Patient and Stakeholder Engagement

Rare Cancer Research from the Patient’s POV

The purpose of clinical research is ultimately to help patients. That may sound obvious, but all too often we forget the human side when it comes to clinical trials. For patients with rare cancers, taking their perspectives into consideration is invaluable to quality research.

But what specific unmet needs do rare oncology patients have? What changes would they like to see in clinical trials? Read on to find out!

Looking for more insights?

Get our white paper on the future of rare oncology research here!

The Unique Struggles of Patients With Rare Cancers

While a cancer diagnosis is never easy, patients and their families face even more challenges than those affected by more common conditions. Researchers need to take the following into consideration while designing trials:

  • Delayed or prolonged diagnosis is common. This is largely because local pathologists may not be familiar with such rare cancers. Additionally, genetic testing is often less well characterized.
  • A high degree of care coordination is necessary. These patients may require specialists and long distance travel. To further complicate things, insurance companies are usually unfamiliar with these conditions and may be unwilling to pay for experimental treatments.
  • There is a general lack of data on disease progression. Patients may be unable to get a prognosis and face a higher degree of uncertainty.
  • Patients have limited treatment options. In many cases, there are no approved treatments. Treatment plans are usually based on extrapolation from studies of similar cancers and mutations.
  • Fewer support channels exist for rare oncology patients and their families. It’s much more difficult to get in touch with others going through the same situation.

What Changes Do Patients Want to See in Rare Oncology Research?

According to a recent paper published by Rare Cancers Europe in 2014, patients and patient advocacy groups are asking for the following changes in clinical research:

  • Increased patient input in the clinical research process
  • Options to accept higher levels of risk than other patient populations
  • More support from the industry and regulatory agencies to recognize and address their needs
  • Innovation in rare cancer clinical research standards

Innovations Rare Oncology Patients Want

Many of the changes rare oncology patients would like to see in clinical research are also good for researchers:

  • Adaptive design strategies
  • Increased use of biomarkers and surrogate endpoints
  • Evidence gathered outside of randomized clinical trials
  • Streamlining of electronic records to facilitate more open data sharing
  • An option for “enduring consent” in which patients can choose to allow researchers to share data more freely

If you’d like to read more about the future of rare oncology, read our white paper.