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How research evolves and brings new hope to patients

Stephen J. Ubl   |     June 11, 2021   |   SHARE THIS

Before medicines can be made available to patients, they often begin as ideas that are based on newly discovered molecules and cells, strange phenomena or little-understood processes in the body. The biopharmaceutical industry’s unique role in the research ecosystem is to utilize its scientific, medical and industrial expertise to take the necessary risks to build on and translate basic scientific research into safe and effective medicines that can be made available to patients. This long and complex process is regularly fraught with more setbacks than successes as less than 12% of medicines entering phase one clinical trials ever end up as U.S Food and Drug Administration (FDA) approved medicine. Setbacks in research are natural for all researchers who take each failure and use them to inform future success, and Alzheimer’s disease is no exception. 

Since 1998, there have been 198 unsuccessful potential medicines in clinical trials for Alzheimer’s. In that same time frame, only four new medicines were approved by the FDA. Despite years of disappointments, the first treatment for Alzheimer’s disease directed at underlying disease mechanisms was approved this week, providing not just hope for patients but also for the researchers committed to solving the challenge of this devastating disease. 

As researchers believe that no single medicine will be able to defeat Alzheimer’s disease, but rather several medicines aimed at multiple targets will likely be needed to combat it, we can expect continued commitment from researchers to bring new treatments to patients. The diverse targets explored across the pipeline provide hope that a successful approach to treatment can be achieved.

And while the initial FDA approval of a medicine is a tremendous milestone, the research doesn’t stop there. Once a medicine is approved, additional knowledge is gained through ongoing research and the accumulation of data from real-world use. Indeed, additional research may even be required, such as in the case of an accelerated approval to verify the clinical benefit in confirmatory trials. Over time, additional research can verify clinical benefit of a medicine or may show even greater efficacy than in the pivotal clinical trials and lead to an improved understanding of how the medicine delivers benefit to patients or works with other therapies. In fact, many diseases successfully treated today with multiple drug classes and treatment approaches, such as HIV and many forms of cancer, did not begin with one breakthrough. But rather, progress came iteratively and evolved over time. 

For example, AZT was the first treatment approved to combat HIV, the virus that causes AIDS. But it took the development and approval of various antiretroviral therapies from multiple drug classes over many years, each working by blocking the virus at specific points of the virus’ lifecycle, that ultimately made highly active antiretroviral treatment (HAART) the effective treatment it is known as today. With appropriate treatment, HIV is now regarded as a chronic and manageable condition and patients can expect to live close to normal life spans.

While Alzheimer’s is one of the most complex diseases to research, I’m optimistic that continued research and development will propel even more advancements against it. 

In addition to the treatment approved this week, biopharmaceutical researchers are currently researching more than 80 potential medicines to attack Alzheimer’s disease in diverse, innovative ways, that researchers hope will halt or prevent it. Approximately 80% of these medicines have the potential to be disease modifying treatments. 

The need to continue addressing Alzheimer’s disease is high with one study finding that after heart disease and cancer, Alzheimer’s is the third leading cause of death for seniors, responsible for one out of every three deaths. In the absence of new disease modifying treatments, Alzheimer’s disease is expected to grow to nearly 13 million people and cost the nation more than $1.1 trillion by 2050.

We should be doing everything we can to encourage continued innovation for diseases like Alzheimer’s, dementias and other diseases with significant unmet medical need. New treatments not only provide patients hope but the opportunity to reduce tremendous burden to the health care system.

Stephen J. Ubl

Stephen J. Ubl Stephen J. Ubl is president and chief executive officer of PhRMA. Mr. Ubl leads PhRMA’s work preserving and strengthening a health care and economic environment that encourages medical innovation, new drug discovery and access to life-saving medicines. Ubl is recognized around the world as a leading health care advocate and policy expert who collaborates successfully with diverse stakeholder groups – including patient and physician groups, regulators, public and private payers, and global trade organizations – to help ensure timely patient access to innovative treatments and cures.

Topics: Research and Development, Alzheimer's

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