Improve FDA’s Rare Disease Review Process

The Jewish Daily – FORWARD

Opinion

By Jonathan Jacoby

The Jewish community has long been a leader in supporting medical research and education efforts, especially with regard to those diseases that disproportionately afflict people of Ashkenazi Jewish descent. Creating coalitions with other patient advocates in the rare disease community would give American Jews an opportunity to advance efforts to fight diseases that disproportionately affect Jews, as well as to participate in an important public policy debate involving millions in this country.

For example, Jewish agencies should get behind suggestions made by John Crowley, the biotech executive whose efforts to find a treatment for his children’s genetic disease were dramatized in the recent film “Extraordinary Measures.” Testifying on behalf of the Biotechnology Industry Organization last month before the Senate Committee on Health, Education, Labor and Pensions, Crowley pressed for improving the federal regulatory environment for pediatric rare diseases, including establishing a specialized review office at the Food and Drug Administration for genetic and biochemical diseases.

Most genetic diseases are rare, including those that afflict Jews in greater numbers. Patient advocates for rare diseases are confronted with the stark reality that, until recently, few biopharmaceutical companies have been interested in developing therapies for uncommon disorders. Indeed, the definition of a rare or orphan disease is based on the assumption that the prevailing financial model for drug development will only be effective when there is an affected population of 200,000 people or more.

Twenty-seven years ago, Congress approved the Orphan Drug Act to provide incentives to the biopharmaceutical industry to develop more products for rare diseases. Despite this act’s achievements (1,892 orphan designations and 326 treatments approved), 95% of rare disorders remain without a specific treatment approved by the FDA. Treatments for many of these diseases may never be developed, because complexities of the regulatory environment make it hard to attract investment for some rare or difficult diseases, even though the science may be available.

A specialized FDA review office for genetic and biochemical diseases — which has been heavily promoted through a growing campaign of patient advocacy groups called Cure the Process — could focus on specific diseases needing increased expertise and provide assistance on rare disease issues to other FDA review offices. A more specialized drug review by experts at the FDA who understand complex genetic disorders would stimulate and facilitate the development of more therapies to effectively treat rare diseases.

The creation of this new division would send a strong signal to the biopharmaceutical industry and to investors that the FDA is working to improve the regulatory path for thousands of rare disorders. This new division would help drive more investment in early stage biopharmaceutical companies focused on rare diseases.

Regulatory changes can have a huge impact. For instance, an FDA policy change in the early 1990s spurred a remarkable explosion of biomedical innovation leading to the approval of 29 new AIDS drugs. These drugs helped make HIV into the chronic but managed disease that it is for many patients today.

Establishing a new division at the FDA may seem like an esoteric objective to the layperson, but it is, in fact, a critical policy matter affecting the lives of millions of Americans, including a significant number of our fellow Jews.

Jonathan Jacoby is CEO of the R.A.R.E Project, which works with rare disease patient-advocates to promote rare disease advocacy, research and education.

Rare Disease Caucus Announced

John Crowley announced the Rare Disease Caucus this week (link here). Leaders include Congressman Fred Upton and Congressman Joseph Crowley. This is exciting news for the rare disease community.

Hoping great things come from this.

// CC

Announcing Global Genes Project!!! Wear Jeans, help sick kids!

Finally the rare disease community has their ‘pink ribbon’!

With a community of 100 million strong in the US and equal those numbers in the EU this community is deserving of the same energy, efforts, attention and support as some of the big individual diseases like AIDS and Breast Cancer.

We live in a time when we have seen huge success from organizations engaging and building a constituencies for their disease and from outside the community – and for the first time, the rare disease community is coming together to create a broad unifying campaign that will educate, inform and transform this community as we know it.  This will be the year for the rare disease community – our coming out, of sorts.  There is a lot planned that will benefit this community in Q1 – Crowley movie, ‘Extraordinary Measures’, 3-Part PBS Series on technology innovations and the impact on rare disease, and the 2nd Annual World Rare Disease Day -  however, Global Genes Project will become a catalyst for this community and provide a platform for ongoing outreach and engagement, garnering the much needed support that these kids deserve.

Check out www.globalgenesproject.org and join the effort on Facebook as well. There will be numerous opportunities and ideas on how individuals, rare disease organizations and corporations can get involved. It can be as easy as wearing denim genes and incorporating the adorable/identifiable jeans ribbon on your site/blog, etc.

Stay tuned for more updates!!!