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FDA Regulations Reform? Risks and Benefit for the Approval Process of New Drugs

Posted on June 2nd, 2017 by in Pharma R&D

Know the rules

Since 1906, the US Food and Drug Administration (FDA) through regulations has been responsible for ensuring the approval of new drugs whose health benefits outweigh the known risks associated with the drug.

The safety and efficacy of new drugs are examined to protect the patients from dangerous therapeutics by conducting early and late-stage clinical trials aimed at investigating the tolerability and side effects of the new drug. The protection of the public health is the responsibility of the FDA through the approval process. Post-approval, pharmaceutical companies have obligations to conduct Phase IV clinical trials and ensure that the drug is safe, pharmacovigilance data is kept current and that the regulatory authorities are informed of any serious adverse events reported in patients/public.

The real question is – what would be the risks and benefits associated with changing regulations? Under the new administration, can the newly appointed FDA commissioner look at doing  an overhaul of the current “red tape” and totally reform the lengthy and unwarranted regulations which cause delays in approving novel drugs needed for unmet medical needs? The advantage will be that relaxing the regulations will expedite the approval process, so the drugs get to the market faster. However, reducing the existing standards for drug approval may allow more drugs which are not as efficacious or safe to get to the market, a responsibility that the FDA has maintained over the years through stringent requirements.

In order to control the excessive cost of bringing a new drug to the market and decrease the cost of research and development, the new administration has proposed streamlining regulations for the new drug application (NDA) process. If the investigational studies are affected with the streamlining process, the standards for the stringent clinical trials may be compromised, putting the patient at risk with sub-optimal drugs. Safety of the patient is paramount, and deregulation of an established process has to be carefully evaluated to assess the cost-benefit ratio.

Fundamentally, one has to ask the question, “Is this drug going to be ‘first in class’ or ‘best in class’? “ One has to compare the drug to the currently available lines of drug therapy and derive ‘incremental benefits’ that the new drug would offer. If the innovation can really ensure safety and shows better efficacy, then it does mandate the paradigm shift in making the drug approval process faster, better and cheaper. This would hopefully also bring down the costs of drug development and thereby ultimately allow better prices for the payers and consumers. With the ‘health care reform’ initiative that the new administration is working on, the FDA streamlining could become a part of the process to help the reform overall.

However, if the drug gets to the market faster with possible costs reduction and the safety of the new drug has not been fully established with careful assessment of adverse drug reactions, an indirect cost will be incurred due to administration of drugs which should not have been marketed. With the need for more innovative medicines and cures for diseases such as Alzheimer’s, diabetes, rare diseases and oncology, studies looking at the long-term effects of drugs are necessary to understand the risk-benefit ratio of the patient being on a drug for an extended period of time. Although clinical trials do not identify every harmful effect of a new drug, studies are designed to give physicians and patients information on the drug to lead to an informed treatment regimen. Streamlining the regulatory process may save the pharmaceutical companies some time, resources and costs; however, it may negatively impact patient safety.

One also needs to look at this issue globally. Many of the international drug regulatory authorities tend to follow the US FDA guidance. Also there are ICH guidelines in place that may need to be looked at at various angles. With BREXIT, one also needs to look at EU and non-EU regulatory authorities and their new stance. European Medicines Agency is quite eager to follow what the US FDA may do in terms of ‘reforms’. Japanese regulatory authorities (MOHW) are also going through some fundamental changes in their organizational structure.

With the focus on ‘health care reform’ and affordability, one needs to look at not only reforming the US FDA regulations, but also rationally changing some fundamental concepts embedded in CMS, Federal Trade Commission, NIH & NIDCD, Center for disease control. It’s going to be an ongoing work-in-progress for the coming months, I do believe, to see any new ‘reforms’ or ‘streamlining of regulations’ take place through the Congress and the Senate. President’s Council on Science & Technology is also being tasked with addressing some of these potential changes, which can bring about rational reform that would optimize the current red tape processes and make it acceptable to the patients and consumers, who really are the biggest stakeholders.

I do predict that these reforms are bound to take place sooner or later, assuming there is a win-win scenario created for the pharmaceutical companies, payers and the patients.

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All opinions shared in this post are the author’s own.

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