How a controversial US drug policy could be harming cancer patients worldwide

Accelerated Approvals and the Impact on Global Healthcare

In August 2021, Amol Akhade, an oncologist at Nair Medical Hospital in Mumbai, India, received an e-mail from the Swiss drug manufacturer Roche recommending the use of a drug named atezolizumab to treat a specific kind of breast cancer. Akhade was surprised. That month, Roche had withdrawn the drug for this purpose in the United States (although it is still approved to treat other kinds of cancer).

The Issue with Accelerated Approvals

Accelerated approvals in the United States are granted on the basis of clinical studies that suggest a health benefit without necessarily demonstrating it fully. The process prioritizes speed over certainty, and it requires companies to complete follow-up studies to confirm a treatment’s benefits. However, there are complications that have made the program controversial. Companies don’t always conduct studies in a timely manner, and the FDA hasn’t always been great about enforcing them. Researchers have noted that the median time to withdrawal for a drug that fails to hold up in confirmatory studies is four years; sometimes it takes decades. And some high-profile accelerated approvals have raised eyebrows.

Global Impact of Accelerated Approvals

The effects of accelerated approval can spread beyond US borders. Regulatory agencies in low- and middle-income countries sometimes rely on decisions made by the FDA and EMA. Some lack their own regulatory framework and so follow the FDA’s decisions directly, under the guidance of the World Health Organization. For India, which has its own regulatory body, the Central Drugs Standard Control Organisation (CDSCO), and others, the position that the FDA takes on a drug can be very influential.

Communication Gaps and the Role of Guidelines

At the heart of the problem is a failure of communication — both in the outcomes of follow-up studies and the nature of accelerated approval itself. Clinicians and patients often view FDA approval as an on–off switch, either a drug is approved or it’s not. The distinction between surrogate endpoints and more direct ones isn’t always obvious, even to clinicians. Guidelines offered to clinicians by the NCCN can strengthen those habits — not only because clinicians rely on them for treatment plans, but also because this guidance determines federal health-care reimbursement.

Reforms and Looking Ahead

Reforms are possible — at least in the United States. In late 2022, the US government enacted the Food and Drug Omnibus Reform Act (FDORA), which aims to make the accelerated-approval process more transparent and gives the FDA greater authority to ensure that drug makers complete confirmatory studies. The FDA now requires companies to begin a clinical trial to gather the data needed for a conventional approval before they apply for accelerated approval. It has also begun re-evaluating previous accelerated approvals, a process that has led to high-profile withdrawals. Other safeguards give the FDA more authority to ensure that companies withdraw drugs when required to do so.

Conclusion

Accelerated approvals have significant implications for global healthcare. The need for clear communication, improved guidelines, and international collaboration is crucial to ensure that patients receive safe and effective treatments. As efforts continue to address these issues, it is important to prioritize the Sustainable Development Goals (SDGs) to promote equitable access to healthcare and improve patient outcomes worldwide.

SDGs, Targets, and Indicators

1. Which SDGs are addressed or connected to the issues highlighted in the article?

• SDG 3: Good Health and Well-being
• SDG 10: Reduced Inequalities
• SDG 17: Partnerships for the Goals

2. What specific targets under those SDGs can be identified based on the article’s content?

• SDG 3.8: Achieve universal health coverage, including financial risk protection, access to quality essential healthcare services, and access to safe, effective, quality, and affordable essential medicines and vaccines.
• SDG 10.2: By 2030, empower and promote the social, economic, and political inclusion of all, irrespective of age, sex, disability, race, ethnicity, origin, religion, or economic or other status.
• SDG 17.16: Enhance the global partnership for sustainable development, complemented by multi-stakeholder partnerships that mobilize and share knowledge, expertise, technology, and financial resources.

3. Are there any indicators mentioned or implied in the article that can be used to measure progress towards the identified targets?

• Indicator for SDG 3.8: Proportion of the population with access to affordable essential medicines and vaccines on a sustainable basis.
• Indicator for SDG 10.2: Proportion of the population reporting having felt discriminated against or harassed in the previous 12 months based on a ground of discrimination prohibited under international human rights law.
• Indicator for SDG 17.16: Total amount of financial and technical assistance provided to developing countries to support the implementation of sustainable development plans and programs.

Table: SDGs, Targets, and Indicators

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Source: nature.com

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