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Compassionate Use Policy

Dicerna Pharmaceuticals, Inc., DCR-PHXC

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1. Dicerna’s Mission

  1. Dicerna Pharmaceuticals, Inc.’s (Dicerna) mission is to discover and develop RNAi-based therapeutics for diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. Our lead product candidate is DCR-PHXC for the potential treatment of all forms of primary hyperoxaluria (PH).
  2. Dicerna engages in clinical research with the goal of obtaining regulatory approval of its products to address important patient needs.

2. Commitment to Patient Communities

  1. Dicerna is committed to working collaboratively with the communities we aim to serve.
  2. Dicerna is committed to supporting the needs of patients, families, and patient advocates by providing disease education materials, resources for connecting with patient communities and support groups, and updates on current development programs.

3. Clinical Trials Overview/Information

  1. There are three stages in the discovery and development process: discovery, development, and delivery.
    1. In the discovery stage, the Company identifies a potential medicine (or drug candidate) for a specific disease.
    2. In the development phase, the Company conducts research and tests the potential therapy, evaluating the drug candidate through clinical trials in humans. Once the Company begins testing the drug candidate in clinical trials, it is considered an investigational therapy.
    3. Finally, in the delivery phase, the Company works with regulatory agencies to seek approval and make the drug available to all patients who need it, based on a review of its benefits, potential risks, and needs within the patient community. Once the drug receives regulatory approval in a country, physicians are able to prescribe the therapy to patients.
  2. Information about an investigational therapy’s efficacy and safety is generally limited at the stage at which expanded access may be sought. Studies relating to the toxicity of the investigational therapy will generally have been completed and analyzed, and early studies looking at how the investigational therapy is handled by the body will have been completed. However, there is still uncertainty about the investigational therapy’s efficacy and safety profile in the larger patient population until clinical trials are completed, including which side effects it can cause, and the best way to give the investigational therapy to patients, such as the exact dose to use and the dose frequency, which may not yet have been established. These uncertainties mean that very careful assessments must be undertaken before making investigational therapies available for compassionate use.

4. Purpose of the Policy

  1. This policy only applies to Dicerna’s lead compound, DCR-PHXC, for the treatment of all forms of PH, which is the Company’s only product sufficiently advanced to consider requests for compassionate use.
  2. In general, Dicerna believes that participating in clinical trials is the best way for patients to access investigational therapies prior to approval. The completion of these rigorous scientific studies is a critical step to securing the regulatory approval needed to provide access to treatment for the greatest number of patients with a specific disease.
  3. In some extreme circumstances when participating in clinical trials is not possible, patients with life-threatening diseases or conditions may seek special access to investigational therapies outside of a clinical trial setting. These situations are typically referred to as compassionate use, but can also be known as expanded access, early access, pre-approval access, and emergency use.

5. Compassionate Use Criteria/Considerations (Eligibility)

  1. We also recognize that not all patients may be eligible or able to enroll in a clinical trial. For this reason, we support access to investigational therapies for people with life-threatening disease who meet specific requirements.
  2. At Dicerna, a compassionate use program, or a single request for compassionate use of an investigational therapy, may only be considered if all of the following conditions are met:
    1. The disease or condition being studied is life-threatening.
    2. There are no comparable or satisfactory alternative therapies or clinical trials available.
    3. Sufficient preliminary efficacy and safety data exist for the investigational therapy and/or investigational therapy delivery device in order for Dicerna to make a benefit-risk analysis consistent with the establishment of a compassionate use program.
    4. Sufficient clinical data are available to identify an appropriate dose.
    5. A patient’s treating physician and Dicerna’s Chief Medical Officer both believe there is the potential for the specific patient under consideration to reasonably expect benefit from the investigational therapy, and there is robust evidence to support the possibility that the patient will benefit.
    6. Patients who have experienced exceptional safety risks outside of a clinical trial, which have not been studied to better understand any potential problems.
    7. Adequate investigational therapy supply can be assured to support both ongoing clinical trials and the approved compassionate use.
    8. The patient is not eligible for or a candidate for one of the Dicerna-sponsored clinical trials. Geographic limitations to participation in a trial would typically not mean a patient is not eligible.
    9. Compassionate access will not adversely impact the clinical development program, in particular, the conduct of any pivotal clinical trial that is required for regulatory approval.
    10. The request is made by the patient’s treating physician, unsolicited by Dicerna or any other individual or organization. This request will provide evidence that the patient will have continual access to the level of medical supervision appropriate to safeguard the patient while being exposed to an investigational therapy.
    11. There is a regulatory mechanism in the country or region that allows for such compassionate access.
  3. The above criteria are those that Dicerna will consider in determining whether to offer compassionate use; however, Dicerna cannot make a guarantee that a compassionate use program will be available, and, even if a compassionate use program is offered, Dicerna cannot make a guarantee that the investigational therapy will be available to a particular patient.
  4. Dicerna is committed to a fair and impartial evaluation of each request for access to its investigational therapies. All decisions are based solely on clinical circumstances and are guided by the principles outlined above.

6. Procedures for Making Compassionate Use Requests

  1. If all the conditions listed in 5(b) are met, Dicerna will consider compassionate use requests from treating physicians consistent with all applicable local and national laws and regulations. Any pre-approval access to an investigational therapy must at all times comply with the applicable country- and state-specific laws and regulations, including information privacy and medicine importation requirements. Approvals from applicable regulatory bodies and by an Institutional Review Board or Ethics Committee from the treating hospital must be secured.
  2. If approved, the patient (or his or her guardian, if applicable) must provide informed consent and consent to comply with the safety and monitoring requirements defined by Dicerna. The treating physician must also agree to comply with safety and monitoring requirements.
  3. Compassionate use will cease to be made available if, as a result of clinical trials, the investigational therapy does not demonstrate a positive risk/benefit to patients or leads to considerable risk of injury to a requesting patient.
  4. For patients that meet Dicerna’s criteria, treating physicians can make a request via compassionateuse@dicerna.com. All requests must include the treating physician’s name, contact information, professional designations or qualifications, a medical rationale for the request and supporting medical documentation, including an explanation of why alternative therapy cannot be used.
  5. Medical professionals at Dicerna who are familiar with the data collected on the investigational therapy evaluate the request and respond based on the scientific evidence available to the company at the time of the request. Dicerna commits to respond to compassionate access requests within no more than five business days of receipt of the formal request and supporting medical documentation.
  6. More information about Dicerna’s clinical trials is available at www.clinicaltrials.gov. Patients or physicians can also search clinicaltrials.org for expanded access records by disease and investigator name.
  7. Dicerna will fulfil all regulatory requirements to post details of compassionate use activities on various independent registers, including ClinicalTrials.gov and the European Medicines Agency Compassionate Use website.
  8. Any question about this policy can be directed to the same email address above.