The Foundation Dedicated to Discovering a Cure for Neuroendocrine Cancer

Caring for Carcinoid Foundation

Make a difference, donate now

Lauren and Nick.

Dave, Suzi, and Lauren at 2011 Focus on Neuroendocrine Tumors Conference in Philadelphia, PA

Dave, Suzi, Lauren - Philadelphia, PA

FDA Review of New Pharmaceuticals for Patients with Rare Diseases

Did you know the Food & Drug Administration (FDA) has responsibility for products that comprise more than 20% of all national consumer spending? And yet the Federal government gives the FDA just $8 per year for each American. The FDA has 10,000 employees to monitor food safety, review the safety and efficacy of medical products (including drugs), assure the safe use of those products, and protect Americans, their pets, and their farm animals from poorly made, counterfeit, and illegal food, drugs, and cosmetics.

More specifically, the FDA is responsible for:
• Protecting the public health by assuring that foods are safe, wholesome, sanitary and properly labeled; human and veterinary drugs, and vaccines and other biological products and medical devices intended for human use are safe and effective
• Protecting the public from electronic product radiation
• Assuring cosmetics and dietary supplements are safe and properly labeled
• Regulating tobacco products
• Advancing the public health by helping to speed product innovations
• Helping the public get the accurate science-based information they need to use medicines, devices, and foods to improve their health

Among its many responsibilities, the FDA approves and regulates new therapies for patients with rare diseases.

In 1962, amendments to the Federal Food, Drug, and Cosmetic Act (FD&C) established that for a new drug to receive FDA approval there must be “substantial evidence of effectiveness” and this evidence must come from “adequate and well-controlled investigations.”

FDA Support for Rare Diseases
The Orphan Drug Act of 1983 was the first legislation to specifically address drug development for patients with rare diseases. The Orphan Drug Act does not modify the standards required by the FDA for approval of products for rare diseases but it does provide financial incentives for drug companies to develop drugs for patients with rare diseases.

A rare disease is a disease that affects 200,000 or fewer people in the United States. There are currently ~7,000 rare diseases affecting ~ 20 million Americans!

Neuroendocrine cancers – including carcinoid cancer, pancreatic neuroendocrine cancer, and related neuroendocrine cancers affect an estimated ~105,000 Americans earning designation as a rare disease. There are many challenges to bringing drugs to market for patients with rare diseases and for carcinoid and pancreatic neuroendocrine tumor patients, in particular.

Advocating on Behalf of Patients
In 2010, I had the honor and privilege to speak on behalf of carcinoid and pancreatic neuroendocrine tumor patients at the FDA public hearing on “Considerations Regarding Food and Drug Administration Review and Regulation of Articles for the Treatment of Rare Diseases.” In my testimony, I encouraged the FDA to create a rare disease review framework to react quickly and effectively to innovative treatments and trials supported by compelling scientific rationale. I also encouraged the FDA to be flexible, consistent, and
predictable in their reviews of products for rare diseases by 1) communicating frameworks that provide consistency and transparency both in and among review divisions, and 2) providing guidance about the situations in which they will consider data from phase II trials for approval of marketing applications.

While the FDA must have “substantial evidence of effectiveness” to approve a marketing application for a rare disease they have historically exercised flexibility in their decisions. By communicating their position of flexibility transparently and consistently this will facilitate more drug development for rare diseases by giving sponsors more clarity.

The National Organization for Rare Disorders (NORD), a 501(c)(3) organization, is dedicated to helping people with rare "orphan" diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service. CFCF is proud to be a member of NORD.

At the same FDA hearing NORD echoed many of my remarks and in particular, NORD urged the FDA to issue a “clear, granular expression of FDA’s historic commitment to exercise flexibility in its review of therapies for rare disorders.”

NORD designed and recently published a study to examine how much flexibility FDA has historically provided in reviewing marketing applications for orphan drugs. Specifically NORD reviewed 135 non-cancer orphan drug new approvals from 1983 – June 30, 2010. NORD found that in 90 out of the 135 approvals FDA exercised flexibility. The NORD study also catalogs the types of situations in which the FDA has elected to exercise flexibility in reviewing and approving new marketing applications for non-cancer orphan drugs.

The full text of this report is available on NORD’s website:

Please review the full report for detailed methodology and also commentary on specific examples of the FDA exercising case-by-case flexibility. The report also includes more information about the regulations and guidances pertaining to the FDA’s flexibility in reviewing new drug marketing applications.

Earlier this year the FDA approved two medications for patients with neuroendocrine tumors and patient input was encouraged prior to these decisions. Once again, I had the honor of testifying before an FDA subcommittee to represent the interests of patients with neuroendocrine tumors. My remarks are available here and here.

The Alliance for a Stronger FDA
A strong FDA is critical to new drug approvals for patients with neuroendocrine tumors.

The Caring for Carcinoid Foundation is proud to support the FDA by joining the Alliance for a Stronger FDA.

According to the Alliance for a Stronger FDA website, the Alliance “works to ensure annual appropriations that will adequately fund the FDA’s essential missions, and we believe that the American people expect this too. There is no backstop, no other agency, that performs this critical work.”

To learn more about the Alliance click here.

Make a Difference Get Involved Today

Sign-up for our e-Newsletter.

E-update Archive


Ron's Blog

Read the latest blog from CFCF's Executive Director.


Latest Video

Check out the CFCF video library.

Connect with CFCF online:

BBB Accredited Charity  Charity Navigator Four Star CharitySeal Exchange