In this issue: Rare Disease Moonshot: Europe’s Public-Private Coalition to Erase the Rare Disease “White Spots”
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Rare Disease Moonshot: Europe’s Public-Private Coalition to Erase the Rare Disease “White Spots”
In their latest issue, the DIA Global Forum publication highlights policy advances for rare disease research at the European level. Read the full article here.
"The knowledge and infrastructure required to unlock rare disease “white spots,” diseases where there is currently no science or translational capability, call for a different strategic paradigm. The mission of the Rare Disease Moonshot is rooted in boosting public-private collaborations. Many challenges in modern medicine are too big for one organization to tackle alone. They require true collaboration between a diverse network of companies, policymakers, regulators, academics, civil society groups, and patients. Rare diseases present one such challenge." ~The authors of the article highlight.
“The Rare Disease Moonshot taps into the potential of complementary organizations committed to accelerating progress in this overlooked field. It includes the patient voice alongside those of researchers and experts in biobanks and data analytics, translational medicine, clinical trials, regulatory science, and the biopharmaceutical industry.”
The Patient Voice
YOUR VOICE MATTERS! Make it count! Turn hope into action an join us!
The patient voice -- or more specifically, patient-reported data -- is crucial for drug development and regulatory approval by the FDA and EMAE for several reasons. Including patient perspectives and experiences in the evaluation process enhances the overall understanding of a drug's effectiveness and safety.
Patient-Centric Focus: Incorporating patient-reported outcomes (PROs) ensures that drug development is centered around patients' experiences and the impact of the condition on their daily lives. This patient-centric focus aligns with the goal of developing treatments that genuinely address patients' needs and improve their quality of life.
Measuring Treatment Benefits: Patient-reported data provides direct insight into the benefits of a treatment from the patient's perspective. This information is particularly valuable in assessing the drug's impact on symptoms, functioning, and overall well-being, which may not be fully captured by traditional clinical measures alone.
Endpoint Selection: Patient-reported outcomes can help inform the selection of meaningful and relevant endpoints for clinical trials. By including endpoints that matter to patients, researchers can design studies that better reflect the real-world impact of a drug, making the results more applicable to patients' experiences.
Clinical Trial Design and Recruitment: Patient-reported data is essential in the design of clinical trials, helping researchers determine the appropriate study population, design patient-friendly protocols, and enhance recruitment strategies. This contributes to the overall success and efficiency of clinical trials.
Regulatory Requirements: Regulatory agencies, such as the FDA, increasingly recognize the importance of patient-reported data in evaluating drug safety and efficacy. In many cases, the FDA requires the inclusion of PROs in clinical trial protocols, and positive patient-reported outcomes can support a drug's approval by demonstrating its meaningful impact on patients' lives.
Labeling and Communication: Patient-reported data can influence the content of drug labels, ensuring that important information about the drug's benefits and risks is communicated clearly to healthcare providers and patients. This transparency aids in informed decision-making and appropriate use of the medication.
Post-Marketing Surveillance: Patient-reported data continues to be valuable after a drug is on the market. Monitoring patient experiences and outcomes post-approval helps identify any previously unrecognized side effects, assess long-term benefits, and guide further recommendations for use.
Health Economics and Market Access: Patient-reported outcomes contribute to health economic assessments and market access strategies. Demonstrating the positive impact of a drug on patients' lives can influence reimbursement decisions and market access, making the drug more widely available to those who need it.
In summary, patient-reported data is integral to the drug development process, providing a comprehensive understanding of a drug's benefits and risks. Its inclusion enhances the credibility of clinical trials, regulatory submissions, and post-marketing evaluations, ultimately contributing to improved healthcare decision-making and better outcomes for patients.
YOUR VOICE MATTERS! Make it count! Turn hope into action an join us!
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