Specialty Pharmacy

With More AML Therapies Available, Payers Apply Various Management Tactics

September 19, 2019

The FDA has approved nearly 10 therapies for acute myeloid leukemia (AML) over the past couple of years. Because most of them target a specific biomarker, it’s critical that people diagnosed with the condition undergo genetic testing to determine whether they fall into a particular patient subgroup.

“Prior to two years ago, we had no new drugs for over a decade, and now we have eight new drugs approved in just the last two years, so the whole field has changed,” said Daniel J. DeAngelo, M.D., Ph.D., chief, division of leukemia, institute physician, professor of medicine, Harvard Medical School, in an interview published on the website obroncology.com.

By Angela Maas

The FDA has approved nearly 10 therapies for acute myeloid leukemia (AML) over the past couple of years. Because most of them target a specific biomarker, it’s critical that people diagnosed with the condition undergo genetic testing to determine whether they fall into a particular patient subgroup.

“Prior to two years ago, we had no new drugs for over a decade, and now we have eight new drugs approved in just the last two years, so the whole field has changed,” said Daniel J. DeAngelo, M.D., Ph.D., chief, division of leukemia, institute physician, professor of medicine, Harvard Medical School, in an interview published on the website obroncology.com.

Many of the newer drugs are oral formulations, which, “in general, are easier to administer,” points out Mesfin Tegenu, R.Ph., president of PerformRx, LLC. “Rather than having to go into a hospital or clinic for treatment, a patient can simply take a medication orally for their condition.”

Payers utilize a variety of management tactics with AML therapies. “Payers often require prior authorization of these therapies due to safety, concern for off-label usage and cost,” says Tegenu. A variety of drugs are used off-label for certain patient populations, he notes.

Asked if AML is a condition suited for value-based contracting, Tegenu asserts that “all therapies associated with high cost should have some kind of value-based payment models to make drug manufacturers an integral part of the health care delivery system. We plan to initiate this discussion with all leading pharmaceutical companies.”

According to Winston Wong, Pharm.D., president of W-Squared Group., the newer drugs would be better candidates for such deals due to AML’s heterogenicity and the fact that “the treatment foundation is still conventional chemotherapy, which for the most part is available as a generic.”

Novartis’ Zolgensma Crisis May Not Impact Pickup

September 11, 2019

The FDA in August put out a statement addressing “data accuracy issues” with Zolgensma (onasemnogene abeparvovec-xioi), a new gene therapy to treat spinal muscular atrophy in people less than two years old who have bi-allelic mutations in the survival motor neuron 1 gene.

The FDA approved the drug from AveXis, Inc., which was acquired by Novartis AG last year, on May 24. On June 28, AveXis notified the FDA that there was “a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA.”

By Angela Maas

The FDA in August put out a statement addressing “data accuracy issues” with Zolgensma (onasemnogene abeparvovec-xioi), a new gene therapy to treat spinal muscular atrophy in people less than two years old who have bi-allelic mutations in the survival motor neuron 1 gene.

The FDA approved the drug from AveXis, Inc., which was acquired by Novartis AG last year, on May 24. On June 28, AveXis notified the FDA that there was “a data manipulation issue that impacts the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA) and reviewed by the FDA.”

The FDA said Novartis learned about the issue March 14, but rather than informing the agency, which was assessing Zolgensma’s BLA at the time, the company conducted its own internal investigation, reporting its findings to the FDA after the investigation was completed. Still, the agency said that it “remains confident that Zolgensma should remain on the market.”

The one-time therapy is the most expensive drug in the world, with a price tag of $2.1 million.

In response, Novartis issued a press release Aug. 6 that read, in part, “we maintain that the totality of the evidence demonstrating the product’s effectiveness and its safety profile continue to provide compelling evidence supporting an overall favorable benefit-risk profile.”

Novartis recently said that Zolgensma has coverage plans from payers representing 40% of commercial lives, and high-profile news reports name Aetna Inc. and Anthem, Inc. among insurers expanding their policies to cover more patients. So will the current situation have any impact on pickup among payers and providers?

Based on the FDA’s statement, “I expect there will be no more than a minor and temporary blip in pickup of Zolgensma among providers and payers,” says Elan Rubinstein, Pharm.D., principal at EB Rubinstein Associates.

More Than 1,000 RM/AT Products Are in Pipeline

August 15, 2019

This past quarter saw two new gene therapies: Novartis AG subsidiary AveXis, Inc.’s Zolgensma (onasemnogene abeparvovec-xioi) received FDA approval May 24 for the treatment of spinal muscular atrophy, and bluebird bio’s Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) received conditional marketing authorization from the European Commission for transfusion-dependent beta thalassemia.

While only a handful of therapies in the broader regenerative medicine/advanced therapy (RM/AT) space are available globally, a new report shows that is likely to change, as there are more than 1,000 products in the pipeline.

By Angela Maas

This past quarter saw two new gene therapies: Novartis AG subsidiary AveXis, Inc.’s Zolgensma (onasemnogene abeparvovec-xioi) received FDA approval May 24 for the treatment of spinal muscular atrophy, and bluebird bio’s Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) received conditional marketing authorization from the European Commission for transfusion-dependent beta thalassemia.

While only a handful of therapies in the broader regenerative medicine/advanced therapy (RM/AT) space are available globally, a new report shows that is likely to change, as there are more than 1,000 products in the pipeline.

The Alliance for Regenerative Medicine published the report, titled Quarterly Global Regenerative Medicine Sector Report: Q2 2019, on Aug. 1. It shows there are 1,069 clinical trials using specific RM/AT technologies, which include gene therapy, gene-modified cell therapy, cell therapy and tissue engineering. Ninety-four of those products are in Phase III trials.

While Zolgensma’s $2.125 million price for a one-time infusion makes it the costliest drug on the planet, many other newer RM/AT products are certainly not cheap. Though many manufacturers are offering various reimbursement schemes, including rebates if a therapy doesn’t work, other outcomes-based deals and multiyear pay-over-time payment options, experts note that many barriers exist in the execution of these strategies.

“In principle, spreading the cost over a five-year period and putting the cost installments at risk based upon efficacy is a good approach,” says Winston Wong, Pharm.D., president of W-Squared Group, about Zolgensma. “However, the devil is in the details. Do we have the systems in place that have the capability to administer a five-year contract?”

According to Wong, “From the manufacturer perspective, a value-based contract implies that no payment would be made if the patient relapses or passes on. Systems are not in place to have the ability to track the patient once therapy is administered.”

Copay Accumulators’ Use Rises; Virginia Passes Law Banning Such Programs

April 24, 2019

Payers are continuing to implement copay accumulators and copay maximizers in an effort to counter copay assistance from pharmaceutical manufacturers, according to a recent survey.

A Zitter Insights report shows that more than 90 million commercial lives are covered by payers that have a copay accumulator program — and it doesn’t look like these arrangements are going away any time soon.

By Angela Maas

Payers are continuing to implement copay accumulators and copay maximizers in an effort to counter copay assistance from pharmaceutical manufacturers, according to a recent survey.

A Zitter Insights report shows that more than 90 million commercial lives are covered by payers that have a copay accumulator program — and it doesn’t look like these arrangements are going away any time soon.

It also explores the use of a similar kind of program: copay maximizers. Among 51 payer respondents covering 177.9 million lives, payers covering more than 50 million commercial lives have implemented a copay maximizer program. But payers representing 58% of covered lives say they do not have plans to incorporate maximizers within their benefits offerings.

Melinda Haren, a senior consultant at Zitter Insights, notes that accumulators and maximizers are focused on specialty drugs, particularly those products that are adjudicated through the pharmacy benefit.

The savings for payers — and the additional burden on manufacturers — are not insignificant. But drugmakers can do little to limit the use of these programs, at least in the short term, says Haren.

One recent effort may prove to be effective against accumulators: On March 21, Virginia Gov. Ralph Northam (D) signed a law that will go into effect Jan. 1, 2020, which states, in part, “When calculating an enrollee’s overall contribution to any out-of-pocket maximum, deductible, copayment, coinsurance, or other cost-sharing requirement under a health plan, a carrier shall include any amounts paid by the enrollee or paid on behalf of the enrollee by another person.”

This approach to counter accumulators “will likely be effective, at least in the near term,” maintains Jeremy Schafer, Pharm.D., senior vice president of payer access solutions at Precision for Value.

Drugmakers Strategize to Sidestep Copay Accumulator Programs

April 8, 2019

As more health plans are implementing copay accumulator programs, manufacturers are struggling to find ways to counter them.

By Angela Maas

As more health plans are implementing copay accumulator programs, manufacturers are struggling to find ways to counter them.

Drugmakers may want to “evaluate [their specialty pharmacy] network and consider narrowing it to independent SPs that will accept contracts precluding sharing with payers/PBMs the patients who are participating in your copay offset program,” recommends a recent survey report from Zitter Insights.

To make sure patients are getting the savings intended, manufacturers could allocate “fewer or no dollars to rebates and more dollars to coupons,” says Elan Rubinstein, Pharm.D., principal at EB Rubinstein Associates. “If the CMS safe harbor proposal is finalized as proposed, and if it becomes effective in January 2020 as proposed, this is in my view an approach that manufacturers will consider.”

However, according to Lisa Kennedy, Ph.D., chief economist at Epiphany, “it is hard to pull back rebates in a highly competitive area especially where there are limited formulary places versus where a manufacturer is in a segment where they have greater capacity for negotiation.”

Some drugmakers are offering patients debit cards to sidestep these programs, as it is more difficult to track these than, say, a coupon that a pharmacy must process, says Melinda Haren, a senior consultant at Zitter Insights. Companies also could offer rebate programs by which the patient pays out-of-pocket for a drug and then is reimbursed.

A few manufacturers are speaking with payers about contracting to exclude their drugs from these programs. But some legal risk exists with this approach because “money paid to patients doesn’t count toward the calculation for best price because it’s going to the patient, not the payer,” says Haren. In contrast, when manufacturers contract with payers, those details are “likely reportable under best price,” she says, acknowledging that this is a “gray area” for drugmakers.

2018 Saw Slowing M&A Activity; Expect More of the Same in ’19

January 24, 2019

The specialty pharmacy and infusion therapy spaces have certainly seen their share of merger and acquisition (M&A) activity over the years. Some challenges within those industries may have helped slow down 2018 activity a bit, observes Reg Blackburn, managing director at The Braff Group. And for 2019, we may see more of the same.

As far as specialty pharmacy trends in 2018, Blackburn points out that “the largest specialty pharmacies continue to get even larger. Payer- and chain-owned dominate. Most new entity growth is coming from large academic hospitals starting their own specialty pharmacies.”

Direct and indirect remuneration (DIR) fees in Medicare Part D that include rebates and price concessions occurring after the point of sale have been around since the start of that program more than a decade ago.

By Angela Maas

The specialty pharmacy and infusion therapy spaces have certainly seen their share of merger and acquisition (M&A) activity over the years. Some challenges within those industries may have helped slow down 2018 activity a bit, observes Reg Blackburn, managing director at The Braff Group. And for 2019, we may see more of the same.

As far as specialty pharmacy trends in 2018, Blackburn points out that “the largest specialty pharmacies continue to get even larger. Payer- and chain-owned dominate. Most new entity growth is coming from large academic hospitals starting their own specialty pharmacies.”

Direct and indirect remuneration (DIR) fees in Medicare Part D that include rebates and price concessions occurring after the point of sale have been around since the start of that program more than a decade ago.

But they started really becoming an issue for specialty drugs around 2016, and it doesn’t look like that’s changing any time soon.

Within the specialty pharmacy space, M&A activity “was lower than in past years,” he explains. Through third-quarter 2018, The Braff Group recorded eight specialty pharmacy deals, compared with 18 for full-year 2017, 20 for 2016 and 10 for 2015.

This lower volume, he says, “is driven by fewer acquisition targets of a size large enough to move-the-needle for strategic buyers and/or large enough to be a platform for PE [i.e., private-equity] buyers. In addition, there is caution from both buyer types given the impact of DIR fees on gross margin.”

Moving forward into 2019, Blackburn expects to see “continued consolidation at a measured pace.” In addition, he says, “smaller independents will remain under pressure for gross margin and closed networks. They will want to exit, but buyers will be limited.”

Within the infusion therapy space over the past year, observes Blackburn, intravenous immune globulin and other specialty infusion products continue to drive revenue growth.

The Braff Group recorded four infusion therapy deals through the third quarter of last year, compared with seven in 2017, six in 2016 and 14 in 2015. 2018, says Blackburn, “continued the recent trend of low deal flow. The sector has experienced significant consolidation over the past five to 10 years.”

In the new year, Blackburn expects to see “continued modest deal activity primarily because of low inventory.”