Radar on Specialty Pharmacy

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.”

Plans Must Cover PrEP at No Cost After USPSTF Recommendation

July 11, 2019

A few recent actions, as well as one expected next year, are expected to help bring down rates of HIV infection. And while the efforts should make it easier for certain populations to gain access to preexposure prophylaxis (PrEP) that helps prevent them from acquiring HIV, some issues are likely to stand in the way of eliminating all infections.

The FDA initially approved Gilead Sciences, Inc.’s Truvada (emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg) in 2004 to treat HIV infection. But in 2012, the agency approved it to reduce the risk of HIV infection in adults, the only therapy indicted for this use.

By Angela Maas

A few recent actions, as well as one expected next year, are expected to help bring down rates of HIV infection. And while the efforts should make it easier for certain populations to gain access to preexposure prophylaxis (PrEP) that helps prevent them from acquiring HIV, some issues are likely to stand in the way of eliminating all infections.

The FDA initially approved Gilead Sciences, Inc.’s Truvada (emtricitabine 200 mg/tenofovir disoproxil fumarate 300 mg) in 2004 to treat HIV infection. But in 2012, the agency approved it to reduce the risk of HIV infection in adults, the only therapy indicted for this use.

In its final recommendation, published June 11, the U.S. Preventive Services Task Force (USPSTF) recommended that “clinicians offer preexposure prophylaxis (PrEP) with effective antiretroviral therapy to persons who are at high risk of HIV acquisition,” giving the recommendation an “A” rating. This grade, the highest one possible, means that the USPSTF recommends the service, as “there is high certainty that the net benefit is substantial.”

The grade also means that, per the Affordable Care Act, “a group health plan and a health insurance issuer offering group or individual health insurance coverage” must provide coverage for free of items or services that the USPSTF gives an “A” or “B” ranking.

Payers will still be able to impose “reasonable medical management techniques,” such as prior authorization, notes Elan Rubinstein, Pharm.D., principal, EB Rubinstein Associates.

“Making PrEP available without cost-sharing eliminates a major barrier to this landmark HIV prevention tool,” said Michael Ruppal, executive director of The AIDS Institute. “At a time when out-of-pocket costs are rising for patients as they seek access to medications, this recommendation is a win both for patients and public health.”

With Zolgensma Adding to SMA Options, Payers Are Likely to Apply Various Management Tactics

June 12, 2019

With the first therapy north of $1 million gaining FDA approval last month, payers likely will implement a variety of strategies to manage Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene therapy from AveXis, Inc., a Novartis company. For now, many uncertainties exist with respect to the new treatment and what its place will be in spinal muscular atrophy (SMA) care.

According to Lee Newcomer, M.D., principal at Lee N. Newcomer Consulting LLC, payers likely will implement “strict enforcement of the label restrictions, advisory panels to develop clinical criteria for therapy [and] highly specific provider networks to ensure that the therapies are given correctly.”

By Angela Maas

With the first therapy north of $1 million gaining FDA approval last month, payers likely will implement a variety of strategies to manage Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene therapy from AveXis, Inc., a Novartis company. For now, many uncertainties exist with respect to the new treatment and what its place will be in spinal muscular atrophy (SMA) care.

According to Lee Newcomer, M.D., principal at Lee N. Newcomer Consulting LLC, payers likely will implement “strict enforcement of the label restrictions, advisory panels to develop clinical criteria for therapy [and] highly specific provider networks to ensure that the therapies are given correctly.”

When it comes to payer strategies, “unlike other conditions, where there might be alternatives that warrant prior authorization or step therapy, here I expect there to be limited management and more care coordination,” Alex Shekhdar, founder of Sycamore Creek Healthcare Advisors, tells AIS Health.

“If I were a payer, I would definitely use prior-authorization for this, but given the nature of the therapy, you can’t use step edits — it doesn’t really make sense clinically,” says an industry expert who declines to be identified. “But prior authorization really needs to occur extremely quickly.”

“Plans will likely implement a medical policy to ensure appropriate use and ensure adequate monitoring,” says April M. Kunze, Pharm.D., senior director, clinical formulary development and trend management strategy at Prime Therapeutics LLC. “This is a one-time IV infusion; any repeated administration has not been evaluated and will likely not be covered until there is data to support that use.”

One concern among some payers is whether members will attempt to be treated with both Zolgensma and Spinraza (nusinersen), another expensive therapy for SMA. “It’s not clear yet if the combination or the sequence is better care until further studies are completed,” says Newcomer.

Use of Biologics, Biosimilars Showed Rapid Uptake in 2018

May 16, 2019

Almost 5.8 billion prescriptions were dispensed in the United States in 2018, an increase of 2.7% over the previous year, according to the IQVIA Institute for Human Data Science’s report Medicine Use and Spending in the U.S.: A Review of 2018 and Outlook to 2023.

By Angela Maas

Almost 5.8 billion prescriptions were dispensed in the United States in 2018, an increase of 2.7% over the previous year, according to the IQVIA Institute for Human Data Science’s report Medicine Use and Spending in the U.S.: A Review of 2018 and Outlook to 2023.

Retail and mail pharmacies dispensed 127 million specialty prescriptions last year, an increase of 15 million since 2014. In 2018, for the second year in a row, specialty prescription volume grew more than 5% although the medicines accounted for only 2.2% of prescriptions overall. With an increase in the availability of oral and self-injected specialty therapies, these drugs “are increasingly dispensed through retail pharmacies,” said Murray Aitken, executive director of the institute, during a May 6 press call.

Researchers found that there has been rapid uptake of the programmed cell death-1 (PD-1) and programmed cell death ligand-1 (PD-L1) inhibitors. In 2014, following the FDA approval of Merck & Co., Inc.’s Keytruda (pembrolizumab) in September, there were 2,403 people treated with an immuno- oncology checkpoint inhibitor. That number rose to 212,473 in 2018, with six products on the market boasting numerous indications.

The use of biosimilars — which the institute defines on a broader basis than only those therapies approved through the 351(k) pathway — “in terms of volume is still modest,” said Aitken, with these therapies representing less than 2% of the total biologics market in 2018. But in those areas where a biosimilar is available, “there is reasonably rapid uptake.”

CBO Says Proposed Drug Rebates Rule Will Increase Government Spending by $177B

May 13, 2019

When HHS unveiled a proposed rule in late January aimed at eliminating drug rebates in Medicare Part D and Medicaid managed care, the proposal was met with mixed responses. A recently released score from the Congressional Budget Office (CBO) calls into question whether the administration chooses to move forward with the proposal in its current form.

The proposal would do away with the safe harbor protection in the anti-kickback statute for rebates negotiated between manufacturers and PBMs starting Jan. 1, 2020.

By Angela Maas

When HHS unveiled a proposed rule in late January aimed at eliminating drug rebates in Medicare Part D and Medicaid managed care, the proposal was met with mixed responses. A recently released score from the Congressional Budget Office (CBO) calls into question whether the administration chooses to move forward with the proposal in its current form.

The proposal would do away with the safe harbor protection in the anti-kickback statute for rebates negotiated between manufacturers and PBMs starting Jan. 1, 2020.

In the CBO’s report, the agency projects that if the rule is implemented as proposed, it will increase federal spending by approximately $177 billion from 2020 to 2029. Of that total, spending on Medicare Part D premiums would increase by about $170 billion. Without rebates to keep premiums low, beneficiaries would face higher premiums. The agency anticipates that “rather than lowering list prices, manufacturers would offer the negotiated discounts in the form of chargebacks,” which are shared with beneficiaries via a manufacturer payment to a pharmacy.

The report, however, also concludes that “no current system could both meet the proposed rule’s standards and facilitate chargebacks.”

If “rebates could no longer be paid to PBMs in Medicare Part D,” but “systems are not available to support retail pharmacy chargebacks,…this would be an untenable situation,” says Elan Rubinstein, Pharm.D., principal at EB Rubinstein Associates, making it “reasonable to delay” the proposed implementation date.

“The increase in premiums was expected by many, but the growth in federal spending was somewhat surprising given that lower upfront prices would generally benefit the end payer, which in this case is the federal government,” says Jeremy Schafer, Pharm.D., senior vie president, director, access experience team at Precision for Value. “It seems changing the safe harbor may not accomplish patient savings or reduced government spending as hoped for by the administration.”