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.

Turning Science into Medicine – a Political Perspective

Our Government has an immense capability to help advance treatments and cures for rare diseases through incentives for industry, research grants, and improving regulatory conditions; however, our politicians often lack the public support to challenge the status quo and make good changes to the system.

Our democratic system was created to be responsive to the people; however you could argue that it was also specifically designed with checks and balances to only allow incremental change.  With an urgent need for treatment, families affected by a rare disease only become frustrated with this process.  Ask any patient advocate in D.C. and they will warn you about the difficultly or even impossibility of moving a bill through Congress.  Most advocates will spend most of their careers trying to prevent the loss of services, benefits, or funding and rarely have the opportunity to make real steps forward to improve access to treatments.

In fact, there has been no major legislative initiative to accelerate treatments for rare diseases since the passage of the Orphan Drug Act in 1983 – more than 25 years ago.  Science has made great progress over this time; however policy has not followed pace – until now.

Congress is listening and they want to help.  The Senate’s H.E.L.P. (Heath Education Labor & Pensions) Committee and Appropriation Subcommittee on Agriculture, FDA and Rural Development both held Congressional Hearings in the last month on how to increase the development of new treatments for rare diseases.  A Rare and Neglected Disease Congressional Caucus is also forming to address the needs of patients.  There is even new funding for rare diseases in the NIH and FDA budgets and new requirements for guidances for the FDA to improve the regulatory process.

While it is great to hear about all of the very exciting programs that have been initiated in the last year, there is no time to rest.  Too many children will still wake up tomorrow without treatment.  We must use this momentum to ensure our progress translates into real treatments.

Now is the time when we need your help.  You must become an advocate and take an active role in the political process.  Organizations like The Kakkis EveryLife Foundation, NORD and others are only powerful when their members and supporters take action.  Congress must hear from you – the patients, parents, grandparents, doctors, care takers, academics, and scientists.

I urge you to contact your Members of Congress – write letters, make phone calls, and meet with staffers in the district office to tell them how important advancing new treatments is to you.  Our Foundation has launched its political action center on its CureTheProcess website to allow you to easily contact your Members of Congress, and learn more about current legislation.

You have the power to make a difference.  Click here now and help turn science into medicine.

Contributed by Julia Jenkins, Kakkis EveryLife Foundation

R.A.R.E. Project’s, Jonathan Jacoby to present at FDA Hearing on Rare Disease

On Tuesday, June 29, 2010, and Wednesday, June 30, 2010, FDA’s Office of Orphan Products Development will host a two-day public hearing and Webcast on the Development of Articles for Rare Diseases.

This public hearing is intended to gain from health care providers, academia, industry, patients, and other interested persons their perspectives on various aspects of the development of medical products for the diagnosis, treatment, or management of rare diseases. The input from this public hearing will help inform the work of FDA’s committee for rare diseases

Jonathan Jacoby, CEO R.A.R.E Project will present case studies on therapeutic candidate generation, focusing on;

• Challenges of patient-driven R&D
• Challenges and successes with biomarker identification
• Experience with Orphan Drug Designations
• Individual investigator INDs and IND exemptions
• NIH clinical trials
• Barriers to recruitment and participation in CTs

Following the presentation, Jacoby will provide recommendations that include the importance of very small CTs as a pathway to regulatory approval. Jacoby will present this testimony from 11:40 – 12:00, June 29, 2010.

The meeting will be webcasted and any interested persons are encouraged to join:

• Dates: June 29-30, 2010
• Time: 9:00 AM to 5:00 PM ET
• Webcast addresses (will be active at the time of webcast):
  June 29 – collaboration.fda.gov
  June 30 – collaboration.fda.gov

Orphan Drugs Rare Disease Opportunity, Investors Eye

Investors are warming up to the economics of rare disease orphan drugs as potentially big moneymakers.  As much as we love to bash Wall Street, the financial sector has found gold in orphan drugs for treating rare disease, and that might just fill in a deficit of research capital, which would otherwise be unavailable where and when its needed for orphan disease and disorder treatment drugs.

From The Motley Fool | 5/25/10 :

Orphan drugs don’t have the potential patient numbers that cholesterol or diabetes drugs have, but that doesn’t mean you should ignore them either.  What they lack in volume they make up for in price.

Technically speaking, a drug can get orphan drug status if it treats a disease that affects less than 200,000 people in the U.S. A lot of cancers fall into that range — for instance, GlaxoSmithKline’s (NYSE: GSK) Tykerb has orphan drug status for gastric cancer. But the true orphan drugs treat diseases you’ve probably never have heard of.

A little protection goes a long way

There really are some financial incentives to gaining rare disease orphan drug treatment status. The government kicks back up to 50% of the cost of clinical trials in the form of tax credits. And the Food and Drug Administration comps the fee established by the Prescription Drug User Fee Act (PDUFA) that drugmakers pay when submitting their orphan disease marketing application.

But the biggest advantage comes from the exclusive sales of the rare disease orphan drug treatment for seven years in the U.S. and six to 10 years in the EU that governments give orphan drugs. It doesn’t matter whether patents expire during that time, the regulatory agencies will hold off approving generic competition until the rare disease orphan drug treatment exclusivity has expired.

The Motley Fool:

Being the first to treat a disease also offers an indirect lockup of patients from other branded drugs. If the drug works well enough, patients are less likely to enter a clinical trial for a competitor’s experimental drug, making it harder for the competitor to get on the market. Being first doesn’t provide an absolute monopoly — in addition to Genzyme’s (Nasdaq: GENZ) Cerezyme, Shire’s Vpriv is approved for Gaucher disease and Protalix and Pfizer (NYSE: PFE) are also working on a Gaucher disease treatment — but a little protection can go a long way.

On the road to bigger things

Sometimes the orphan disease drug status is just a starting point. Because the proteins that drugs interact with are often involved with other diseases, treating an orphan disease can be a good starting point before launching into another disease that may not be so rare.

READ THE COMPLETE ARTICLE AT MOTLEY FOOL

Medical Foods Equity Act and PKU

Drug Development Under the Orphan Drug Act – It needs a fix!

There is a tremendous amount of frustration with the current state of rare/orphan drug development. With over 7,000 rare diseases only 344 therapies & treatments have been developed. So what have been the road blocks and what is the community currently doing to accelerate drug development.

One parent advocates’ perspective on this ongoing debate:

http://addiandcassi.com/wordpress/numbers-dont-add-up-only-344-new-drugs-approved-by-fda-for-30-million-americans-with-rare-disease-in-past-25-years/

‘Denim Jean’ Ribbon Unveiled To Promote Rare Disease Awareness

Campaign Brings Together Support from over 90 Corporations, Non-Profit Foundations, Research Institutions, Hospitals and Advocacy Groups; New Blue Jeans Denim Ribbon Unveiled To Promote Rare Disease Awareness

DANA POINT, CA – February 1, 2010 – Hope – It’s In Our Genes.™ The Children’s Rare Disease Network today announced the Global Genes Project, an initiative designed to raise awareness about the prevalence of rare diseases that afflict millions of children worldwide. Rare disease advocates organizing the campaign selected a blue jeans denim ribbon as the international symbol of hope for children suffering from life threatening and chronically debilitating rare diseases.

More than 90 corporations, non-profit foundations, research institutions and children’s hospitals have signed on to support the Global Genes Project including: 23andme, Amicus Therapeutics, Better Health, BioMarin, CheckOrphan, CollabRx, Counsyl, Coriell Institute, DonateGames.org, FasterCures, FreeForm, Genetic Alliance, Geni.com, Genzyme, Hide and Seek Foundation, Jackson Laboratory, Kakkis EveryLife Foundation, Knome, Lili Claire Foundation, Life Technologies, Mayo Clinic and Shire.

Numerous rare disease foundations from countries including Australia, Argentina, Belgium, Canada, China, New Zealand, Philippines, the United Kingdom and United States are helping support the global movement by organizing activities throughout February and leading up to World Rare Disease Day 2010.

“The Global Genes Project is providing a new platform to help unite people under a common goal and to make a difference for all children suffering from rare disease,” said Nicole Boice, Founder, Global Genes Project. “We want to create a world where children suffering from chronic disease have equal access to life saving drug treatments.”

Growing Public Health Concern; Inequity Exists

Rare disease is an important and growing global public health concern that can no longer be ignored. While each disease may be rare when considered by itself, rare disease collectively impacts the lives of millions. According to estimates from the National Institutes of Health (NIH), approximately 6,800 rare diseases exist, many of which are genetic in origin, translating to an affected population of 25-30 million Americans or approximately 1 in 10 people. In Europe, the number affected is almost identical. Approximately 75% of those affected by rare diseases are children, making the rare disease category one of the most deadly and debilitating for children worldwide.

Despite the success of the Orphan Drug Act of 1983, pharmacologic treatments exist for only a small fraction of rare diseases, or approximately 200 of the 6,800 rare diseases. After 25 years of orphan drug regulations and legislation, the development path to approve new drugs has not stimulated orphan drug development for small populations of patients facing rare disease.
Current studies also suggest that it takes more than a dozen years and hundreds of millions of dollars to take a potential drug from discovery to the marketplace. As a result, severe inequity exists in access to care and treatment for millions of rare disease patients.

Denim Blue Jeans Ribbon Campaign

Leading up to the annual World Rare Disease Day on February 28, 2010, the Global Genes Project will help facilitate a series of denim focused awareness events, charitable activities and engage corporate sponsors, government officials, celebrities and the media to help spread its lifesaving message of hope.

“Blue is the color of strength and our blue jeans denim ribbon is an ideal symbol that expresses solidarity,” added Boice. “Genes and jeans are a natural fit – both are universal, come in pairs and are unique to the individual. It’s a simple concept that anyone can embrace and a powerful way to raise awareness for rare disease.”

The Global Genes Project is asking rare disease advocates and supporters across the world to wear denim jeans and blue jeans denim ribbons the week of February 22 and leading up to World Rare Disease Day 2010. The organization is also asking supporters to display the blue jeans denim ribbon symbol on their websites and to actively invite co-workers, friends and family and their community to join in spreading this global message.

Blue jeans denim ribbons can be obtained by visiting www.globalgenesproject.org. Denim jeans and ribbon wearing supporters can submit digital photos on Facebook. To join the growing list of supporters, visit http://www.globalgenesproject.org/sponsors.php.

About The Global Genes Project

The Global Genes Project is a campaign of the Children’s Rare Disease Network and a grassroots effort with the goal to increase awareness about the prevalence of rare diseases worldwide. The Children’s Rare Disease Network is a registered 501c3 non profit organization. For more information, visit www.crdnetwork.org or www.globalgenesproject.org.

CNN Story Highlights Global Problem Facing Millions Suffering From Rare Diseases

CNN is running a comprehensive package about rare diseases and the crisis patients with rare diseases are facing with by being priced out (or left out) of the drug development market.   The CNN story discusses the fact that 6800-7000 rare diseases are still mostly overlooked by pharmaceutical companies because of the small patient population and lack of financial incentives to pharma and biotech companies.   With this package, CNN also provides a rare disease resource guide so that people can get informed.

Between the US and EU, there are 60 million (!) people suffering from rare diseases (not exactly a small patient population) of different types.   According to the National Institutes of Health, pharmaceutical companies only have treatments for 200 of these rare diseases!

Eurordis and rare disease advocates in Europe are calling for an EU resolution to be passed in June to create a national plan on rare diseases.   We need millions of rare disease advocates working together and marching on Washington to help highlight the problem here in the United States.

The entire drug development system in the United States is broken for millions of people and there are no solutions in sight.   After weeks of waiting, we don’t even have a Director of NIH named.