Market Access

Trends That Matter for FDA Orphan Label Application Reviews

January 17, 2019

The Orphan Drug Act (ODA) offers multiple incentives to manufacturers that bring a drug to market with an orphan designation. A recently released U.S. Government Accountability Office (GAO) report revealed that while the number of applications both received and granted for this designation has grown, FDA reviewers were not consistently recording or evaluating required information that is mandated to consider granting this designation, AIS Health reported.

The Orphan Drug Act (ODA) offers multiple incentives to manufacturers that bring a drug to market with an orphan designation. A recently released U.S. Government Accountability Office (GAO) report revealed that while the number of applications both received and granted for this designation has grown, FDA reviewers were not consistently recording or evaluating required information that is mandated to consider granting this designation, AIS Health reported.

The report, titled Orphan Drugs: FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue (GAO-19-83), shows that from 2008 to 2017, both orphan designation applications received as well as orphan designations granted rose.

Researchers also assessed whether FDA reviewers were using consistent criteria to evaluate applications. On this, however, reviewers’ performance left a bit to be desired.

From October to December 2017, after the implementation of the modernization plan, the agency analyzed 148 review templates. According to the GAO report, “of the five review template sections where reviewers are required to record information, we found that OOPD [i.e., the Office of Orphan Products Development] does not ensure that all required information is consistently recorded in the background information section and evaluated when making designation decisions.”

Of the 148 templates, the FDA granted orphan designation to 26 applications that were missing required information. GAO recommends that the “FDA should ensure that all required information for reviews of orphan designation applications is consistently recorded and evaluated. The agency concurred with our recommendation.”

MMIT Reality Check on Multiple Sclerosis (Jan 2019)

January 11, 2019

According to our recent payer coverage analysis for multiple sclerosis treatments, combined with news from key healthcare influencers, market access is shifting in this drug landscape.

According to our recent payer coverage analysis for multiple sclerosis treatments, combined with news from key healthcare influencers, market access is shifting in this drug landscape.

To help make sense of this new research, MMIT’s team of experts analyzes the data and summarizes the key findings for you. The following are brief highlights. To read the full piece, including payer coverage, drug competition and prescriber trends, click here.

Payer Coverage: A review of market access for multiple sclerosis treatments shows that more than half of the lives under the pharmacy benefit in commercial and health exchange formularies have utilization management restrictions.

Trends: Contracting is prevalent among interferons, where formulary preference drives choice. Via AIS Health.

MMIT Reality Check on Immune Globulin PID (Jan 2019)

January 4, 2019

According to our recent payer coverage analysis for immune globulins for primary immunodeficiency (PID), combined with news from key healthcare influencers, market access is shifting in this drug landscape.

According to our recent payer coverage analysis for immune globulins for primary immunodeficiency (PID), combined with news from key healthcare influencers, market access is shifting in this drug landscape.

To help make sense of this new research, MMIT’s team of experts analyzes the data and summarizes the key findings for you. The following are brief highlights. To read the full piece, including payer coverage, drug competition and prescriber trends, click here.

Payer Coverage: A market access breakdown reveals that more than 38% of the covered lives under the pharmacy benefit in commercial and health exchange formularies have utilization management restrictions

Trends: The FDA gave an additional approval to Hizentra (immune globulin subcutaneous [human]) as a maintenance therapy in adults with chronic inflammatory demyelinating polyneuropathy to prevent relapse of neuromuscular disability and impairment

Trends That Matter for Migraine Drugs

January 3, 2019

Therapy for chronic migraine — a condition that’s been notoriously difficult to treat and which often leads to significant direct and indirect health care costs — has been upended with the recent approval of three injectable monoclonal antibody products in a new preventive medication class that’s significantly more effective than older preventive migraine drugs, a researcher says.

Therapy for chronic migraine — a condition that’s been notoriously difficult to treat and which often leads to significant direct and indirect health care costs — has been upended with the recent approval of three injectable monoclonal antibody products in a new preventive medication class that’s significantly more effective than older preventive migraine drugs, a researcher says.

These new calcitonin gene-related peptide (CGRP) inhibitors — Amgen, Inc. and Novartis AG’s Aimovig (erenumab), Teva Pharmaceuticals’ Ajovy (fremanezumab) and Eli Lilly and Co.’s Emgality (galcanezumab) — also may usher in an era of value-based contracting for migraine products, with plan sponsors willing to pay more to get better results, Machaon Bonafede, Ph.D., outcomes research practice leader at IBM Watson Health, told attendees Oct. 23 at the Academy of Managed Care Pharmacy Nexus annual meeting, AIS Health reported.

Express Scripts Holding Co. already has inked a value-based deal for two of the three drugs in the new migraine class. The PBM’s new SafeGuardRx Migraine Care Value program, which starts April 1, will cover Aimovig and Emgality. It will include a comprehensive clinical care program with access to CGRP inhibitors. In addition, Express Scripts is offering what’s in effect a money-back guarantee for plan sponsors when a patient discontinues therapy in the first 90 days.

These new medications have the potential to remake migraine treatment, the direct and indirect costs of which have been estimated at $36 billion annually in the U.S., Bonafede said. Indirect costs — such as lost productivity — can be difficult to capture and quantify, he added.

MMIT Reality Check on Epilepsy (Dec 2018)

December 28, 2018

According to our recent payer coverage analysis for epilepsy treatments, combined with news from key healthcare influencers, market access is shifting in this drug landscape.

According to our recent payer coverage analysis for epilepsy treatments, combined with news from key healthcare influencers, market access is shifting in this drug landscape.

To help make sense of this new research, MMIT’s team of experts analyzes the data and summarizes the key findings for you. The following are brief highlights. To read the full piece, including payer coverage, drug competition and prescriber trends, click here.

Payer Coverage: A review of market access for epilepsy treatments shows that almost 29% of the covered lives under the pharmacy benefit in commercial formularies have utilization management restrictions on epilepsy medications.

Trends: Epidiolex, the first FDA-approved treatment that contains a purified drug substance derived from marijuana, is now available in the U.S. Via AIS Health.

Radar On Market Access: GAO Recommends FDA Improve Orphan Label Application Reviews

December 27, 2018

The Orphan Drug Act (ODA) offers multiple incentives to manufacturers that bring a drug to market with an orphan designation. A recently released U.S. Government Accountability Office (GAO) report revealed that while the number of applications both received and granted for this designation has grown, FDA reviewers were not consistently recording or evaluating required information that is mandated to consider granting this designation, AIS Health reported.

The Orphan Drug Act (ODA) offers multiple incentives to manufacturers that bring a drug to market with an orphan designation. A recently released U.S. Government Accountability Office (GAO) report revealed that while the number of applications both received and granted for this designation has grown, FDA reviewers were not consistently recording or evaluating required information that is mandated to consider granting this designation, AIS Health reported.

The report, titled Orphan Drugs: FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue (GAO-19-83), shows that from 2008 to 2017, both orphan designation applications received as well as orphan designations granted rose.

The report, titled Orphan Drugs: FDA Could Improve Designation Review Consistency; Rare Disease Drug Development Challenges Continue (GAO-19-83), shows that from 2008 to 2017, both orphan designation applications received as well as orphan designations granted rose.

Of the 3,690 orphan drug designation applications that the FDA received from 2008 to 2017, about 71% were granted that designation by April 2018; 21% were deemed pending, 5% were denied, and 2% were withdrawn.

In June 2017, the FDA issued the Orphan Drug Modernization Plan, intended to eliminate within 90 days the backlog of 138 applications for the designation that had been pending for more than 120 days. In addition, it sought to ensure that future applications would be reviewed within 90 days of the agency’s receiving them. The FDA achieved the first goal, and, as of July 20, 2018, had “overall met its 90-day timeliness goal for reviewing designation applications since mid-September 2017 and has completed most application reviews within 60 days of receipt,” according to the GAO report.

Researchers also assessed whether FDA reviewers were using consistent criteria to evaluate applications. On this, however, reviewers’ performance left a bit to be desired.

From October to December 2017, after the implementation of the modernization plan, the agency analyzed 148 review templates. According to the GAO report, “of the five review template sections where reviewers are required to record information, we found that OOPD [i.e., the Office of Orphan Products Development] does not ensure that all required information is consistently recorded in the background information section and evaluated when making designation decisions.”

Of the 148 templates, the FDA granted orphan designation to 26 applications that were missing required information. GAO recommends that the “FDA should ensure that all required information for reviews of orphan designation applications is consistently recorded and evaluated. The agency concurred with our recommendation.”