This has been the case for spinal cord injury; for example, Acorda was granted orphan drug status in developing the drug 4-aminopyridine (fampridine) to treat spasticity, since spasticity affects less than 200,000 individuals with chronic spinal cord injuries. In addition, Proneuron received orphan drug status for its autologous incubated macrophage therapy (Procord), which is designed to improve motor and sensory neurological outcomes in individuals with acute complete spinal cord injuries.
Some of the health outcomes of a spinal cord injury—such as spasticity, chronic pain, and pressure sores—also affect individuals with other diseases and conditions. The existence of these larger markets offers a potentially greater incentive for the private sector to invest in research and development. For example, medications to alleviate spasticity would benefit not only individuals with spinal cord injuries but also individuals with multiple sclerosis. This is especially compelling for interventions for the alleviation of pressure sores, a chronic problem affecting many elderly and bed-ridden patients.
To increase the amount of industry investment in spinal cord injury research, mechanisms need to be developed to provide incentives and overcome impediments. Public-private partnerships, joint postdoctoral internships between industry and academia, and other mechanisms that would facilitate collaborative efforts and move the science closer to clinical applications should be explored. As an initial step in this process, a conference or workshop is needed to bring together the relevant stakeholders to discuss current barriers and develop collaborative approaches and incentives. One topic could be modifying the standards used to obtain “orphan” status to be based on the percentage of the United States population affected by the disease or disorder rather than the total number of affected individuals. Discussions are needed among the range of stakeholders in the development of new therapeutics for spinal cord injuries, including federal and state health agencies; professional societies in neuroscience and clinical medicine; academic institutions; basic and clinical researchers; and biotechnology, medical device, and pharmaceutical companies.
Registries are online systems for storing and relating information about individuals (Box 6-5). A population-based spinal cord injuries registry would collect standardized information on the incidence, type, and causes of spinal cord injuries for a geographically defined population, including spinal cord injury mortality among cases not seen or admitted for hospital care. This information would allow greater insights into how spinal cord injuries might be prevented but would be of limited use in the development