The Prescription Drug User Fee Act must refrain from adding Right To Try provisions

Last month, the HELP Committee advanced the Prescription Drug User Fee Act (PDUFA) Reauthorization Bill to the Senate floor – legislation that provides critical funding to the new drug approval process. HELP Chairman Lamar Alexander (R-Tenn.) and ranking member Patty Murray (D-Wash.) pledged a “bipartisan and non-controversial” user fee bill in light of the importance of passing this bill.

Nevertheless, it is our understanding that a set of highly controversial measures, known as “Right to Try,” which pose substantial risk to patient health and are completely unrelated to FDA funding, are being considered for attachment as amendments to PDUFA.

{mosads}As rare disease patient advocates, we urge Congress to do what is best for patients in advancing the user-fee bills smoothly and cleanly.

To date, 37 states have signed Right to Try bills into law. Each of the laws mimic the model legislation crafted by libertarian think tank, The Goldwater Institute. Although the bills are promoted as providing a right to direct access to experimental medication, they all fail to achieve this one goal. To date, there has not been a single substantiated instance of access granted solely due to a Right to Try law, despite laws being on the books since the spring of 2014.

At its base, “Right to Try” is an attempt to completely cut out the FDA from oversight of access to experimental medications early in development. Right to Try proponents insist that, by loosening regulations on the drug approval and clinical trial process, they will incentivize drug makers to provide their drug.

However, FDA deregulation will hurt not only patients, but the integrity of drug approval and safety in this country. The FDA drug approval processes, which include clinical trials to measure everything from dosage, to efficacy, to safety, aren’t in place to “punish” drug makers, but to protect patients. Critics have repeatedly denounced Right to Try as a sham, which is both bad policy and bad for patients.

Procedurally, the process by which the Right to Try amendments may appear in PDUFA are as equally troubling as the substance itself. State-level Right to Try has been critiqued, battled, and published against, since April 2014 when the movement gained traction. Yet, there’s been virtually no public discourse or understanding of the more harmful provisions of federal Right to Try bills. The secrecy by which amendments, including Right to Try and the recently announced Hatch Amendment, are quietly being attached to PDUFA will not benefit patients’ access to treatments. Public discourse is vital to creating tangible solutions.

Because of the complete failure of state-level Right to Try, some have tried to push the federal government to enact even more alarming laws that might enable some portions of state Right to Try bills. The federal bill contains provisions that remove all rights from dying, vulnerable patients to bring a cause of action against any entity in their chain of care – from sponsors to manufacturers to prescribers.

Of potentially greater concern, the bill explicitly restrains every branch of the federal government from taking any action whatsoever that could be construed as “interfering” with early access – which would prohibit law enforcement and prosecutors from impeding intentional acts of wrong-doing against patients, the judiciary from hearing claims of gross negligence, and other branches of the government from ensuring that patients aren’t being taken advantage of for-profit, or being exposed to medications that meet basic standards of manufacturing.

Therefore, the federal Right to Try amendment being considered for attachment to the wholly-unrelated PDUFA legislation encourages the worst hypothetical scenarios: for-profit sales, of unproven and potentially harmful medical interventions for dying patients, with no oversight of any independent entity – including the FDA ­– and no ability for any branch of the federal government to take legal action to prevent, deter or penalize wrongdoing. 

As patient advocates, we are not alone in our stance against Right to Try. The National Organization for Rare Disorders, Parent Project Muscular Dystrophy, and more have rallied together to urge the FDA to improve their expanded access program, as the best means to help patients access unapproved therapies.

A range of other medical experts, including NYU School of Medicine Working Group on Compassionate Use and Pre-Approval Access, and national organizations such as the American Medical Women’s Association, reiterate that not only is Right to Try an empty gesture, but it detracts from real ways to help patients.

Similarly, the Association of Clinical Research Organizations (ACRO), which is committed to ensuring patients are able to access treatment, released a policy statement opposing Right to Try as “deeply flawed” legislation, insufficient to protect patients from wrongdoing; undermining to the essential role of the FDA to assess the safety and effectiveness of medical interventions; and likely to compromise the entire clinical trials process that leads to development of treatments and cures to diseases.

The American Society of Clinical Oncology (ASCO), an organization which supports many terminally ill patients – the exact patients Right to Try purports to represent – likewise denounces Right to Try as “ineffective and potentially harmful to patients.” 

As advocates for patients suffering from diseases which are often rare and life-threatening, we understand the need to expand terminally ill patients’ access to investigational treatments. However, the integrity of clinical trials must be preserved so that all who might benefit from a drug know that it works and is safe. Last month, the HELP Committee announced an amendment to the user fee bill which proposes to do just that, the Enhanced Clinical Trial Design Act of 2017, co-sponsored by Senator Orrin Hatch (R-Utah).

The ‘Hatch Amendment’ seeks to address many of the concerns held by Right to Try supporters. Amongst other things, the Amendment calls for the FDA and National Institutes of Health to hold a public meeting to discuss barriers to clinical trials; a GAO report on the FDA’s expanded access program; streamlining IRB reviews of expanded access protocols; and allow drug companies to make expanded access policies available for products in the breakthrough therapy, fast track, or regenerative advanced therapy pathways.

While Hatch is a small step in the right direction, it is far from perfect. Its hefty requirements will affect every single clinical trial going forward. The Hatch Amendment was introduced as a stand-alone bill to attach to PDUFA last month; there has been no time to debate or analyze it. The Hatch Amendment has the potential to significantly damage the drug development process through burdens on industry and clinical trials through overregulation. Congress, together with the FDA, the NIH, and patient advocacy groups, should continue to explore solutions to increasing clinical trial access that does not negatively impact drug development.

We must improve expanded access to investigational treatments by strengthening the FDA’s existing expanded access program, rather than by tagging on ill-advised, incomplete amendments to must-pass bills. Patients deserve more –– they need more ­­–– and we hope those reauthorizing PDUFA agree.

Alexandra Hall, MA, is the Managing Director of Policy, Patient Advocacy and Industry Liaison at The Isaac Foundation. Andrew McFadyen is the Executive Director of The Isaac Foundation and a member of the NYU School of Medicine’s Working Group on Compassionate Use and Pre-Approval Access. Beth E. Roxland, J.D., M.Bioethics, is a Senior Consultant on Law, Policy, and Ethics, and an Associate of the NYU Langone School of Medicine’s Division of Medical Ethics.

The views expressed by this author are their own and are not the views of The Hill.