Research Strategic Plan

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We are driven by a singular commitment to improve care and support the development of new therapies to mitigate the long-term effects brought on by hypothalamic-pituitary tumors and treatments...

Assessing the Research Gaps in Craniopharyngioma

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Author: Nathalie Kayadjanian, Ph.D., Scientific Advisor

Because the development of new therapies is long, costly, and risky, RAWF has undertaken an assessment of the research gaps in knowledge, research needs, tools, and infrastructure to maximize efficiency, reduce the time of development, and increase the chances of success of finding meaningful treatments for individuals with CP. As a result, RAWF is developing a patient-centered research strategic plan that translates RAWF’s vision into an action plan, helps define priorities, provides an evidence-based framework for decision-making, and structures the long-term research activities of the organization.

A gap analysis was conducted at each stage of the drug development process for CP including the biology underlying the tumor and comorbidities, preclinical models, clinical trials, and clinical research. In addition, existing research infrastructure and tools and networks of experts and/or organizations were assessed, and interviews were conducted with researchers, clinicians, patients, and caregivers engaged in CP research and care. As a result, a RAWF research report was developed summarizing the state-of-the-art in CP research and defining research priorities.

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3 Research Priorities

To drive the successful implementation of the RAWF’s strategic plan for research, three research priorities were identified:

Develop a patient registry to facilitate research, inform medical decision-making, identify patterns, and improve patient care and outcomes through a deeper understanding of diseases and their treatments

Gather evidence-based data on the viewpoints of patients and caregivers on symptoms of importance, treatment priorities, and benefit/risk evaluation.

Develop an international network of craniopharyngioma experts including clinicians, researchers, surgeons, and patients and caregivers.

Other research tasks of importance should be performed including:

  • Monitor therapeutics of interest for craniopharyngioma (CP) and hypothalamic-pituitary tumors and establish connections with sponsors for symptoms defined as priorities by patients and caregivers (e.g., hypothalamic obesity, sleep disruption, psychosocial implications).
  • Guide the development of a list of clinical research priorities to improve survivors’ current care.
  • Develop a database of clinical care centers in the United States to facilitate clinical trial enrollment.
  • Identify and engage researchers in new areas of CP research (e.g., sleep disruption, psychosocial implications).
  • Identify and develop relationships with new and key research groups in basic research, diagnosis biomarkers, CP animal and cell models, and clinical research.
  • Establish and cultivate strategic partnerships, as appropriate, with FDA, NIH, industry partners, and other nonprofit organizations to foster potential collaborations in pursuit of RAWF’s research goals.

Priorities Breakdown


  • Identify and recruit patients for clinical trials
  • Identify and recruit patients and caregivers for clinical research studies
  • Understand and gather data on views of caregivers and survivors
  • Test new outcome measures to assess drug efficacy in clinical trials
  • Examine longitudinal changes in clinical characteristics and treatment outcomes (natural history of CP)
  • Assess effectiveness, safety, and harm of treatments in real-world settings
  • Assess disparities in clinical care


  • Accelerates clinical research, clinical trials, and improves care
  • Represents a valuable tool for clinicians, survivors/caregivers, industry
  • Helps identify underserved population (adults, African Americans)
  • Facilitates data-sharing

Action plan:

  • Build an online registry that is modular, adaptable, possibly interoperable
  • Investigate implementation of Globally Unique Identifiers (GUID) to avoid data duplication
  • Implement the first module with a minimal dataset (three minimum to complete)
  • Build a registry that is owned by RAWF
  • Collect data initially from survivors and caregivers
  • Aim for an international outreach (collaboration with other organizations?)

Tasks Required:

  • Identify patients through a communication campaign toward healthcare professionals, caregivers/survivors, and other patient organizations
  • Scientific communication to patients and caregivers to increase patient engagement in research: therapeutic development, importance of participation in research
  • Establish a registry advisory board that includes clinicians, caregivers, and survivors
  • Develop a program management plan
  • Plan to apply for a future RO1 grant titled Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01) Clinical Trials Not Required (2024)


  • Gather evidence-based data on treatment priorities, symptoms of importance, benefit and risk assessment toward new treatments
  • Guide RAWF’s research agenda and priorities
  • Build a patient community around shared goal

Action plan:

  • Develop patient and caregiver focus groups and/or advisory boards
  • Identify concepts of interest
  • Develop survey(s), preferably implemented in the registry


  • The CP research ecosystem is fragmented, mainly centered on tumor or HO research and treatment
  • The voice of survivors and caregivers is lacking
  • There is no dedicated multidisciplinary research platform centered on CP treatment and quality of life


  • Create an integrated network of CP experts in research and care centered around patients’ and caregivers’ needs and treatment priorities
  • Refine the RAWF’s research strategy and implementation
  • Build on existing networks/consortia
  • Share and disseminate data, information, tools
  • Create a multi-stakeholder and disciplinary collaborative network

Action plan:

As a patient organization solely dedicated to accelerating treatment and improving the quality of life of CP patients, the RAWF will bring together an international group of clinicians, researchers and representatives from the pharmaceutical industry, government, patient, and caregiver communities. This multidisciplinary convening of stakeholders will work to integrate their expertise and views on opportunities, gaps, and challenges facing CP patients to drive consensus on important patient-centered outcomes research.

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