The Future of Patient Advocacy and Orphan Drug Development: Challenges and Opportunities

Despite advances in the orphan drug development process and the more prominent role that patient advocacy groups now play, researchers in this area still face a number of challenges. But, fortunately, increased collaboration between patient groups, industry leaders, and regulatory bodies can help overcome these hurdles.

Barriers to orphan drug research include:

  1. Small population By definition, rare diseases affect only a fraction of the world’s population, making it difficult to collect enough volunteers for a scientifically and statistically sound clinical trial.
  2. Geographically widespread population Not only are rare disease patient populations small, but they are often spread out all over the world, raising additional logistical challenges to trial recruitment and research.
  3. Research naive sites Maintaining the accuracy and credibility of data during a trial is of the utmost importance, and it presents a particular challenge in the rare disease space.
  4. Unknown or sparse natural history A natural history is an important tool that allows researchers to understand how a disease progresses without intervention, but there is a frustrating lack of this data available currently.
  5. Lack of defined biomarkers As scientists make progress in genomics and begin to identify more precise metrics for success, it will become increasingly important to employ use of biomarkers in designing trials and treatments.
  6. Lack of surrogate endpoints The central question of a trial is how to define success, but in the case of rare and poorly understood diseases, this may mean thinking beyond conventional metrics to consider other patient-reported factors.
  7. Recruitment and retention Since people with rare diseases are often quite ill, it can be a challenge — both logistically and psychologically — to recruit and retain them for clinical observation.

Patient advocacy groups are in a unique position to provide insights and recommendations that address each of these obstacles. Encouraging greater collaboration and participation of patients is an important step in overcoming existing challenges in drug development. Yet, it’s important to consider the particular needs and wants of both the pharmaceutical industry and patient groups to ensure that all parties are benefiting from increased engagement.

In general, we can identify three main areas in which the pharmaceutical industry can benefit from patient input:

  1. Input Who better than patient advocates to help steer researchers in developing trials and identifying outcome measures?
  2. Access Patient groups can provide relationships and connections to opinion leaders for a particular disease and sites where trial recruitment can be more successful.
  3. Data As mentioned, natural history and pre-screening data are incredibly important, yet not widely available to researchers, so advocacy groups can play a key role here.

On the other hand, there has often been a tenuous relationship between the pharmaceutical industry and patient groups. In order to foster a more productive and beneficial relationship, it’s important to consider the following needs of advocacy groups:

  1. Involvement Transparency and open communication throughout the research and development process are essential.
  2. Partnership Advocacy groups want to make their voices heard, and they also expect that industry groups will offer financial and other kinds of community support that go beyond a specific trial.
  3. Respect Patient groups need to maintain credibility within the patient community, so relationships with pharmaceutical companies must be perceived as mutually beneficial and respectful to all involved.

Orphan drug development has been a long, uphill battle, but due to recent legislation and greater collaboration between patient advocacy groups, the pharmaceutical industry and government regulators, currently, there are unprecedented opportunities for advancement. All parties involved are seeking more cures, for more patients, than ever before, and systems are in place that can make this a reality.

Learn more about the future of orphan drug development and patient advocacy by watching rare disease expert, Juliet Moritz speak at World Orphan Drug Conference Europe.

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