State “Right-to-Try” Legislation, While Well-Intentioned, Is Unlikely to Achieve Goal of Bringing Safe and Effective New Medicines to Patients

State “Right-to-Try” Legislation, While Well-Intentioned, Is Unlikely to Achieve Goal of Bringing Safe and Effective New Medicines to Patients

06.14.14 | By Sascha Haverfield

The development of innovative, safe, and effective medicines for serious or life-threatening diseases represents an urgent and unique challenge that requires special attention. The biopharmaceutical industry is committed to meet this challenge by working with all stakeholders to ensure that there are appropriate and targeted regulatory approaches to accelerate the development and availability of new medicines for the patients who need them.

Successful completion of the clinical trial process is required to demonstrate to the Food and Drug Administration (FDA) that an investigational drug is safe and effective so that it can be approved and made available to a broad patient population. Clinical trials are the primary route by which patients can participate in the drug development process, receive access to unapproved investigational drugs, and contribute to the collection of safety and efficacy data necessary for FDA approval.

For patients with a serious or life-threatening disease who are ineligible or unable to participate in a clinical trial, use of an unapproved investigational drug through an expanded access program may be an option. The current FDA process for a patient to gain access to an investigational drug through expanded access was established in 2009 in close consultation with patients, physicians and the biopharmaceutical industry. Expanded access programs are part of many biopharmaceutical companies’ commitment to patients.

We have recently seen the introduction of so-called “right-to-try” legislation in a number of states with the intent of providing terminally ill patients access to unapproved investigational drugs after only basic safety testing. While these bills may be well-intentioned, they seek to bypass FDA oversight and the clinical trial process, which is not in the best interest of patients and public health, and is unlikely to achieve our shared goal of bringing innovative, safe, and effective medicines to patients as quickly as possible.

Because of FDA’s critical role in ensuring the safety and effectiveness of prescription drugs, and the Agency’s ultimate oversight of clinical trials and new drug approvals, state-by-state “right-to-try” legislation is unlikely to help optimize the existing federal expanded access process.

Instead, patients deserve earlier access to approved medicines that have satisfied FDA’s standards for safety and effectiveness. All stakeholders – patients, physicians, biopharmaceutical companies, academia, and FDA - must come together to identify ways to modernize the clinical trial, drug development and FDA review processes by harnessing 21st Century science to accelerate the availability of new medicines for the patients who need them.

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