. 5, the FDA approved Breyanzi for the treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma and follicular lymphoma grade 3B.

Breyanzi joins Novartis Pharmaceuticals Corp.’s Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) from Gilead Sciences Inc. unit Kite Pharma, Inc. as the three CAR-T therapies in NHL.

For the Managed Care Oncology Index: Q2 2020, between June 1, 2020, and June 30, 2020, Zitter Insights polled 51 commercial payers with 129.6 million covered lives. Those with 96% of lives anticipated that they would manage Breyanzi to label. Asked how impactful its introduction into NHL would be for their management of the indication, payers with 71% of lives said it would have either no impact or minimal impact, while 28% said it would have a moderate impact.

“All three CAR-T therapies have the same approved indication for relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy,” points out Winston Wong, Pharm.D., president of W-Squared Group. “I believe…that unless the managed care organization has a pathway program in place, there will be few organizations that will manage the selection of any of the CAR-T agents for the lymphoma indication….Since efficacy and safety appear to be similar across the three CAR-T agents, it will come down to cost.”

Breyanzi launched with a list price of $410,300 for the one-time treatment compared with $373,000 for Yescarta and Kymriah in NHL.

Wong adds that “it is my expectation that the oncology practice will be the deciding party to choose a preferred CAR-T, based upon experience and cost to the practice.”

Zitter Insights also polled 102 oncologists between June 1, 2020, and June 30, 2020. Approximately half said they did not anticipate administering Breyanzi, slightly more than those with the same response for Yescarta and Kymriah.

Radar On Market Access: New Heart Failure Drugs Offer More Therapy Options

March 2, 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 target for Colanor is to slow down heart rate,” Tegenu explains, adding that Corlanor typically is placed on formularies as a preferred or non-preferred brand, and may be started when the patient’s condition is not being adequately controlled on optimally dosed beta blocker therapy.

Entresto and Corlanor represent advances in treatment for chronic heart failure, Tegenu says. “Both therapies were poised to change the landscape of treatment for patients with heart failure, given their unique mechanisms of action,” he says.

Health plans also are turning to targeted disease and care management programs to focus on preventing hospital readmission, reducing mortality and reducing costs in heart failure, Tegenu adds.