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# Law Professor Argues for Conditional Drug Approvals
- URL: https://www.fdaweb.com/law-professor-argues-for-conditional-drug-approvals/
- Published: 2017-03-21T12:00:00.000Z
- Updated: 2026-09-14T22:12:52.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5138258

A *Health Affairs* online post by California Western School of Law professor **Robert Bohrer** calls for a conditional drug approval system that would accelerate access to new drugs while restraining their price until full approval is granted. Bohrer [writes](http://healthaffairs.org/blog/2017/03/20/a-better-balance-between-accelerated-access-and-high-priced-new-drugs-a-new-conditional-approval-option/?ref=fdaweb.com) that such an approach would address two major issues roiling the pharmaceutical industry — opposition to the high prices of drugs and the need to accelerate access to drugs for seriously ill patients.

He notes that accelerated access provisions rely on clinical trials that measure surrogate endpoints that frequently fail to predict whether patients will live longer or have a better quality of life. They are used, he says, because they can be measured in a relatively short time, whereas it can take years to collect evidence on a drug’s real effectiveness.

Likewise, Bohrer writes, any reasonable approach to drug pricing requires substantial knowledge of a drug’s effectiveness for its value to be considered when evaluating the drug’s price, and that knowledge is not available when a drug is approved before its performance is known on truly meaningful endpoints such as overall survival (in cancer) or long-term ability to function (in diseases such as Parkinson’s or muscular dystrophy).

Bohrer uses the recent FDA accelerated approval of eteplirsen (Sareptas Therapeutics’ Exdonys 51) for Duchenne Muscular Dystrophy (DMD) to discuss issues with that process. He notes the pressure that was on the agency to approve the drug since there currently are no effective treatments, and says that while it is now on the market for $300,000 per patient per year, it may be years before data from additional clinical trials can provide substantial evidence of whether it is effective.

“If the drug turns out not to provide meaningful clinical benefit,” he writes, “then the $300,000 per year cost of providing patients the drug is a terrible waste of our healthcare dollars. However, if insurers do not pay for the drug and it actually would provide significant therapeutic benefit to Duchenne’s patients, then there would be even more terrible unnecessary suffering and death among DMD patients.”

He suggests a revised use of the 1992 parallel track initiative that FDA used to respond to the AIDS crisis, involving an accelerated approval that permits wide distribution before final approval and a mechanism that limits the price of the conditionally-approved drug while more data are collected.

Bohrer says that under conditional approval, any doctor treating a patient with the targeted indication could prescribe the drug and would agree to collect and report basic data on the duration of treatment, responses to the drug, and any other changes in their patients’ conditions.

A possible mechanism for the discounted price would be to require a drug company to specify its intended initial market price for the new drug and then limit the price to 25% of the initial market price during the conditional approval period. Or, Bohrer says, the conditional approval price formula might be a predetermined percentage of the average introductory price of breakthrough drugs approved during the previous two years. He suggests that the 25% formula should cover the costs of manufacture, distribution, a limited marketing outreach (little marketing needed due to strong patient demand for a potentially life-saving drug for which there is no effective alternative), and the process of data collection, and still provide a modest profit.

It would be necessary, the post says, to require insurers and government payers to cover the drugs during the conditional approval period in the same way they currently cover drugs approved under the accelerated access and breakthrough drug procedures.

“If the data confirm the benefit of the treatment, full approval would be granted and the drug sponsor could charge whatever price it can justify in the marketplace,” Bohrer concludes, “but with much better evidence as to what the drug’s real worth actually is. Patients desperate for treatment would get access to drugs, insurers would be paying less than under the current system, and patients, providers, and insurers would get the data they need on the drug’s efficacy.”