Rapid Change, Real Promise: The Future of Rare Oncology Research

Our growing understanding of cancer’s genetic and molecular origins is fueling rapid gains in rare oncology research. Rare cancer therapies accounted for more than 40 percent of FDA orphan drug designations in 2014, and increasing international cooperation among big pharma, biotech, and academia is making this research more efficient than ever.

We examine trends and issues in rare oncology research in Rapid Change, Real Promise: The Future of Rare Oncology Research, a new white paper available now for download.  The paper provides insight on major issues in rare oncology today, including:

  • Patient perspectives on cancer research.
  • Innovative trial designs.
  • The evolving regulatory landscape.
  • Legislation that may affect how studies are conducted.

Rare, but many

Each rare cancer afflicts a very small percentage of the population, but there are a lot of them: 186, to be exact. As a result, 27 percent of new cancer diagnoses in the U.S., and 22 percent in the European Union, are in rare indications. The mortality rate is high, with these instances accounting for about a quarter of cancer deaths.

With many lives at stake, it’s important that researchers consider these disorders not just from a clinical view, but also from the patients’ perspective. Patients and their families face big challenges that include:

  • Care coordination. Finding a doctor with the right experience could require long-distance travel and coordination with a local care team. And insurance companies may balk at funding experimental treatments.
  • Sparse data. Scarce or nonexistent data on a disease’s natural history makes it difficult to characterize progression and assess prognosis.
  • Limited treatment options. Patients often receive a “best-guess” treatment extrapolated from a cancer with a similar histology, or with the same mutation.
  • Going it alone. Shared experience can mitigate the stress of living with a rare cancer, but small study populations often leave patients without support.

Speaking with a louder voice

Patient advocacy groups are demanding more input into the clinical research process and greater innovation through approaches such as adaptive trial design, use of biomarkers and surrogate endpoints, and more data-sharing. Some advocates are pursuing options to accept risk levels that are higher than for other patient populations.

Advocacy groups can be an invaluable resource for sponsors, identifying potential investigators and spreading the word about studies. And because they know what patients are up against, they can help sponsors develop protocols that improve the participant experience and improve recruiting and retention.

Sponsors also must pay close attention to site selection. Sure, sites must have the experience and willingness to conduct research according to good clinical practice — but more than that, they must openly share information with physicians and deftly manage these vulnerable patients. Keeping sites motivated is important, too, and demands constant effort.

Changing regulatory climate

The FDA and its counterpart, the European Medicines Agency, have enacted programs to expedite review and approval of investigative drugs that target unmet needs. The FDA offers Fast Track and Breakthrough Therapy designations, along with accelerated approval and priority review processes. Likewise, the EMA’s Accelerated Assessment can cut review times by nearly 30 percent, and Conditional Marketing Approval speeds availability of treatments for seriously debilitating and life-threatening diseases.

Legislation pending in Congress could significantly affect the future of rare cancer treatments. The 21st Century Cures Act seeks to promote development and hasten approval of new drugs and devices, increasing funding to the National Institutes of Health, creating an innovation fund to stimulate new discoveries, and promoting non-traditional trial designs to speed approvals.

Take a closer look at these topics and more by downloading our white paper.

Author Details

Juliet Moritz
Juliet Moritz is Executive Director, Rare Disease & General Medicine, Strategic Development. She has worked in clinical research for more than 25 years, and her extensive background covers the spectrum from single-site studies to large, multinational trials. She joined Premier Research in 2016 to specialize in rare diseases, supporting the strategic development of products that address unmet medical needs associated with rare and orphan afflictions.
Sachin Kulkarni
Sachin Kulkarni has 14 years of experience in clinical research and project management. As an Executive Director, Strategic Development, Mr. Kulkarni is responsible for providing the strategic planning, coordination, knowledge and expertise in a specific subject matter for a project or standalone consultative services.
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