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New internal policy says the US agency cannot force a drug maker to supply an unapproved drug to a patient for expanded access and that the provider must seek company approval to provide the product.





FDA makes clear in its new internal policy on expanded access that while it will speed approval of applications, it cannot mandate a company to provide an investigational drug for a patient and will not even facilitate those discussions.



It serves as another reminder that FDA's role in the expanded access process is limited even as efforts to expand right-to-try programs continue.



Indeed, while FDA is often drawn into the spotlight on this issue, some of the most high-profile expanded access cases have arisen from drug sponsors denying access to a patient.



The new Manual of Policies and Procedures document posted Aug 17 outlines how Center for Drug Evaluation and Research staff should handle individual expanded access requests for emergency use INDs.



Expanded access is intended to allow desperately sick patients to receive drugs that are unapproved if it is thought the products may help treat or diagnose their condition.



FDA often approves INDs to allow access where appropriate. But the agency made clear in the MaPP that it "cannot compel a pharmaceutical company to provide access, including emergency access, to its investigational drug for treatment use."



"When a company provides access to its investigational drug for treatment use, it is doing so voluntarily," FDA said in the MaPP.



The agency also wrote in the MaPP that the company developing the drug must agree to provide the product to the treating physician before an IND can be approved.



Among the duties of CDER staff is to "inform the physician that the pharmaceutical company will need to agree to provide the investigational drug, if the company has not already done so."



FDA also said it may not be able to disclose the company studying an investigational drug to a provider looking for expanded access because the information is confidential.



"It is the responsibility of the [emergency IND] sponsor to identify the entity developing the investigational drug and to request and obtain that entity’s permission to access the investigational drug to treat his or her patient before contacting FDA to request an EIND," FDA said in the MaPP.



The 2014 case of Chimerix Inc. refusing to give its antiviral brincidofovir to a critically ill child generated social media outrage and heavy mainstream media attention. Eventually a protocol agreement allowed access for the child and 19 others. (Also see "Chimerix And FDA’s Compassionate-Use Solution" - Pink Sheet, 26 Mar, 2014.)



Johnson & Johnson developed a compassionate use review committee lead by the New York University School of Medicine intended to judge requests fairly and equitably. (Also see "J&J's Compassionate Use Advisory Cmte. May Become Industry Model" - Pink Sheet, 9 May, 2016.)



FDA has approved nearly all the expanded access applications it receives. But it has been targeted as among the problems for patients and providers to gain expanded access to investigational drugs.



The agency in 2015 condensed the length of the forms required for approval to simplify the process after criticism that they took too long to complete. (Also see "FDA Makes Expanded Access Applications Easier As Industry Lawyers Urge Caution" - Pink Sheet, 4 Feb, 2015.)



Policy Limits Paperwork In Emergency Situations

The MaPP is intended in part to explain the duties of various CDER staff in pushing an emergency IND through its review process quickly.



In emergency situations, FDA will allow the provider opening the emergency IND to submit the paperwork within 15 working days of FDA authorization. Emergency use of an investigational drug also is exempt from prior institutional review board approval as long as the use is reported to an IRB within five working days of treatment initiation.



FDA will approve an emergency IND before receiving all the necessary paperwork if the situation warrants, according to the MaPP. Agency officials also will not force the provider to produce the documentation for the drug sponsor before it provides the drug for the patient.



The agency finalized several expanded access guidances in 2016, but expanded access proponents continued to argue that the process is too cumbersome. (Also see "FDA Can't Win On Guidances: Expanded Access Policy, Duchenne Worries Collide" - Pink Sheet, 6 Jun, 2016.)



Right-to-Try Debate Ongoing

The MaPP was released amid a Capitol Hill push to improve expanded access policies.



The FDA Reauthorization Act, which would renew agency user fee programs, includes a provision requiring FDA and NIH to host a public meeting discussing clinical trial inclusion and exclusion criteria. The bill is awaiting President Trump's signature. (Also see "User Fee Bill Or Drug Pricing Bill? House Members Makes Both Cases" - Pink Sheet, 12 Jul, 2017.)



A July GAO report on FDA's expanded access program said the agency should clarify how it will use adverse event data from expanded access INDs when considering product approval. (Also see "Expanded Access: FDA To Clarify How Adverse Events Impact Drug Approval Process" - Pink Sheet, 20 Jul, 2017.)



Just before the Senate voted to send FDARA to the White House, Sen. Ron Johnson, R-Wis., pushed a bill through the chamber that would not allow FDA to use adverse events or other clinical outcomes from expanded access programs to delay drug review or approval.



Sponsors also are shielded from liability for denying expanded access in the bill. S 204 still must be approved by the House and signed by Trump. (Also see "Not Quite FDARA Add-Ons: Right-To-Try, Patient Experience, Opioid Measures Also Clear US Senate" - Pink Sheet, 3 Aug, 2017.)



Adverse events related to expanded access patients have been a point of contention in the debate over increasing access to investigational therapies. But FDA has argued safety concerns raised by an expanded access program rarely trigger a clinical hold and companies usually cite other reasons for denying access. (Also see "'Right To Try' Or 'Right To Ask'? Hearing Spotlights Adverse Events As Key Barrier To Expanded Access" - Pink Sheet, 26 Sep, 2016.)

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