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European Biopharmaceutical Review

Patient Voices in the Industry




Orphan drugs have become a $153 billion pharmaceutical market since the first orphan drug legislation passed in the US in 1983. This market is expected to continue to grow over the next five years at approximately 12% per year, with approximately 20% of global drug sales being for orphan disease treatments (1). In 2018, the FDA approved drugs for 89 different orphan disease indications (2). In Europe, the overall number of approved drugs is smaller, but a similar increase in approvals has occurred, and 2018 was the largest year to date with 21 approved products (3). Despite this increase in drug approvals, an estimated 95% of approximately 7,000 rare diseases are without effective treatment, resulting in substantial patient suffering and loss of life (4). The rapid growth in the market for rare disease treatments has placed substantial burdens on patients. The expansion of drug development to meet the rare disease market has increased pressure on patients and in the drug regulatory process. Patients are also frequently being called upon as advocates by pharma companies, healthcare payers, and patient associations.

The Patient Role in Rare Disease Advocacy

Many patients spend several years in search of an accurate diagnosis about their condition. For example, in the UK and US, the average time to diagnosis for rare diseases was found to be five to seven years, and in this time there were two to three incorrect diagnoses for a given condition (5). Once a diagnosis is obtained, patients are likely to be overwhelmed with information and frequently disappointed about the lack of effective treatment for their disease. This lack of effective treatment and the need to better understand their disease are two of the main motivating factors for patients to seek out or form self-help groups of similar patients.

These patient groups often develop into vocal advocates for improved recognition and treatment of a specific rare disease. These activities may include a resource for finding local treatment, participation in a clinical trial, the development of a patient registry, support for research, and local political activities to advocate more funding for research and treatment. As a large number of rare disease groups are run predominately by volunteers, several umbrella organisations have developed to act as a single regional voice for rare disease patient advocacy groups and provide resources for individual patient group activities. Examples of these include the National Organization for Rare Disorders, the European Organisation for Rare Diseases (EURORDIS), the Chinese Organization for Rare Disorders, and the Japan Intractable Diseases Information Centre.

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William C Maier, MD, PhD is Chief Scientific Officer and Head of Rare Disease Research, Commercialisation and Outcomes Services at ICON. William has over 25 years of experience in drug development and commercialisation at pharmaceutical companies in Europe, Canada, the US, and Asia. At ICON he works with pharmaceutical companies throughout the world to provide regulatory, strategic, and scientific guidance on medical treatment development and commercialisation. William is a member of the EMEA’s European Network of Centres for Pharmacoepidemiology and Pharmacovigilance. Additionally, he is a frequent speaker at medical conferences and has had academic appointments in the Royal Society of Medicine in the UK.

Kelly Franchetti, RN CCRN CEN is Vice President and Global Head of Patients Insights and Engagement at ICON. Kelly brings extensive experience in partnering with patients, advocacy, and clinical research with a specialty in rare disease programs. She is a leading developer of patient engagement solutions in both clinical research and in therapeutic practice. Kelly has two decades of direct patient experience in hospital and industry settings. She was an active critical care registered nurse for over 18 years and an experienced caregiver for children with rare disease for over 20 years. She maintains her nursing status at multiple university health systems to grow her knowledge of patient needs and behaviours.
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William C Maier
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Kelly Franchetti
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