BackgroundRare diseases are a global public health concern, affecting an estimated 350 million individuals. Only 5% of approximately 7000 known rare diseases have a treatment, and only about half have a patient advocacy organization. Biopharmaceutical companies face complex challenges in developing treatments for rare diseases. Patient advocacy organizations may play a major role by positively influencing research and development, clinical trials, and regulations. Thus, collaboration among patient advocacy organizations and industry is essential to bring new therapeutics to patients.MethodsWe identified an unmet need for guidelines on day-to-day decision-making by rare disease patient advocacy organizations when working with biopharmaceutical partners. We convened an Independent Expert Panel experienced in collaborations between patient advocacy organizations and biopharmaceutical companies (April 2017) to develop consensus guidelines for these relationships. The guidelines were based on an original version by the International Fibrodysplasia Ossificans Progressiva Association (IFOPA). The Expert Panel reviewed and broadened these to be applicable to all patient advocacy organizations. Comments on the draft Guidelines were provided first by Panel participants and subsequently by six independent experts from patient advocacy organizations and industry.ResultsThe Panel comprised four experts from the rare disease community who lead patient advocacy organizations; three leaders who perform advocacy functions within biopharmaceutical companies; and two facilitators, both having leadership experience in rare diseases and industry. The finalized Guidelines consist of four main sections: Identification and Engagement With Companies, Patient Engagement and Patient Privacy, Financial Contributions, and Clinical Trial Communication and Support. The Guidelines address the daily considerations, choices, and consequences of patient advocacy organizations as they engage with biopharmaceutical companies, and offer recommendations for volunteer/paid leaders of the organizations on how to interact in a thoughtful, responsible, ethical way that engenders trust.ConclusionsThese Guidelines recommend best practices and standards for interactions between patient advocacy organizations and industry that will ultimately have a positive effect on the development of novel treatments. Patient advocacy organizations will be provided free access to these Guidelines to help bring clarification to day-to-day decision-making around their interactions, and for use as a living document with the potential for regular revisions and updates.

Because of the variability of HIV, the first AIDS vaccine is likely to be only partially effective. There is some concern among scientists that a low-efficacy vaccine could worsen the HIV epidemic if vaccinated individuals increase their risk behavior under the false assumption of immunity. To address this concern, we constructed a dynamic compartmental model that simulated the course of the HIV/AIDS epidemic in a population of injection drug users in Bangkok, Thailand. The model calculated long-term HIV prevalence, number of AIDS cases, and total population size for two scenarios: vaccination program versus no vaccination program. We used sensitivity analyses to evaluate the impact of postvaccination risk behavior change on HIV prevalence. A 75% effective vaccine led to a 40-year HIV prevalence of 37% with vaccination and 50% without vaccination. Postvaccination behavior change had only a limited effect on the results with a 75% effective vaccine but a significant effect with a 30% effective vaccine. If 90% of low-risk individuals responded to a 30% effective vaccine with increased high-risk behavior, the benefit of vaccination disappeared. These results agree with analyses of the epidemic among gay men. If injection drug behavior is indeed modifiable, our findings have significant policy and planning implications.

Because of the variability of HIV, the first AIDS vaccine is likely to be only partially effective. There is some concern among scientists that a low-efficacy vaccine could worsen the HIV epidemic if vaccinated individuals increase their risk behavior under the false assumption of immunity. To address this concern, we constructed a dynamic compartmental model that simulated the course of the HIV/AIDS epidemic in a population of injection drug users in Bangkok, Thailand. The model calculated long-term HIV prevalence, number of AIDS cases, and total population size for two scenarios: vaccination program versus no vaccination program. We used sensitivity analyses to evaluate the impact of postvaccination risk behavior change on HIV prevalence. A 75% effective vaccine led to a 40-year HIV prevalence of 37% with vaccination and 50% without vaccination. Postvaccination behavior change had only a limited effect on the results with a 75% effective vaccine but a significant effect with a 30% effective vaccine. If 90% of low-risk individuals responded to a 30% effective vaccine with increased high-risk behavior, the benefit of vaccination disappeared. These results agree with analyses of the epidemic among gay men. If injection drug behavior is indeed modifiable, our findings have significant policy and planning implications.