As more companies develop drugs that treat orphan diseases and other unmet medical needs, patients and physicians are advocating for early access to them, before they are approved, labeled, packaged and fully commercialized. Positive early data can ignite demand from patients and patient advocacy groups (PAGs). Patients with terminal or severely debilitating diseases may have few current treatment options and can’t wait for a drug to wind its way through the final one or two years of the regulatory process. These patients are not eligible or able to enroll in randomized clinical trials (RCTs).

Managed access programs (MAPs) offer companies an ethical, controlled and compliant way to distribute investigational products to the patients and providers who need and are demanding them. Doing so, however, can be complicated.  

Administering MAPs takes time and money, with little perceived return on investment (ROI). Companies aren’t legally required to provide them, and many don’t seriously consider them until late in development, when it’s too late to design an effective MAP. Additionally, peak demand for pre-approval access typically comes on the brink of a major regulatory submission, a time when a company’s internal resources are maximally stressed. Thus, MAPs can pose serious challenges for developers.

That said, in addition to the patient benefit, MAPs offer an opportunity to collect real-world evidence and experience ahead of market launch. They provide a platform for building relationships with and educating stakeholders in the appropriate and safe use of a new product. All this has the potential to improve ROI by generating revenue pre-approval and by providing data to support successful commercialization efforts down the road.

Companies can use MAPs to help patients while helping themselves. Organizations should evaluate the following lessons learned to design and run an efficient, cost-effective MAP.

Collect real world evidence (RWE) to support reimbursement and pricing

Regulatory requirements (product purity, safety and efficacy in a tightly defined set of patients) are different than those of payers, who look for real-world outcomes and cost benefits. MAPs offer companies the opportunity to bridge those differences with data collection mechanisms that are less expensive than RCTs but help strengthen the product value story.

Choosing useful—and even country- or population-specific—outcomes can demonstrate the real-world benefits of a drug, expand its safety database, and reveal subpopulations or indications that should be studied formally. On a price per patient basis, collecting patient data in a MAP is about 25% of the cost of running an RCT.

Invest in technologies and personal relationships to manage risk

MAPs involve assuming substantial risks, including the risk of an underfunded or poorly executed program attracting negative publicity or damaging relationships with stakeholders. Of course, not doing a MAP is also risky: failure to provide access for patients to a needed treatment also can result in negative publicity and reputational damage. Companies can mitigate these risks by leveraging technology and working to build trust with patients and providers.

Technology
From the moment a patient qualifies to receive medicine through a MAP, the clock starts ticking. The right technology can enable rapid set-up and quicker response times – critical when patients face a life-threatening disease, or the number of requests for a medicine is rising rapidly.

Customized software running on a web-based platform can expedite responses without being hampered by the scalability and quality issues often met by overburdened teams. PAREXEL has designed a technology solution that thus far has been used to treat more than 11,000 patients, and processed more than 26,000 compassionate use drug orders with a seamless scale-up. The configurable system allowed physicians to request treatment for patients and receive an immediate response on their eligibility in no more than 30 minutes (and often in as few as five), rather than wait days for paper-based forms to be reviewed manually. Similarly, physicians were able to self-register, and were guided through the workflow process by user prompts and notifications, saving time and increasing accountability for compliance with paperwork requirements.

Relationships

MAPs involve quandaries that demand personal, face-to-face interactions, such as:

 How do you convince patients and physicians that MAP drug distribution decisions are fair, impartial and transparent?

 How do you terminate a MAP once the product reaches market

 How do you terminate a MAP if the product never reaches market?

Managing denials fairly and humanely is key to administering a MAP successfully, and it’s a contingency for which many companies fail to plan. Dedicated teams are needed to educate physicians about the proper use of an experimental agent, and explain eligibility criteria or safety issues that may arise.

MAPs Enable Companies to Do Well by Doing Good

A well-executed MAP provides life-changing benefits to patients. It confers competitive advantages because a MAP helps developers do the things they must do anyway, earlier, and can convey additional benefits. They help to build relationships with stakeholders who can facilitate commercial uptake and success. They provide physicians with a chance to administer a new drug to a more heterogeneous set of patients than they can in RCTs, learning how it works best and how to give it safely. They also allow payers an opportunity to evaluate the real-world benefits of the product.

If the decision to run a MAP is made early enough, its provisions can be rolled into the overall development plan, improving the chances of success and increasing the expected ROI.