Provides for a utilization review protocol for the treatment of rare diseases

3 Co-sponsors

• Richard Gottfried
• Linda Rosenthal
• Micah Kellner

30 Multi-sponsors

• James F. Brennan
• Richard Brodsky
• Alec Brook-Krasny
• Karim Camara
• Barbara Clark
• Vivian Cook
• Jeffrey Dinowitz
• Adriano Espaillat
• Ginny Fields
• Deborah Glick
• Timothy P. Gordon
• Aileen Gunther
• Earlene Hooper
• Janele Hyer-Spencer
• Rhoda Jacobs
• Ellen C. Jaffee
• Susan John
• David Koon
• Vito Lopez
• Alan Maisel
• Margaret Markey
• Nettie Mayersohn
• Felix Ortiz
• Crystal Peoples-Stokes
• Audrey Pheffer
• Robert Reilly
• Mike Spano
• Albert A. Stirpe
• Harvey Weisenberg
• Kenneth Zebrowski

BILL NUMBER:A301

Title of bill

An act to amend the public health law and the insurance law, in relation to protocol for treatment of rare disease

Summary of specific provisions

Section 1 of the bill amends PHI. §4900 to define rare disease treatment for disabling and life-threatening conditions and diseases, referencing federal guidelines.

Sections 2 and 4 amend PHL §§ 4905 and 4914 to establish evidentiary standards for utilization review (UR) and external appeal when determin- ing whether to pay for a rare disease treatment, using the standard already in law for treatments under federally approved clinical trials:; "likely to benefit" the ,patient, providing that the treatment is not excluded from coverage for other reasons.

Section 3 amends PHI. §4910 to include the DR denial of payment for a rare disease treatment among the cases that can be taken to external appeal for review.

Section 5-8 amends Insurance law §§ 4900,4905,4910 and 4914 to track the changes made to PHL §§ 4900,4905,4910 and 4914.

Justification

The utilization review and external appeal law works well to expand access to treatments in clinical trials by providing a special standard for coverage determinations. However, the law fails to provide for a small sub-class of individuals who are stricken with disabling and life-threatening rare disease.

The ordinary standard for proving "medical necessity" does not apply for patients in clinical trials because, by definition, the treatment is still on trial. So the utilization review and external appeal process uses the standard of "likely to benefit."

This standard should apply to patients who need rare disease treatment. Because there is little economic incentive for undertaking expensive clinical trails for rare disease treatments, these patients are also inherently unable to meet the ordinary standards for proving medical necessity. A list of rare diseases is maintained by the National Insti- tutes of Health Office of Rare Diseases under the federal Rare Disease Act of 2002.

Prior legislative history

A.8882-A: Third Reading Calendar

Fiscal implications

None

Effective date

Immediately