Navigating the Road to Successful Orphan Drug Commercialization and Launch

Navigating the Road to Successful Orphan Drug Commercialization and Launch

May 24, 2019PAP-Q2-2019-NI-003
The Advanced Therapeutics Issue Feature: Inside the World of Orphan Drugs

Developments in Orphan Drugs: Part 3

Some drug candidates with strong potential to treat rare diseases may not be suitable for commercialization. The development and approval process may be too lengthy and expensive. Establishing a geographically dispersed and highly complex supply chain may not be feasible, and implementing the required postmarketing clinical studies may be very difficult. Pharmaceutical companies are taking a variety of approaches to overcome these challenges.

Drug Repurposing

One such approach to reducing the challenges of orphan drug development is drug repurposing (DRPx). Approximately one-fifth of approved orphan drugs are repurposed existing drug products.1 For these drugs, development costs and times are significantly reduced (1/20th and ½ compared with NCEs, respectively). The risk of failure is also lower, because the safety profiles of repurposed drugs have already been approved, and the manufacturing processes intellectual property strategies are already established. 

Patient-Centric Packaging

Medication adherence continues to be a key issue for the pharmaceutical industry and regulatory agencies. Even for the newest orphan drugs designed to treat rare diseases for which no medications have been previously available, inconvenient or difficult-to-use products may lead to patient noncompliance. 

Since orphan drugs generally target much smaller patient populations, it is essential to take into consideration the specific needs and characteristics of the targeted group. Pediatric patients often have very different requirements than adults, and elderly patients have their own preferences. Patient needs with respect to the delivery technology and packaging design should be considered from the earliest stages of a development project, with revisions made throughout clinical trial phases based on actual patient experiences.2

Five factors should be addressed when planning for an orphan drug supply chain: the number of patients, where they will be treated, any special handling requirements, the expected inventory management needs and the design of the ordering process.

Supply Chain Solutions Q2_Feature_sidebar15

Although the total number of potential patients is relatively small, orphan drugs must often be delivered to more different locations around the world than non-orphan drugs, including highly remote sites. When orphan drugs are developed as personalized medicines, the complexities are multiplied. Ensuring that the right dose reaches the right patient at the right time and under the right conditions (e.g., temperature control) can be challenging. 

If patients cannot receive new medications designed to treat their rare diseases, regardless of how effective the drugs may be, there is no value in commercializing them. Fortunately, these issues can and have been overcome.

Advanced and precise planning is a priority when developing supply chain solutions for rare disease treatments.3 Five factors should be addressed when planning for an orphan drug supply chain: the number of patients, where they will be treated, any special handling requirements, the expected inventory management needs and the design of the ordering process.4

Consideration of patient needs from the beginning is important, as is understanding the properties of the drug product and how they may impact labeling and shipping requirements (e.g., packaging materials and design, temperature control, time sensitivity) and customs documentation.3 Insurance coverage and financial questions for patients must be addressed. It is also essential to understand the impact of increasing patient numbers on manufacturing and distribution needs. Distributors may also need to collect clinical data to support the manufacturer’s requirements for demonstration of treatment effectiveness.

Further value can be added by providing patient and provider education programs that cover the unique characteristics of the drug, any specific requirements for administration and any available prescribing tips.4 Provision of financial assistance and assistance with reimbursement efforts is also a recommended component for any orphan drug distribution program. Drugmakers can provide value for themselves through the implementation of a system for the collection of transactional and other data that allows for analysis of trends in purchasing, inventory, product returns and other factors.

A comprehensive risk assessment must be performed to ensure that the commercial viability of a rare disease treatment is balanced with the feasibility, cost and effort required to establish an effective supply chain.3

The Role of Commercialization Partners

Often, outsourcing partners that can provide real-time insight into patient and provider needs and key market issues, such as preferred patient treatment experiences and adherence behaviors, can have a large impact on the success of an orphan drug commercialization effort.5 For instance, they can provide information on the typical comorbidities suffered by patients with specific rare diseases that affect their needs and preferences for obtaining and taking their medications, as well as the potential for undesirable drug interactions. For many rare diseases, a hub model supported by a case manager is an effective approach to addressing these issues and others, such as reimbursement authorization and affordability.

An effective commercialization partner can also ensure that the right IT systems are in place to collect and analyze the most valuable data to demonstrate the health and economic benefits of their orphan drugs once they reach the market.5

Successful Orphan Drug Launches

McKinsey has identified four strategic pillars of successful orphan drug launches: a commitment to the rare disease community, use of innovative methods for patient identification, use of a tactical approach to patient access and extensive support for patients and caregivers as they navigate the healthcare system.6 For each drug and each rare disease, however, the solution will be unique. The launch team — which must be cross-functional and operate in a highly collaborative manner — needs to integrate all insights gained to develop a launch strategy optimized to support the specific patient population.

Read Part 1: An Expanding Orphan Drug Market
Read Part 2: Managing the Risks Associated with
Orphan Drug Development and Manufacturing



  1. “The potential of drug repurposing in orphan drug development.” Pharmaceutical Technology. 9 Mar. 2018. Web.
  2. Allen, Daphne. “Contract manufacturing and packaging help bring orphan drug to market.” Packaging Digest. 26 Sep. 2016. Web.
  3. Ribbink, Kim. “Supply and Demand: Getting Rare Disease Treatments to Patients.” PharmaVOICE. Jun. 2017. Web.
  4. Kissling, Kevin. “Prescribe Robust Distribution to Commercialize Your Orphan Drug.” McKesson Blog. 18 Jun. 2018. Web.
  5. Odutola, Akin. “Partnering Up for Successful Orphan Drug Commercialization.” PM360. 10 Apr. 2017. Web.
  6. Ascher, Jan, Arafat M’lika, Jeff Graf and Maha Prabhakaran. “How to successfully launch a rare disease drug in a patient-centric world.” McKinsey. Jan. 2017. Web.
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