Don’t Get Lost in the Crowded Orphan Drug Market

Don’t Get Lost in the Crowded Orphan Drug Market

MedImpact Offers Insight on Managing Blockbuster Drugs

MedImpact Expects Orphan Drug Approval to Increase

“We can expect approvals to pick up steam.” — Stephen Jung, PharmD, BCPS, Manager Drug Information, MedImpact

More than 70 orphan drug indications — half of which relate to oncology — received FDA approval in 2017.  

When Scott Gottlieb, MD, was appointed FDA commissioner in 2017, the agency had about 200 orphan drug designation requests pending review. Through the Orphan Drug Modernization Plan announced last June, the FDA committed to completing reviews of all older requests and responding to all new requests within 90 days of receipt.

The July/August 2018 issue of Specialty Pharmacy Continuum features in-depth coverage of MedImpact’s presentations at the 2018 Academy of Managed Care Pharmacy's

(AMCP) Managed Care & Specialty Pharmacy Meeting. The article provides MedImpact’s insight on market trends and payer challenges in response to the FDA’s recent commitment to expediting orphan drug approval.

As the FDA streamlines orphan drug approval, it’s more imperative than ever to plan for the potential financial impact of blockbuster agents to treat rare diseases, according to MedImpact’s specialty team.

The FDA granted approval for several drugs for rare and ultra-rare diseases over the last few years, including nusinersen for spinal muscular atrophy and cerliponase alfa to treat neurodegenerative lysosomal storage disorder.

The price tags for these drugs are high: Nusinersen costs $900,000 for the first year and $450,000 for subsequent years; and cerliponase alfa is $842,400 per year, MedImpact presenters explained at AMCP.

To learn more about the challenges plans face when estimating budget impact and MedImpact’s recommendations, read the full article, "Not Getting Lost in the Crowded Orphan-Drug-Market."

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