Brigham Study Finds That More Than 50 Percent Of Cancer Drugs Granted Accelerated Approval By U.S. FDA Do Not Work And Sometime Kills!

Pharma News: The U.S. Food and Drug Administration's (FDA) accelerated approval program has long been hailed as a crucial pathway for providing early access to potentially life-saving drugs, particularly in the realm of cancer treatment. Established in 1992 initially to address the urgent need for HIV/AIDS medications, this program allows for expedited approval of drugs that show promising initial results, often based on surrogate endpoints such as tumor response rate or progression-free survival (PFS). While this pathway has undoubtedly facilitated quicker access to therapies for patients facing severe or fatal illnesses, recent studies have raised important questions about its efficacy and ethical implications, particularly in the context of cancer drugs.


Brigham Study Finds That More Than 50 Percent Of Cancer Drugs
Granted Accelerated Approval By U.S. FDA Do Not Work And Sometimes Kills
 
The Recent Study: Findings and Implications
A recent cohort study conducted by researchers at Brigham and Women's Hospital and Harvard Medical School sthat is covered in this Pharma News report, sheds light on the efficacy of cancer drugs granted accelerated approval between 2013 and 2017. The study analyzed 46 cancer drug-indication pairs and their performance in confirmatory trials over a period of more than five years.
 
The findings of the study are striking and raise significant concerns. Out of the 46 drug-indication pairs analyzed, only 43% demonstrated a clinical benefit in terms of overall survival (OS) or quality of life (QOL) in confirmatory trials. This means that a majority of the drugs granted accelerated approval did not ultimately prove to significantly improve patients' survival or quality of life, despite initial promising results!
 
Some of these drugs while were successful in killing the tumors and cancer kills, also killed the patient subsequently!
 
Among 48 drug-indication pairs converted to regular approval, 19 (40%) were converted. Most of these conversions to regular approval relied on measures other than OS:
-40% were converted based on OS
-44% on progression-free survival
-10% on response rate plus duration of response
-4% on response rate
-2% despite a negative confirmatory trial
 
The Role of Surrogate Endpoints: A Double-Edged Sword
One of the central issues highlighted by the study is the reliance on surrogate endpoints for accelerated approval. Surrogate endpoints, such as tumor response rate or PFS, provide indirect measures of a drug's efficacy. While these endpoints can offer valuable initial insights into a drug's potential benefits, they do not always translate into meaningful clinical outcomes for patients.
 
For example, a drug that shows a high tumor response rate may not necessarily extend patients' lives or improve their overall well-being. This discrepancy between surrogate endpoints and patient-centered outcomes underscores the complexity and challenges of evaluating drug efficacy, especially in the context of life-threatening diseases like cancer.
 
Ethical Considerations: Balancing Early Access and Evidence-Based Medicine
The accelerated approval program presents a delicate ethical balance between providing early access to promising therapies and ensuring rigorous evidence-based medicine. On one hand, accelerated approval can offer hope and potentially life-saving treatments to patients who have limited alternatives. It can also incentivize innovation and expedite the development of new drugs, particularly in areas of high unmet medical need.
 
However, this expedited pathway also comes with risks and uncertainties. The study's findings raise questions about the adequacy of post-approval confirmatory trials and the reliability of surrogate endpoints in predicting long-term clinical benefits. Patients and healthcare providers alike must navigate this landscape of uncertainty while making informed treatment decisions.
 
Transparency and Informed Consent: Empowering Patients
Central to the ethical considerations surrounding accelerated approval is the importance of transparency and informed consent. Patients have the right to be fully informed about the uncertainties and risks associated with drugs granted accelerated approval. They should understand that these medications may not have undergone the same level of rigorous testing as drugs approved through traditional pathways.
 
Healthcare providers play a crucial role in facilitating informed decision-making. They must communicate openly with patients about the limitations of drugs granted accelerated approval, the ongoing uncertainties regarding their efficacy, and the need for continued monitoring and evaluation. Empowering patients with comprehensive information allows them to actively participate in their treatment decisions and weigh the potential benefits and risks.
 
Regulatory Updates and Continuous Evaluation
In response to the challenges highlighted by studies like the one conducted by Brigham and Women's Hospital and Harvard Medical School, regulatory agencies such as the FDA have taken steps to enhance oversight and evaluation of drugs granted accelerated approval. Recent updates to the program give the FDA more authority to withdraw drugs if companies fail to meet their post-approval commitments.
 
These regulatory changes aim to strike a balance between facilitating early access to innovative therapies and ensuring robust evidence of their efficacy and safety. However, ongoing evaluation and monitoring are crucial. The healthcare landscape is constantly evolving, and new insights into drug efficacy and patient outcomes may emerge over time.
 
The Importance of Long-Term Data and Real-World Evidence
One of the key lessons from the study is the importance of long-term data and real-world evidence in evaluating drug efficacy. While surrogate endpoints provide valuable initial indicators, they may not capture the full spectrum of a drug's effects on patients' lives. Longitudinal studies and real-world evidence can offer insights into how drugs perform in diverse patient populations and under varying clinical conditions.
 
Incorporating real-world data into post-approval monitoring and evaluation processes can enhance our understanding of a drug's benefits and risks beyond the controlled settings of clinical trials. It also allows for continuous assessment of a drug's performance and may prompt adjustments in treatment guidelines or regulatory decisions based on evolving evidence.
 
Conclusion: Navigating Complexity and Uncertainty
The landscape of accelerated approval in cancer treatment is characterized by complexity and uncertainty. While this pathway offers expedited access to potentially life-saving therapies, it also poses challenges in terms of evaluating drug efficacy, ensuring patient safety, and maintaining ethical standards.

Studies like the one conducted by Brigham and Women's Hospital and Harvard Medical School provide valuable insights into the performance of drugs granted accelerated approval and the need for ongoing evaluation and transparency. Healthcare providers, regulatory agencies, and patients must work collaboratively to navigate these challenges, prioritize patient-centered outcomes, and uphold the highest standards of evidence-based medicine and ethical practice.
 
The study findings were published in the Journal of the American Medical Association.
https://jamanetwork.com/journals/jama/fullarticle/2817337

For the latest Pharma News, keep on logging to Thailand Medical News.