Patient Focused Drug Development: A Call for Advocacy

Quick Overview

This talk discusses the importance of patient-focused drug development and the need for a coordinated advocacy strategy at both the state and federal level. The speaker emphasizes the role of patient voices in every stage of drug development and highlights the various stakeholders involved in the process. They discuss the history and role of the FDA, as well as the importance of transparency and patient input in decision-making. The speaker also addresses the role of Congress in funding and legislation related to rare diseases, and the need for advocacy to influence priorities and actions. Access and coverage of therapies are discussed, along with the importance of data collection and patient experience in informing decision-making. The speaker concludes by calling for action and involvement in advocacy efforts at various levels.

Patient-focused drug development is crucial for the rare disease community to have a well-coordinated advocacy strategy at both the state and federal level. It is important for everyone to get involved and lend their voice in the process to ensure that patient voices are integrated into every stage of drug development.

The Drug Development Ecosystem

Drug development is a complex ecosystem that involves various stakeholders, including patients, families, researchers, physicians, Congress, federal agencies, and pharmaceutical companies. These stakeholders make decisions that impact the lives of patients. It is essential for decision-makers to understand the lived experiences of patients and for patients to understand how they can impact legislation, policies, and regulations to benefit their community.

A Multi-Pronged Approach

To effectively advocate for rare diseases, a multi-pronged approach is necessary. This approach includes focusing on regulatory matters, engaging with Congress, and addressing issues related to access and coverage. It is important to have a comprehensive strategy that encompasses all rare diseases, not just specific conditions.

The Role of Regulatory Agencies

Regulatory agencies, such as the FDA, play a crucial role in drug development. The FDA’s primary responsibilities include protecting the public from unsafe events, promoting public health, and following laws and regulations set forth by Congress. It is important for patients and advocacy groups to engage with regulatory agencies to ensure that their lived experiences and preferences for treatments are considered in the decision-making process.

Patient-Focused Drug Development

Patient-focused drug development is a game-changer for the rare disease community. It involves incorporating patient and family perspectives into the drug development process and making the FDA more transparent about how they make decisions. The FDA has launched initiatives, such as the patient-focused drug development initiative and the Benefit Risk Framework, to engage directly with patients and families and provide insight into their decision-making process.

Engaging with Regulators

Direct engagement with regulators is crucial to ensure that they understand the rare disease community. It is important for patients and families to share their lived experiences, preferences for treatments, and what constitutes meaningful benefit. Opportunities for engagement include attending listening sessions and webinars, as well as participating in interviews, focus groups, and surveys. Patient experience data, including natural history data, qualitative interviews, and registry data, is essential for informing regulatory decisions.

Advocacy with Congress

Congress plays a significant role in shaping policies and funding priorities that impact the rare disease community. Advocacy efforts with Congress involve building relationships with elected officials, sharing personal stories, and influencing funding priorities. Legislation that impacts rare diseases can be passed through collective advocacy efforts, leading to positive changes in how regulatory agencies operate and how research is funded.

Access and Coverage

Advocacy efforts should also focus on access and coverage for rare disease treatments. While there may not be approved therapies for all rare diseases, it is important to start early and work with pharmaceutical partners to ensure that when therapies are approved, they are accessible to patients. Advocacy efforts can include raising awareness at the state level, engaging with state Medicaid officials, and participating in state-specific policy advisory councils.

The Importance of Data

Data is crucial in advocating for rare diseases. Patient experience data, including caregiver preferences for treatments and clinical outcome assessments, helps inform regulatory decisions and access and coverage policies. Participating in surveys, filling out caregiver impact surveys, and updating registry profiles are all ways to contribute to data collection efforts.

Call to Action

Everyone can get involved in advocacy efforts for rare diseases. Opportunities include engaging with Congress, participating in state-specific activities, and contributing to data collection efforts. Advocacy is consistent and persistent, and it requires the collective effort of the rare disease community to make a lasting impact.

In conclusion, patient-focused drug development and advocacy are essential for the rare disease community. By engaging with regulators, Congress, and other stakeholders, patients and families can ensure that their voices are heard and that decisions are made with their best interests in mind. Together, we can make a difference in the lives of those affected by rare diseases.