On this page you can find important Policy Reports from the nation’s leading thought leaders in the Rare Disease Community.

September Sequester Surprise: FDA User Fees on the Chopping Block

BY ELIZABETH M. WROE & PHILIP S. BONFORTE

The threat of a delayed or deterred (re-)authorization of key Food and Drug Administration (FDA) user fee programs (hereinafter referred to as the ‘‘FDA user fees’’) caused significant angst among biomedical product stakeholders over the course of the last year and a half.

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Senator Harkin’s Report on Sequestration

Under Threat Sequestration’s Impact on Nondefense Jobs and Services, July 25, 2012
A report by Sen. Tom Harkin, Chairman, Senate Appropriations Subcommittee on Labor, Health and Human Services, and Education, and Related Agencies

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Quantum of Effectiveness Evidence in FDA’s Approval of Orphan Drugs

Cataloguing FDA’s Flexibility in Regulating Therapies for Persons with Rare Disorders
by Frank J. Sasinowski, M.S., M.P.H., J.D., Chairman of the Board, National Organization for Rare Disorders

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Translational Research Report Crossing Over the Valley of Death

Report by FasterCures, 2011

Stem cells. Genes linked to Alzheimer’s, autism, diabetes.  Cancer drugs tailored to treat an individual tumor.

Every day we see stories in the media about the latest medical “breakthroughs” that could lead to cures for dreaded diseases. And yet, over the years, many breakthroughs like these have yet to bear fruit for patients. Why? Perhaps the media over-hype early discoveries. After all, science is complex and unpredictable. We have to first fail – numerous times – before we succeed, but
we tend not to hear about the failures. No one gets rewarded for failure.

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The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases

Published in – Orphanet Journal of Rare Diseases – July 06, 2011

by B. Miyamoto & Emil Kakkis, MD PhD of the EveryLife Foundation for Rare Diseases

The potential investment impact of improved access to accelerated approval on the development of treatments for low prevalence rare diseases. Patients with rare diseases have had difficulty obtaining disease-specific treatments due to inadequate biotechnology investment despite the existence of promising science. The 1983 Orphan Drug Act was passed to address this problem, offering financial incentives to companies developing rare disease drugs.

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BIO Report: Unleashing the Promise of Biotechnology June 2011

Advancing American Innovation to Cure Disease and Save Lives

Biotechnology companies are working every day to solve the greatest challenges facing our society — whether it’s finding a cure
for cancer, protecting against bio-terror threats, or creating renewable energy sources. Yet despite the urgent need for scientific
breakthroughs in these areas, current government policies are holding back the potential and promise of biotechnology.

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Brownback/Brown Amendment for Rare & Neglected Diseases: FDA Report 2011

Report to Congress:  Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases

In Response to the Brownback/Brown Amendment, Section 740
The Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriation Act, 2010

Department of Health & Human Services: Food and Drug Administration, March 2011

INTRODUCTION

Section 740 of the fiscal year (FY) 2010 Appropriation Act (Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriation Act, 2010, Public Law 111-80), dated October 21, 2009, required the Commissioner, Food and Drug Administration (FDA), to establish two review groups within FDA to make recommendations to the FDA Commissioner on appropriate preclinical, trial design, and regulatory paradigms and optimal solutions to prevent, diagnose, and treat (1) rare diseases and (2) neglected diseases of the developing world. Section 740 requires these groups to submit a report to Congress that describes their findings and recommendations. The language in section 740 of the Appropriation Act follows:

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The US Food and Drug Administration: A Cornerstone of America’s Economic Future

By:  Alliance for a Stronger FDA, March 2011

The U.S. Food and Drug Administration: A Cornerstone of America’s Economic Future was prepared by the Alliance for a Stronger FDA to assist policymakers in understanding the FDA’s role in the nation’s economy. FDA is a pre-eminent public health agency that assures that our food supply is safe and that drugs and medical devices are safe and effective. Its primary mission is to serve and protect the American people. In addition to its public health role, the agency and the industries it regulates have a significant, positive role in our nation’s economy and in stimulating economic growth and job creation.

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COMPETITIVENESS AND REGULATION: THE FDA AND THE FUTURE OF AMERICA’S BIOMEDICAL INDUSTRY

by CHI-California Healthcare Institute, February 2011

This is a report about the relationship between FDA regulation and the competitiveness of the U.S. biopharmaceutical and medical technology industry. Since the invention of biotechnology in California in the 1970s, America has emerged as the global leader in biomedical innovation.

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PhRMA Orphan Drug Report

ORPHAN DRUGS IN DEVELOPMENT FOR RARE DISEASES: More Than 400 Medicines in Development to Treat or Prevent Rare Diseases

AMERICA’S BIO PHARMACEUTICAL RESEARCH COMPANIES (PhRMA), February 2011

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