​Treating cancer like a rare disease
Brad Smith, PhD
Jun 01, 2017

With the advent of targeted kinase inhibitors and the use of genomics to target specific, and often rare, patient populations, oncology is rapidly transitioning from a number of broad indications, to a therapeutic area composed of dozens of rare diseases. Each new treatment and test targets an ever smaller subset of the cancer patient population, forcing oncology researchers to rethink their approach to recruiting patients into clinical trials.

One place to find answers to these challenges is from rare disease researchers, who are implementing effective methods to succeed in focusing on patients rather than screening. There are a number of similarities between the emerging oncology treatment paradigm and rare disease research. Both areas must leverage a deep knowledge of disease molecular biology matched with drug mechanisms of action. Both hope to provide long term improvements in patient outcomes by correcting the defects underlying disease. Rare disease researchers are leveraging innovative approaches such as connecting directly with patient communities, supporting registries to better understand patient and disease natural histories and customized recruiting strategies to accommodate the unique needs of these small and dispersed populations. Approaches that will enable oncology clinical researchers include:

  • Build a patient registry. Rare disease researchers often registries to gather data about the patient’s history with the disease, the current standard of care and to identify patients and treating physicians when they begin recruiting. Once the treatment is developed, the data collected in these registries can help demonstrate effectiveness to regulators and payers as part of the approval process, all of which is especially beneficial for precision oncology research. For the patients and investigators, registries may provide access to novel tests and information about their condition.
  • Support Direct-to-Patient activities including online communities. When patients are diagnosed with a disease, they often turn to online support groups for information, advice, and connection with other patients and care givers. These groups can be particularly important for sufferers of rare diseases – including targeted cancers – where patients may feel isolated. However they are often poorly funded and depend on dedicated leaders of patient advocacy groups because the population of patients is so small. Developers of targeted oncology drugs can benefit from sponsoring these sites, providing resources, education, and information about current research in various direct-to-patient approaches. In this way they establish themselves as an ally to the patient community and they have a place to share updates and trial information with the patients and physicians they want to recruit. These efforts may have very tangible benefits for patient retention and compliance and new options for patient reported outcomes.
  • Use real-world data to build sites around patients. When dealing with dispersed patient populations, developers can’t expect to simply open trial sites and hope the patients will come. Instead, they should begin by first identifying patient pools and medical centers where targeted patients are likely to seek treatment and establish trial sites near them. This information can be gathered by evaluating electronic medical records, insurance claims, diagnostic data, and patient registries to make evidence-based decisions about where patients are located. By bringing the trial to patients, the research team makes it physically convenient for patients while enabling them to be treated by their own physicians.
  • Speed trial and site ramp up. When patients are dealing with a rare disease or a cancer diagnosis, they don’t have time to waste waiting for a trial to begin. The timing of a trial launch and site availability may mean that a patient can’t participate. One way we are tackling this challenge, is by establishing a pre-approved network of investigative sites across the U.S. that are pre-screened and ready to participate in any oncology trial for which they have access to patients. In this model, a site is only opened after the first patient is identified, which eliminates the risk of investing in sites that won’t ever find recruits, while opening the trial to patients across the nation. Most often, these networks are disease focused, similar to what rare disease patient advocacy groups have done with investigator consortia.

While there are no silver bullets for recruiting patients to targeted oncology trials, the lessons learned in the rare disease research space can speed the process of finding and recruiting patients and eliminate much of the waste that comes with opening dozens of trial sites that never recruit a single patient. More broadly, these lessons can help move oncology clinical research and practice to be more patient centric, better suited to long-term care of chronic disease.

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