‘Right to try’ preserves patient freedom, puts regulators on right path

On March 21, the House of Representatives passed its “Right to Try” bill H.R. 5247 by a 267-149 margin, with 35 Democrats joining Republicans in voting for the legislation.

The bill moves to the Senate, which unanimously passed a similar bill on August 3 of last year, but where its current path into law is uncertain. The Senate should pass the House Right to Try legislation.

Designated the ‘‘Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act,” H.R. 5247 gives informed, terminally ill patients the freedom to take, and their physicians the freedom to prescribe, promising medicines that have completed initial safety testing but have not been approved by the Food and Drug Administration.

Thirty-eight states have passed Right to Try laws, but federal law supremacy probably renders these merely symbolic. A federal Right to Try law is unlikely to have much direct impact for different reasons.

First, experimental medicines are a rational choice for relatively few patients. Second, pharmaceutical manufacturers, which cannot profit from the sale of unapproved drugs, have little incentive to provide them to patients and many reasons not to do so.

Neither the Senate nor the very similar House proposal force companies to provide early access to medicines or require insurers to pay for them. This is appropriate.

Although proposed Right to Try legislation prohibits FDA from using clinical outcomes to interfere with the approval process, this protection is not absolute.

Once FDA officials are aware that untoward events have been associated with a medication, even if they ultimately prove unrelated to its use, such knowledge could consciously or unconsciously influence their review of it.

Pharmaceutical companies that provide unapproved drugs could also be subject to lawsuits by patients who claim to be injured by them. The law would eliminate many, but not all, liability suits. Consequently, Right to Try legislation is unlikely to directly impact the numbers of patients who have access to investigational therapies.

Why then will a Right to Try law be helpful? For one thing, it will highlight the seriousness of the issue among patients, physicians, drug companies and government officials. However, the most important, lasting effects of a Right to Try law will assuredly be on the FDA.

The FDA has an expanded access program. However, the agency’s process has been criticized as slow and unduly burdensome. In the past, medical doctors were required to file full investigational new drug applications (INDs), as if they were sponsors undertaking clinical trials.

According to the FDA, completion of the paperwork alone consumed 100 hours! Few if any physicians had that kind of time to devote to paperwork, no matter how compelling the therapy or how much the doctor cared about her or his patients.

The FDA historically has authorized over 99 percent of expanded access requests, but that figure arguably misrepresents the actual state of affairs. Rather, the agency’s paperwork demands and its preliminary discussions with potential applicants discouraged many from applying.

The small number of applications, and anecdotes about patients traveling to other countries to gain access to unapproved medicines, support this contention.

The FDA has recently taken steps to rationalize its process. Prescribing physicians can now submit a “single patient IND” using Form 3926, which it claims takes as little as 45 minutes to complete. Moreover, the FDA reportedly reviews most requests within 24 hours. These steps are laudable.

However, the FDA first issued regulations that provided for access to experimental medications in 1987. Without the efforts of the Right to Try movement, would these basic, common-sense improvements, which took over 30 years to implement, have occurred?

The enactment of a federal Right to Try law will place perpetual pressure on the FDA to continually review and update its program, ensuring that it meets contemporary needs.

All medicines can induce harm. This danger is less well understood with investigational drugs. In addition, treatment with experimental drugs may have unforeseen impacts on health insurance. For example, an adverse event could potentially leave a patient saddled with large, unpaid medical bills.

Nevertheless, novel genetically driven and immuno-oncology therapies produce dramatic responses in some patients. In our new world of precision medicine, patients may have a greater chance of benefiting from investigational therapies than in the past.

If a dying person’s doctors believe that she or he may benefit from an investigational medicine; if the patient chooses to assume the risks of the treatment and is willing to pay for it; if a manufacturer is willing to provide the drug, should the federal government deny the individual his or her last hope?

Right to Try laws preserve patient autonomy and freedom. They also encourage regulators to act in the best interests of patients.

Dr. Roger D. Klein is the principal at Klein and Klein Co., L.P.A. and Roger D. Klein, MD JD Consulting. He is an expert for the Transparency Project and former adviser to the FDA. Dr. Klein graduated from Yale with both an MD and JD.