Expanded access

Policy on Compassionate Use and Expanded Access

 

Endocyte is a biopharmaceutical company focused on developing targeted therapies for the personalized treatment of cancer. To do this, we conduct or sponsor clinical trials which allow patients, in consultation with their physician(s), to enroll in these trials when a patient meets the eligibility criteria. Drugs that are not yet approved and are in clinical trials are called investigational drugs. Clinical trials are research studies specifically designed to determine if an investigational drug is safe and effective for patients. Results generated from these trials provide regulatory authorities with the necessary information to evaluate whether the investigational drug should be approved as a new medicine for potential widespread use.

We appreciate the urgency to bring potential new breakthrough treatments to every patient who needs them as fast as possible. We know patients are waiting and this sense of urgency drives us every day.

Endocyte believes that participating in clinical trials accepted by the U.S. Food and Drug Administration, European Medicines Agency, Japan’s Pharmaceuticals and Medical Devices Agency, Germany’s Federal Institute for Drugs and Medical Devices (BfArM), Health Canada, Medicines Australia, and other global regulatory authorities is the best way for patients to access investigational drugs for their diseases and is the most efficient way to determine if these investigational drugs can be approved by regulators as new medicines.

When terminally ill patients don’t qualify for clinical trials and exhaust all available treatment options, the law in the United States and most countries allows an investigational drug to be given outside of a clinical trial as a last resort under certain circumstances if the drug’s manufacturer, the treating physician and the patient all consent. This is called compassionate use, or, variously, right-to-try, or also expanded access. However, new medicines only can be approved when supported by evidence from adequate, well-controlled clinical trials. Because it is not known during clinical development if an investigational drug is indeed safe or effective for its intended human use, compassionate use, and expanded access through right-to-try legislation, may present risks for both the immediate patient and the clinical development program designed to help all future patients. For the requesting patient, compassionate use may bring potential safety risks or a false sense of hope that the investigational drug will work. Compassionate use also has the potential to delay or undermine the approval of a new medicine being sought by so many, especially when the investigational drug is in short supply.

For these reasons, enrollment in clinical trials is currently the only way for patients to gain access to Endocyte investigational therapies. We recognize that not all patients will meet eligibility requirements for these trials, and as more clinical data on the safety and effectiveness of our experimental medicines become available, this position may change. Endocyte supports a wide variety of clinical trials, ranging from those conducted by Endocyte to those initiated by investigators which our company supports with free product and other resources. Physicians interested in conducting their own trials with our investigational drugs may contact us with a research proposal.

Additional Information

We encourage patients to speak first with their physician about their eligibility to enroll in a clinical trial.

Treating physicians, patients and/or caregivers interested in learning more about Endocyte’s investigational drugs currently undergoing available clinical studies can go to https://clinicaltrials.gov/, and search by our company name, disease, or the particular investigational drug. To learn more about clinical trials sponsored by Endocyte, including our new global phase 3 registration trial evaluating 177Lu-PSMA-617 for treatment of men with progressive, metastatic castration-resistant prostate cancer (mCRPC), called the VISION study, visit https://endocyte.com/pipeline/clinical-trials/, and https://visionclinicaltrial.com/. It is important to remember that the safety and efficacy of our company’s investigational drugs has not been established. There is no guarantee that any of the agents profiled in these hyperlinks will receive regulatory approval and become commercially available for the intended uses being investigated.

Physicians seeking help on behalf of their patients may contact Endocyte by writing to expanded-access@endocyte.com. We anticipate acknowledging receipt of any such requests within seven (7) business days of receipt.

As authorized by the 21st Century Cures Act, Endocyte may revise this position statement at any time. The posting of this position statement by Endocyte shall not serve as a guarantee of access to any specific investigational drug by any individual patient.