Rare Disease

Latest News

RAre disease ribbon-Chinnapong-stock.adobe.com
5 Rare Diseases That Now Have Their First FDA-Approved Treatments

February 28th 2025

Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, signifying progress in closing treatment gaps for rare disease.

FDA approved. | Image Credit: Pawel - stock.adobe.com
FDA Approves Mirdametinib for Neurofibromatosis Type 1 With Plexiform Neurofibromas

February 12th 2025

Gene therapy, gene editing concept | image credit: vchalup - stock.adobe.com
Delandistrogene Moxeparvovec Fails to Meet Primary End Point in Phase 3 Study

November 15th 2024

Patients with SMA and their caregivers stand to benefit from shared decision-making processes | image credit: Intelligent Horizons - stock.adobe.com
Shared Decision-Making Is Essential Prior to SMA-Enhancing Treatments

November 8th 2024

SMA type is a strong predictor of some oral functioning in affected patients | image credit: RTimages - stock.adobe.com
Oral Function Impairment Varies Across Adult Patients With SMA

October 28th 2024

More News

Atopic Dermatitis: Determining Utilization Management Criteria for Long-Term Systemic Therapies
October 31st 2016

Atopic Dermatitis: Determining Utilization Management Criteria for Long-Term Systemic Therapies

Identifying the Appropriate Patients for Atopic Dermatitis Treatments
October 31st 2016

Identifying the Appropriate Patients for Atopic Dermatitis Treatments

Atopic Dermatitis: Reimbursement Challenges
October 31st 2016

Atopic Dermatitis: Reimbursement Challenges

Atopic Dermatitis: Cost and Value
October 31st 2016

Atopic Dermatitis: Cost and Value

Measuring Outcomes in Atopic Dermatitis
October 31st 2016

Measuring Outcomes in Atopic Dermatitis

Understanding Emerging Therapies in Atopic Dermatitis
October 31st 2016

Understanding Emerging Therapies in Atopic Dermatitis

Weighing Quality of Life Improvements in Atopic Dermatitis
October 31st 2016

Weighing Quality of Life Improvements in Atopic Dermatitis

Atopic Dermatitis: Current and Future Management
October 31st 2016

Atopic Dermatitis: Current and Future Management

Persistent and Recurrent Atopic Dermatitis
October 31st 2016

Persistent and Recurrent Atopic Dermatitis

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October 26th 2016

Reconsidering the Economic Value of Multiple Sclerosis Therapies

Tiffany Shih, PhD Craig Wakeford, MA Dennis Meletiche, PharmD Jesse Sussell, PhD Adrienne Chung, PhD Yanmei Liu, MS Jin Joo Shim, MS Darius Lakdawalla, PhD
Availability of multiple sclerosis (MS) therapies provides substantial value to the currently healthy (who may contract MS in the future), particularly when treatment is fully covered by insurance.
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October 12th 2016

Radiomics to Predict Response to Antiangiogenic Therapy in Glioblastoma

Surabhi Dangi-Garimella, PhD
Radiomic combines imaging and computational technologies and can identify patients with recurrent glioblastoma who may benefit from using angiogenesis inhibitors.
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October 11th 2016

OOP Expenses Remain a Problem in Medicare, Especially for Beneficiaries Taking Specialty Drugs

Priyam Vora
Medicare beneficiaries still face huge out-of-pocket expenses because of uncapped cost sharing in the catastrophic coverage phase. This is especially true for beneficiaries who take specialty drugs.
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October 10th 2016

What We're Reading: Anthem Will Not Cover First Drug Approved for Duchenne

AJMC Staff
What we're reading, October 10, 2016: Anthem will not cover Exondys 51, the first drug approved for treatment of Duchenne muscular dystrophy; big soda has sponsored 96 health and medical groups; and new Illinois program seeks to fill care gaps for working poor.
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October 9th 2016

Jakafi Named Recommended Treatment for Myelofibrosis in NCCN Guideline

Laura Joszt, MA
As part of the National Comprehensive Cancer Network's new clinical guidelines for myeloproliferative neoplasms, a group of rare blood cancers, Jakafi has been recommended for the treatment of myelofibrosis.
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October 4th 2016

Specialty Pharmaceutical Pipeline: Increased Competition, Biosimilar Uncertainty

Laura Joszt, MA
Perennial favorite, Aimee Tharaldson, PharmD, senior clinical consultant of emerging therapeutics at Express Scripts, opened the Academy of Managed Care Pharmacy 2016 Nexus, October 3, 2016, in National Harbor, Maryland, with a discussion of specialty pharmaceutical drugs in the pipeline.
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October 1st 2016

Sanofi and Regeneron Find Dupixent Effective in the Treatment of Moderate-to-Severe Atopic Dermatitis

Positive data from two Phase 3 clinical trials that show Dupixent (dupilumab) is effective in the treatment of moderate-to-severe atopic dermatitis was announced at the 25th European Academy of Dermatology and Venereology Congress in Vienna, Austria.
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September 19th 2016

What We're Reading: Government Wants Clinical Trial Findings More Widely Available

AJMC Staff
What we're reading, September 19, 2016: the government update rules on reporting clinical trial findings; hospitals say they aren't receiving discounts for Valeant's heart drugs; and the FDA approved the first treatment for Duchenne muscular dystrophy.
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September 16th 2016

What We're Reading: Trump Would Expand Insurance Coverage Through Medicaid

AJMC Staff
What we're reading, September 16, 2016: Donald Trump mentions using Medicaid to expand coverage and making birth control available without a prescription; medical marijuana could curb the opioid epidemic; and how rare diseases are becoming more common.
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August 18th 2016

Non-Orphan Use of Orphan Drugs Drives Up Cost

Laura Joszt, MA
The use of orphan drugs for non-orphan diseases is driving up the cost of these already expensive medications. A new report from America’s Health Insurance Plans found that almost half of the utilization for 46 orphan drugs were used for non-orphan indications.
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August 9th 2016

Insurance Status Affects Outcomes for Patients With Cancer

Laura Joszt, MA
Health insurance status can have a big impact on a person’s health outcomes after they are diagnosed with cancer, according to 2 new studies published in CANCER.
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July 20th 2016

Judge Denies Order to Keep Generic Crestor Competitors Off the Market

Mary Caffrey
AstraZeneca is trying to invoke the Orphan Drug Act to protect its blockbuster, on the grounds it is approved to treat a rare pediatric condition.
The Future of PAH
July 18th 2016

The Future of PAH

Managed Care's Role in Future Treatment of PAH
July 18th 2016

Managed Care's Role in Future Treatment of PAH

PAH Treatment Driven by Data From Peer-Reviewed Literature
July 18th 2016

PAH Treatment Driven by Data From Peer-Reviewed Literature

Hospitalization in PAH
July 18th 2016

Hospitalization in PAH

Optimizing PAH Treatment in Managed Care
July 18th 2016

Optimizing PAH Treatment in Managed Care

PAH: Multidisciplinary Care and Medication Benefits
July 18th 2016

PAH: Multidisciplinary Care and Medication Benefits

Long-Term Outcomes and Managing PAH Costs
July 18th 2016

Long-Term Outcomes and Managing PAH Costs

The Importance of a Patient-Centered Approach in PAH
July 18th 2016

The Importance of a Patient-Centered Approach in PAH

Understanding the Newer Options in PAH
July 18th 2016

Understanding the Newer Options in PAH

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