Addressing Pre-Approval Global Demand

Published by Idis, 2012

Situation

A large US biotechnology company with a diverse pipeline of specialty and orphan drugs had a product in development for Stem Cell Transplantation in NonHodgkin’s Lymphoma, Multiple Myeloma and Hodgkin’s disease. While awaiting US and EU approvals, the company was receiving a high level of demand from physicians and patients outside the US generated by heightened awareness of published clinical trial data. The drug represented a major advancement over existing therapies that were currently being used offlabel with limited efficacy and significant side effects.

Access Challenge

The company wanted to provide access, but lacked the pre-approval regulatory expertise and resources to address global demand for the drug on a localized level. They needed support to address the growing amount of requests for access to the drug so that internal resources could stay focused on US/EU approvals and commercial launches.

Access Objectives

The main objective was to provide ethical access outside the US for patients with an unmet medical need. The company wanted to make sure that only appropriate patients gained access to the medication. They also needed to educate the physician community on the appropriate use of therapy as well as have a system in place to manage the capture of Adverse Events outside of the clinical trial setting. Lastly, the company needed to develop relationships with key opinion leaders (KOLs) and other clinicians in the disease area outside the trial setting.

Idis Solution

Idis designed, implemented and managed a customized Managed Access Program that enabled patients outside the US with a demonstrated need to gain access to a new treatment option. A screening mechanism was implemented to assure that a local country medic had oversight and approval of every patient prior to access. Idis also provided necessary information and training to physicians to ensure safe and timely access to the drug, and conducted due diligence in each market to assure compliance with local regulations.

MAP Results

Managed Access Programs bridged the gap between patient demand and patient access along the entire approval lifecycle, allowing patients to access the new drug prior to approval and launch. Pre-approval access was ultimately provided to more than 1300 patients across 43 countries (see figure 1 below). Over 950 physicians participated in the program.

The number of patients gaining access to the drug via the Managed Access Program grew prior to FDA approval and then continued to grow leading up to EMA approval. Figure 2A shows the number of new patients per month as well as the cumulative number of patients gaining access to the drug via the Managed Access Program (2B). The number of patients gaining access to the drug steadily increased leading up to FDA approval and continued to increase leading up to EMA approval as awareness of the drug continued to grow. Upon EMA approval, new demand via the Managed Access Program tapered off as commercial supply became available.

Figure 1

Figure 2A

Figure 2B

MAP Benefits

For Patients

• Patients gained access to a new and potentially life-saving therapy in a safe and regulated way

For Physicians

• Physicians were provided with education and experience with a new treatment option prior to approval

For the Company

• The program enabled the company to provide controlled and ethical access to meet the needs of patients who did not have viable alternative treatment options
• Management of the program by Idis allowed the company to focus internal resources on US/EU approval and commercial launches
• The company was able to develop strong relationships with healthcare professionals through the provision of local support that helped assure the best patient outcome possible