House Passes “Right to Try” bill – Compromising Public Health and Drug Development

On March 21, only one week after an initial defeat in the U.S. House, the controversial “Right to Try” bill was passed by a vote of 267-149. The legislation is now on its way to the U.S. Senate.

“Right to Try” would provide access to experimental therapies to patients with life-threatening illnesses while weakening FDA oversight and compromising public health and medical research. The FDA already offers patients access to experimental drugs or medical devices outside of clinical trials via the Expanded Access (sometimes called Compassionate Care) program and approves the overwhelming majority of all applications received—about 99%. Under Expanded Access, the FDA continues to supervise administration of the experimental drugs which both helps reduce individual patient risk and works to improve overall public health outcomes.

The current “Right to Try” bill permits patients and their doctors to bypass the FDA and work directly with pharmaceutical companies for access to drugs which have merely completed Phase I clinical trials. Some patient groups argue that by cutting out FDA oversight and creating an alternative avenue for accessing experimental drugs, Right to Try actually increases patient risks and is demonstrably less safe than Expanded Access.

Over 75 patient groups sent a letter to the House opposing passage of the bill, citing the dangers it presented to patients such as the seven-day lagtime between patient access to the investigational therapies and FDA notification of any possible side effects or negative outcomes. Additionally, the patient groups cited the removal of FDA-sanctioned dosing and safety measures. They also cited shortcomings of the bill such as its failure to address significant barriers to patients such as access and cost.

The bill strips patients of potential legal remedies by protecting doctors and drug companies from liability in the case of negative outcomes for patients.

The legislation is also poised to compromise medical research and drug development by preventing the FDA from using any data from negative clinical outcomes in its drug-approval assessments. Barring FDA from using such data would shroud the successes or failures of the experimental drugs in obscurity—possibly preventing further large-scale advances in overall research and development.

Ultimately, “Right to Try” strips the FDA of established regulatory authority and protections, increases risk to patients, and obfuscates data and outcomes vital to continued success in research and development–all of which could result in serious, wide-ranging public health issues.

We at Contracts Associates will continue to monitor this important issue.

Sidestep FDA Form 483—And The Resulting Setbacks

Bringing a new drug or medical device to market is incredibly exciting for any sponsor. Ideally, you’d like the clinical trial phase to proceed without delays, so you can get your new drug to market quickly—and start helping patients.

The FDA can issue a Form 483 after an inspection where an investigator has observed conditions or issues at a facility that might constitute violations of the Food Drug and Cosmetic Act and serves to alert the organization’s management. As a best practice, if issued a 483, companies or research centers should respond in writing without delay. The response should include a corrective action plan addressing the violations which the organization should immediately implement. Failure to respond or failing to take remedial measures could result a Warning Letter being issued by the FDA, or cause delays in studies and development.

The FDA maintains a list of Inspectional Observation Summaries broken down by program area (biologics, bioresearch monitoring, drugs, devices, etc.) which can be found on the FDA website. By viewing this data, sponsors, clinical sites, CROs and IRBs have the opportunity to learn the most common reasons for 483 and how to avoid similar pitfalls during the clinical trial process.

For example, in the program area of Bioresearch Monitoring, 248 Form 483s were issued between 10/1/2016 and 9/30/2017. Common reasons for the 483 include:

• An investigation was not conducted in accordance with the signed statement of investigator and/or investigational plan (frequency: 140 times)
• Failure to prepare or maintain adequate and/or accurate case histories with respect to observations and data pertinent to the investigation and/or informed consent (76 times)
• Informed consent was not properly documented in that the written informed consent used in the study was not approved by the IRB and/or was not signed by the subject or the subject’s legally authorized representative at the time of consent and/or was not dated by the subject or the subject’s legally authorized representative at the time of consent. (14 times)

Stick to the plan

An investigational plan forms a roadmap for any clinical trial, giving a brief overview of the type and scale of the study. Mandated by the FDA, it’s the outline that gets a trial from start to finish: quickly and safely. Sponsors and investigators must either stick to this plan, properly amend it—or risk receiving a Form 483.

An investigational plan is detailed and thorough, but a sponsor won’t typically be on site to ensure it’s being followed. As such, it’s important to know that your contracts and terms with your CROs are solid and equitable. Failing to review contracts could leave you open to both liability and FDA delays. Our attorneys possess the necessary industry experience and a meticulous, efficient approach to contract reviews which means sponsors can be confident that they’re protected should an investigational plan go awry.

Let the record show everything

To gain the best results from a trial and to pass inspections, meticulous record keeping is a must. Much like an investigational plan, proper record keeping requires diligence and thoroughness. It involves preparing and maintaining case histories and taking detailed documentation at every stage, from observation to delivery. Failing to properly monitor and maintain records for a trial isn’t just a common reason for a Form 483, but can also compromise the study.

Contracts Associates will ensure that each sponsor/CRO agreement includes detailed language about monitoring and record keeping. The agreement is intended to protect the sponsor in the event your CRO fails to keep proper records. Working with us will allow sponsors to harness our knowledge and expertise, which means you won’t have to worry about this type of exposure.

Obtain informed consent

The FDA doesn’t just monitor the safety of publicly available drugs or devices. It also monitors the safety of clinical trial participants, and obtaining informed consent is a core element of that.

Sponsors must ensure trial participants know their rights in relation to a clinical trial they’re considering. This includes exposure to unknown risks, possible side effects, and potential outcomes. Ensuring that your study is compliant with FDA regulations about informed consent is crucial to keeping your company protected and ensuring your milestones are met.

Your in-house counsel might not have the time or manpower to carefully review a trial’s informed consent forms before signing off on them. The attorneys at Contracts Associates can provide the additional assistance you need, on an as-needed basis. We can help you stay on schedule and ensure compliance with informed consent requirements. There’s no better plan than to have informed consent contracts reviewed quickly and thoroughly by experienced industry professionals like us.

Implications of Form 483

It’s important to keep in mind that Form 483 does not constitute the final Agency determination of whether cited conditions are absolute violations of the FD&C Act. Form 483 is a factor for consideration, along with all evidence or documentation collected on-site, and any responses made by the company. Only then does the FDA make a final determination as to what future actions might be necessary.

Proactive approaches with Contracts Associates

At Contracts Associates, we work with sponsors to ensure their clinical trials don’t fall behind schedule or expose the company to undue risk. We can even produce and maintain a personalized “risk register” for you, complete with contract terms and other matters that we become aware of in our reviews—helping you avoid future problems.

Our extensive industry experience means we don’t require training—or the time, cost, and effort associated with a learning curve. We provide quick and meticulous turn-arounds, and can help you meet your milestones. Contact us today to find out how we can help you.