Regulatory Focus™ > News Articles > New Legislation Seeks Creation of Provisional Approval Pathway for New Drugs

New Legislation Seeks Creation of Provisional Approval Pathway for New Drugs

Posted 23 May 2013 | By Alexander Gaffney, RAC

Three US legislators have moved to introduce legislation they say would accelerate US Food and Drug Administration (FDA) approvals while giving patients the option to obtain therapies outside of clinical trials.


US regulators now have a variety of tools at their disposal with which to accelerate the approval of pharmaceutical products. The most prominent of those tools are geared toward patients with serious diseases, and include fast track designation, accelerated approval, priority review and breakthrough product designation, among others. The programs are intended to allow the agency to give additional review resources to an application, accelerate the review schedule, and allow for products to be approved based on surrogate or incomplete data.

The pathways have been used to great success in recent years, and many of FDA's approvals, and in particular its oncology drugs, have benefited from those tools.

In addition, FDA also has a variety of tools at its disposal with which to work around clinical trial protocols, allowing patients and doctors to petition for "compassionate use exemptions" and, in some cases, purchase the drugs as well. FDA recently released two guidance documents seeking to clarify the processes for both, noting that companies can charge an individual for access to a drug, but doing so is entirely their choice.

Focus: FDA Draft Guidance: When is it Acceptable to Charge for an Investigational Drug?

Focus: New FDA Draft Guidance Aims to Clarify Compassionate Use Process

The larger problem, it noted, is that by allowing some patients-typically those who would not otherwise live long enough to benefit from the drug and are too sick to participate in the trial-to opt out of trials, the trial itself can become delayed, preventing the population of patients from accessing the drug as quickly.

A New Bill

A statement from the co-sponsors of the bill-Reps. Morgan Griffith (R-VA), Scott Peters (D-CA) and Michael McCaul (R-TX)-however, indicate that as quickly as FDA might be moving, it's not quick enough for the estimated 500,000 patients who die each year from cancer. "For diseases like melanoma, Lou Gehrig's disease, and Parkinson's disease, either very few drugs are available or those in the pipeline cannot make it through FDA's delays and regulations," they said.

The solution, they said, lies in their new bill, the Patient Choice Act of 2013, which allows patients to purchase access to experimental treatment options, giving them an additional chance at a life-saving therapy.

"There are research facilities in San Diego developing potentially life-saving medicines and therapies, which have been proven safe after rigorous testing. Unfortunately, because of the lengthy FDA approval process, patients with the most urgent need aren't able to access them," Peters wrote in a statement. "We should be doing more to promote research and innovation here in the United States, and this bill is a way to incentivize researchers to stay here while getting patients remedies that they need."

A Provisional Pathway

The ability to purchase drugs isn't exactly new, but other parts of the bill call for a radical reimagining of the current approval system. For example, the legislation calls for a new "provisional approval" pathway for "adequately safe fast track products" that would permit any drug given FDA's fast track designation to be considered "adequately safe," and thus given provisional approval, allowing it to be marketed.

Provisional approval would come with several restrictions, including requiring informed consent by patients and the "continued pursuit of safety and efficacy data" for purposes of obtaining full approval. The status would not automatically be granted by FDA, but would rather need to be explicitly requested by a company, perhaps alleviating industry's fears that the pathway would distract from their attempts to obtain full approval by siphoning off patients from clinical trials.

However, the drug would be permitted to market itself so long as copies of promotional materials were submitted to FDA at least 30 days in advance-a major departure from current rules that only permit marketing to be done for drugs that have obtained full approval. FDA would, however, be permitted to impose Risk Evaluation and Mitigation Strategies (REMS), including elements to ensure safe use (ETASU) on the drug products.

Terms and Conditions Apply

The bill defines "adequately safe" as a drug product for which use of the drug is unlikely to create greater harms than the effects of the disease's likely death or morbidities. In the case of a new molecular or chemical entity, this would rely heavily on the company's own data, but the legislation says FDA would be unable to impose any new or different requirements for Phase 1 or Phase 2 clinical study data.

However, there is another provision that could act as a workaround: If a drug has been approved and marketed in a country or region (defined under 802(b)(1)(A)) for a period of four years, that drug will have been deemed to be adequately safe. Key among the regions on that list: Europe, legislators said.

The drug's provisional approval would terminate as of the date of the drug's full approval or five years after it was first granted, whichever comes first, according to the legislation. Approval extensions may be sought. Orphan drugs would not be subject to the same five-year limitation. FDA would also be permitted to withdraw the drug in the event that it is shown to not be safe.

An important caveat for sponsors, however: Any marketing exclusivity granted by FDA approval would be in addition to the time spent to obtain provisional approval, meaning that sponsors would not be penalized for participating in the provisional approval pathway.

Griffith, Peters, McCaul Introduce the Patient Choice Act 

H.R.2090 - To amend chapter V of the Federal Food, Drug, and Cosmetic Act to permit provisional approval of fast track products.

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