Rare Diseases in Oncology and Hematology Therapeutic Expertise
Drug developers turn to the PPD™ clinical research business of Thermo Fisher Scientific for our extensive experience supporting rare diseases in oncology and hematology clinical trials.
Rare diseases in oncology and hematology encompass a wide range of conditions that affect the blood, bone marrow and various organs, often involving complex and life-threatening symptoms.
Collaborating with a CRO partner that understands the nuances of a wide range of indications, including rare cancers, uncommon blood malignancies and benign hematologic conditions, is of paramount importance in developing effective strategies to treat them.
Advancements in the characterization of genetic mutations in cancers are making even common cancers, such as lung cancer, exhibit characteristics of rare diseases. Genetic testing plays a crucial role in distinguishing between tumor types, guiding personalized treatment plans and predicting patient outcomes.
Improving the lives of individuals impacted by rare diseases in oncology and hematology through research advancements and patient-centric clinical trials drives us. We prioritize understanding the needs of patients, caregivers and health care professionals. Through our innovative research and patient-centric approach, we bring hope and transformative therapies to those battling these conditions.
Our medical, operational and regulatory expertise — combined with our real-world experience — enables us to design and operationalize studies with a customized approach for each indication.
Multiple indications in both rare cancers and rare hematology conditions
Oncology
- Biliary tract cancer
- Cholangiocarcinoma
- Endometrial stromal tumors
- Giant cell tumors
- Glioblastoma
- Glioma
- Neuroendocrine tumors
- Neurofibromatosis
- Osteosarcoma
- Ovarian cancer
- Pancreatic cancer
- Renal cancer
- Rhabdomyosarcoma
- Small cell lung cancer
Malignant hematology
- Acute lymphoblastic leukemia
- Acute myelogenous leukemia
- Acute myeloid leukemia
- B-cell lymphoma
- Chronic lymphocytic leukemia
- Cutaneous T-cell lymphoma
- Diffuse large B-cell lymphoma
- Follicular lymphoma
- Mantle-Cell lymphoma
- Myelodysplastic syndromes
- Myelofibrosis
- Multiple myeloma
- Prolymphocytic B-cell leukemia
- Systemic mastocytosis
Benign hematology
- Anemia
- Beta-thalassemia
- Graft vs. host disease
- Hemophilia A
- Hemophilia B
- Paroxysmal nocturnal hemoglobinuria
- Polycythemia vera
- Sickle cell disease
- Thrombocytopenia
- Thrombocytopenic purpura
- Von Willebrand disease
- Waldenstrom macroglobulinemia
In the past five years, our expert rare oncology and hematology team has supported:
oncology trials
hematology trials
patients
global sites
indications
countries
27 Phase I studies
25 Phase I/II studies
53 Phase II studies
62 Phase III
End-to-end operational expertise for rare cancers and rare hematology conditions
Fast-profiling of key biomarkers and comprehensive genomic profiling for precision oncology clinical trial recruitment
Leverage a team of oncology and hematology experts to build or optimize planning and design scenarios, recruit patients and support sites
Optimized, data-driven country and trial site selection and activation
Deep expertise and regulatory insights with complex rare studies
Deep connections with sites and key opinion leaders
Partnership sites with strategic relationships that prioritize our clinical studies
Strategies to optimize patient and caregiver experience
A global network of experienced principal investigators, with indication-specific expertise
Making patients the center of everything we do
We recognize how burdensome clinical trials in rare disease can be for patients and their caregivers. Our host of supportive concierge services reduces the burden for both sites and patients and makes it easier for patients to participate:
- Minimize blood draws
- Use of local labs
- Direct-to-patient solutions
- Transportation services
- Patient concierge options
- Home health care
- Telemedicine and home health care services
- Digital and decentralized protocols
- Wearable and mobile pagers
Continuous engagement and support from our hematology and oncology rare disease center of excellence
- Our hematology and oncology (HemOnc) rare disease center of excellence offers access to more than 20 rare disease experts, including former U.S. Food and Drug Administration (FDA) officials, who provide strategic insights across all indications to inform and optimize clinical and regulatory strategy development.
- We recognize that there are special challenges in rare cancer and rare hematology conditions, so we provide patient and caregiver services informed by our long-term partnerships with patient advocacy groups, as well as a compassionate, open discourse with individual participants and researchers.
- Our HemOnc rare disease center of excellence focuses on delivering transformational science and diverse, cross-functional services to the life science leaders we work with every day. This means driving innovative and executable trial designs, implementing operational strategies, and focusing intently on reducing the burdens of clinical research participation — all in an effort to meet the needs of patients and families with rare diseases and deliver hope through therapies.
- Our dedicated, experienced teams deliver cross-functional expertise providing innovative, patient-centric solutions and training to our study teams and sites to skillfully address the most complex challenges of small, globally dispersed populations — across all therapeutic areas
Rare diseases in oncology and hematology
Contact us to support your next radiopharmaceuticals trial.