FDA Hearing on HCV Oral Drugs Early Access

FDA Hearing on HCV Oral Drugs Early Access

The link for the Notice is at http://edocket.access.gpo.gov/2010/pdf/2010-5055.pdf

"6. How can pharmaceutical companies, government, academia, and community physicians and activists collaborate to provide for the treatment use of multiple new agents with the goal of maximizing response and reducing adverse reactions?

7. In the course of developing DAAs for marketing, what types of studies should be conducted to best address unmet medical needs for patients with CHC including those with the greatest risk of progression of liver disease and/or the lowest predicted virologic response rates?"

"Under what circumstances and in which populations would early access to multiple DAAs be appropriate?......Some patients with CHC who have not responded to approved treatments and/or who are at substantial risk of liver disease progression may benefit from access to new therapeutic options before approval through the Expanded Access Rule. On the other hand, receiving preapproval treatment access via a treatment protocol may have potential risks such as adverse reactions or the development of drug or drug-class resistance. Historically, early access programs with antiretrovirals for the treatment of HIV allowed many people to gain access to life-saving drugs. For some individuals, however, early access to a drug resulted in what amounted to sequential monotherapy and the emergence of multidrug resistance. Similar to HIV treatment concerns, drug resistance and drug-class resistance are concerns for DAAs to treat CHC. Because treatment of CHC requires multiple agents to achieve acceptable SVR rates and to reduce the emergence of drug resistance to single agents or drug classes, treatment INDs that include two or more investigational agents or that allow for co-enrollment in several treatment IND programs are options to consider, particularly for previous null responders or for patients who cannot take interferon-based regimens. However, the use of multiple agents in the context of a treatment IND adds to the complexity of the implementation and design of treatment IND protocols. In light of the foregoing, FDA is soliciting advice from the public on how treatment access protocols for hepatitis C DAAs may best be designed."

DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA–2010–N–0107]

Expanded Access to Direct-Acting Antiviral Agents for the Treatment of Chronic Hepatitis C Infection in Patients With Unmet Medical Need; Public Hearing; Request for Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public hearing; request for comments.

SUMMARY: The Food and Drug Administration (FDA or the agency) is announcing a public hearing to obtain

input on the scope and implementation of potential expanded access programs with direct-acting antiviral agents (DAAs) for the treatment of chronic hepatitis C (CHC) infection in patients with unmet medical need. This public hearing is being held to obtain comments from the public on eligibility criteria that should be used for patient enrollment in expanded access protocols involving DAAs and to elicit

suggestions for designs of protocols for treatment investigational new drug applications (INDs) involving DAAs and other expanded access protocols. In addition, the agency would like public input on types of studies that should be conducted to obtain information on patients with unmet medical need including those with the greatest risk of progression of liver disease and/or the lowest predicted virologic response rates.

DATES: The public hearing will be held April 30, 2010, from 9 a.m. to 4 p.m. The meeting may be extended or may end early depending on the level of public participation. Submit written or electronic requests for oral presentations and comments by April 8, 2010 (see

section III of this document for details). Written or electronic comments will be accepted after the hearing until June 25,

2010 (see section V of this document for details).

ADDRESSES: The public hearing will be held at the Hilton Hotel, 1750 Rockville Pike, Rockville, MD 20852. Additional

information on parking and public transportation may be accessed at http://www1.hilton.com/en_US/hi/hotel/IADMRHF-Hilton-Washington-DCRockville-Hotel-Executive-Meeting-Ctr-Maryland/index.do. (FDA has verified the Web site addresses throughout this document, but FDA is not responsible for any subsequent changes to the Web sites after this document publishes in the Federal Register.) Submit written comments to the Division of Dockets Management (HFA–305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852. Submit electronic comments to http://www.regulations.gov. All comments should be identified with the docket number found in brackets in the heading of this document. Transcripts of the hearing will be available for review at the Division of Dockets Management and on the Internet at http://www.regulations.gov approximately 45 days after the hearing (see section VI of this document).

FOR FURTHER INFORMATION CONTACT:

Susie Dill, Center for Drug Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 51, rm. 6183, Silver Spring,

MD 20993–0002, 301–796–3437, FAX: 301–847–8753, e-mail: AccessToDAA@fda.hhs.gov.

II. Scope of the Public Hearing

FDA is interested in obtaining public comment on the following issues related to expanded access of DAAs for the treatment of CHC:

1. What types of patients with CHC are most appropriate for participation in DAA expanded access for CHC with regard to disease stage, previous treatment, and other disease characteristics?

2. Under what circumstances and in which populations would early access to a single DAA be appropriate?

3. Under what circumstances and in which populations would early access to multiple DAAs be appropriate?

4. How can pharmaceutical companies, government, academia, and community physicians and activists collaborate to provide for the treatment use of multiple new agents with the goal of maximizing response and reducing the emergence of drug or multidrug resistance?

5. What potential adverse reactions should be contemplated in formulating DAA treatment IND use protocols?

6. How can pharmaceutical companies, government, academia, and community physicians and activists collaborate to provide for the treatment use of multiple new agents with the goal of maximizing response and reducing adverse reactions?

7. In the course of developing DAAs for marketing, what types of studies should be conducted to best address unmet medical needs for patients with CHC including those with the greatest risk of progression of liver disease and/or the lowest predicted virologic response rates? Examples of studies that help to support clinical protocols or treatment use protocols in populations of unmet medical need may include renal and hepatic impairment studies and drug-drug interaction studies with antiretrovirals.

III. Attendance and/or Participation in the Public Hearing

The public hearing is free and seating will be on a first-come, first-served basis. Attendees who do not wish to make an oral presentation do not need to register. If you wish to make an oral presentation during the hearing, you must register by submitting a written or electronic request by close of business on April 8, 2010, to Susie Dill (see FOR FURTHER INFORMATION CONTACT). You must provide your name, title, business affiliation (if applicable), address, telephone and fax numbers, e-mail address, and type of organization you represent (e.g., industry, consumer organization). You also should submit a brief summary of the presentation, including the discussion topic(s) that will be addressed and the approximate time requested for your presentation. We encourage individuals and organizations with common interests to consolidate or coordinate their presentations to allow adequate time for each request for presentation. Persons registered to make an oral presentation should check in before the hearing. Participants should submit a copy of each presentation to Susie Dill (see FOR FURTHER INFORMATION CONTACT). We will file the hearing schedule, indicating the order of presentation and the time allotted to each person, with the Division of Dockets Management (see ADDRESSES). We will mail, e-mail, or telephone the schedule to each participant before the hearing. In anticipation of the hearing presentations moving ahead of schedule, participants are encouraged to arrive early to ensure their designated order of presentation. Participants who are not present when called risk forfeiting their scheduled time.