Managed Access to Investigational Therapy

Patients with serious or life-threatening conditions sometimes seek medical products that are not yet approved or available in their country. To address these needs, PellePharm evaluates early access to medicine for eligible patients under a “Managed Access” program.  Managed Access is also sometimes called Early Access, Named Patient, Special Access, Compassionate Use, or Expanded Access, and refers to the use of an investigational medicine outside of a traditional clinical trial setting. We recognize the difficulty of receiving a rare disease diagnosis for you or someone in your family, and the impact that this diagnosis may have on your quality of life, particularly when no safe and effective therapies are approved for the disease. Our goal is to support patients, families and physicians as you explore the options that may be available to help.

We demonstrate our commitment to patients and families by developing potential new therapies for rare skin diseases, evaluating their safety and efficacy, and making them available to patients around the world through a variety of mechanisms allowed by regulatory authorities.  We work hard to advance clinical trials as quickly as possible and to navigate these therapies through the approval process to make them available by prescription through your doctor. But even then, urgent need can occur between the time that we start work on an investigational treatment and the time that it becomes generally available.

As an important part of our commitment to patients with rare skin diseases, PellePharm may make investigational therapy available to qualified patients, under certain circumstances of critical and compelling need, when the patient is not eligible for a clinical trial, and when the data and other factors support the proposed use, through our Managed Access program.

Clinical Study Information

The primary and preferred mechanism for access to investigational therapies is by participation in a clinical study.  An investigational therapy is a potential new medicine that has not yet been approved by regulatory authorities such as the FDA in the United States or the European Medicines Agency (EMA) in Europe.  Before a product can be approved or prescribed, it must undergo well-controlled clinical studies. Clinical studies collect the information necessary to understand the safety and efficacy of the potential therapy allowing for regulatory authority review and potential approval.

To participate in a clinical study, you must meet certain eligibility criteria.  These criteria identify disease characteristics which can allow researchers to better evaluate the investigational therapy.  You should discuss participation in a clinical study with a physician who is familiar with your medical history and current status.

For information on ongoing PellePharm clinical studies:

  • Visit and search registered clinical studies for the disease. To search by company, PellePharm, click here.
    • If you locate a clinical study with a participating site that you are interested in, you or your physician may contact that site directly for more information.
Managed Access

At PellePharm, we recognize that some patients with serious or life-threatening diseases may not be able or eligible to participate in clinical studies and may not have other treatment options for their condition.

PellePharm evaluates all requests for individual patients to receive investigational therapies outside of a clinical study on a case-by-case basis.

As a company, PellePharm is committed to helping and supporting patients through their challenges wherever and whenever possible.  Unfortunately, not every situation will be one where we can help.

Who is Eligible

PellePharm’s decisions for managed access will be made on a fair and equitable basis.  Each disease that we are studying may have different specific criteria for managed access, due to potential differences in urgency and the data available to support safety (potential risks of the therapy) and efficacy (potential benefits of the therapy).   The following criteria must be met for a patient to participate in PellePharm’s Managed Access program:

  • The patient has a serious, irreversibly disabling or immediately life-threatening disease or condition;
  • The patient is not eligible to enroll in a clinical trial;
  • The patient has exhausted all reasonable available treatment options;
  • The possible benefit from investigational patidegib topical gel therapy, based on scientific evidence and confirmed by the treating physician, outweighs any known or potential risks;
  • Supply levels of the investigational therapy are available to meet the request;
  • An appropriate local regulatory pathway exists*;
  • Provision of the investigational therapy will not interfere with ongoing clinical trial(s) or overall development program; and
  • The patient must meet any other important medical criteria established by the medical experts working on the product development program.

*Country specific laws and regulations will apply and may affect availability of the investigational therapy in that country or region.

Requesting Access

Requests for managed access to investigational therapies must be made by a qualified physician.  You and your family should discuss your situation with your treating physician including current status, available treatment options, and reason for considering treatment with an investigational therapy.  Your physician can make the request for PellePharm’s Managed Access program on your behalf.  It is important that managed access requests are made by medically trained personnel in order to avoid delays.

For Physicians / Healthcare Professionals

All requests for Managed Access for a single patient should be submitted by the patient’s treating physician.

The patient’s treating physician must provide adequate information to demonstrate that the patient meets the outlined considerations for early access.

The patient’s treating physician must be appropriately licensed, fully qualified to administer the investigational therapy and have the expertise and facilities for monitoring, managing and reporting any adverse events during the course of treatment.  The patient’s treating physician must be willing to meet and follow all applicable legal and regulatory requirements for early access while caring for the patient receiving investigational therapy and must comply with all applicable reporting requirements.

Qualified physicians can submit a request by contacting PellePharm at

Evaluation & Response

Each request will be acknowledged immediately, and reviewed carefully and fairly by the appropriate PellePharm medical experts with every effort made to provide a response promptly once we have all of the necessary information.

Please note that your request will be assessed in consideration of applicable local laws and regulations.