OCE Rare Cancers Program

The mission of the Oncology Center of Excellence (OCE) Rare Cancers Program is to promote the development of safe and effective new drugs and biologics to treat patients with rare cancers. To achieve this goal, we leverage multiple OCE projects and initiatives to address the challenges of developing new treatments for cancers that affect a small number of patients. An important aspect of our work is engagement with multiple stakeholders both inside and outside of FDA to identify opportunities to decrease obstacles, better harness scientific knowledge, and strengthen coordination to facilitate efficient drug development.

Given that rare cancers comprise a multitude of cancer types affecting a heterogenous array of patient populations, OCE recognizes that a unidimensional, one-size-fits-all approach is not sufficient to achieve this mission. Instead, OCE uses the following  programs to develop a context-specific approach that is tailored to the unique patient needs and aspects of each rare cancer:

• Oncology Real World Evidence Program

• OCE Scientific Collaborative

• Patient-Focused Drug Development Program

• Pediatric Oncology Program

• Project Catalyst

• Project Community

• Project Confirm

• Project Equity

• Project Facilitate

• Project Orbis

• Project Renewal

• Project Significant

• Project Socrates

Frequently Asked Questions

What is a rare cancer?

Although there is no universally accepted definition for a rare cancer, the Orphan Drug Act defines a rare disease or condition as one which affects less than 200,000 people in the United States (U.S). The National Institute of Health defines rare cancers as those that affect fewer than 40,000 people in the U.S., and many cancer types are even rarer. Additionally, there are also rare subsets of some more common cancers, such as lung cancer or breast cancer. These subsets can be defined based upon molecular subtype (such as ROS-1 positive lung cancer) or in some cases, on unique demographic characteristics of patients affected by the cancer (such as breast cancer in men or pediatric melanoma).

What are some of the challenges to developing drugs to treat rare cancers?

Although the challenges to rare cancer drug development vary and may not be unique to rare cancers, they can include one or more of the following:

• Difficulty enrolling sufficient number of patients to clinical trials
• Decreased financial incentives for drug development
• Insufficient understanding of the cancer pathophysiology, molecular characteristics, and natural history
• Limited or lack of timely access to molecular testing to determine eligibility for treatment with targeted therapies
• Difficulty conducting randomized trials, due to small patient numbers or lack of appropriate therapy to use as a comparator.

How does OCE work to address challenges to developing drugs to treat rare cancers?

The OCE works in conjunction with the Office of Oncologic Diseases to proactively address the challenges that are common to rare cancer drug development as a whole and those that are specific to each rare cancer type. A few examples are provided below.

• Project Community and Project Equity work together to help promote a collaborative, inclusive approach to clinical trial awareness and enrollment for a diverse patient population.
• The Pediatric Oncology Program issues Pediatric Written Requests to incentivize development of drugs for pediatric cancers and works with stakeholders in the pediatric community to facilitate drug development.
• The OCE Scientific Collaborative supports research to enhance knowledge of the pathophysiology of rare cancers and development of preclinical models of rare cancers to support drug development.
• Oncology RWE Program works with OOD review divisions to provide advice regarding use of real-world data and real-world evidence to facilitate drug development; particularly relevant when considering strategies for evidence generation in rare populations.
• Project Significant explores use of Bayesian and other statistical methodologies to demonstrate effectiveness of drugs in trials with small patient numbers.

The OCE also works closely with other FDA offices such as the Office of Orphan Product Development and the Division of Rare Diseases and Medical Genetics, other international regulatory agencies, and external stakeholders to expedite drug development for rare cancers. 

FDA Guidance

• Demonstrating Substantial Evidence of Effectiveness with One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence

• Framework for FDA’s Real-World Evidence Program

• Rare Diseases: Common Issues in Drug Development

• Rare Pediatric Disease Priority Review Vouchers

• Expedited Programs for Serious Conditions - Drugs and Biologics

• FDARA Implementation Guidance for Industry on Pediatric Studies of Molecularly Targeted Oncology Drugs

• Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease

• Developing and Labeling In vitro Companion Diagnostic Devices for a Specific Group of Oncology Therapeutic Products

• Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products

• Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics

• Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry

Helpful Links

• CDER's Accelerating Rare disease Cures (ARC) Program

• Rare Diseases Program

• Developing Products for Rare Diseases and Conditions

• NIH: Genetic and Rare Diseases Information Center

Previous Events

• FDA Rare Disease Day 2023
• Conversations on Cancer: Beginning the Rare Cancer Journey: Charting the Best Path to an Accurate Diagnosis. Watch on YouTube
• Creating a Public-Private Partnership to Support Development of Anti-Cancer Therapies for Ultra-Rare Tumor Indications
• FDA Rare Disease Day 2022
• FDA Rare Disease Day 2021 

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For questions or more information contact: 

OCE-RareCancerProgram@fda.hhs.gov