An Update on the Abigail Alliance for Better Access to Developmental Drugs

by

Frank Burroughs

President, Abigail Alliance

The Abigail Story

The genesis of the Abigail Alliance for Better Access to Developmental Drugs can best be told in the Abigail story.

The Abigail Alliance for Better Access to Developmental Drugs was incorporated in the state of Virginia in November of 2001 as a 501 (C3).  However, the Alliance really started in early March of 2001 when my beloved daughter and only child Abigail, who had just turned twenty-one, had run out of FDA approved options in her battle against cancer, and was being treated at Kimmel Cancer Center at Johns Hopkins. Abigail's very talented oncologist urged us to try and get the EGFR targeted drug Erbitux from Imclone Systems or as a backup Iressa from Astra Zeneca.

Abigail's cancer cells had a very high EGFR expression and her oncologist strongly felt these drugs had a very significant chance of saving her life.  As happens to 97 percent of people who could benefit from investigational drugs and try to get into clinical trials, Abigail could not qualify for the strict protocol of the clinical trials.  Two and a half years after the loss of University of Virginia Echols Scholar and honors pre-law student Abigail, at just 21, Erbitux was found to work even better in the head and neck cancer that Abigail had than in the earlier Erbitux colon cancer clinical trials.

Abigail was still strong when we launched, with Abigail's involvement, a three-pronged approach to get better access to developmental drugs. We lobbied the two pharmaceutical companies with much vigor, including getting help from some very influential people. Then we worked hard and furiously to solicit Congressional help. With Abigail, we launched a media effort that resulted in numerous stories in the press. As tired and weak as she was, Abigail did multiple newspaper and television interviews. We were finally able to get Abigail into a clinical trial of a third drug (OSI774). However, she died on June 9, 2001. Within the day, I decided that I would continue with her efforts.

After a third Washington Post story published in June 2001 about her, I was asked to testify before Representative Dan Burton's Government Oversight Committee later that month. During the four-hour hearing on the issue of expanded access and compassionate use, I was asked by Representative Burton as to whether I was mad at the pharmaceutical companies. My reply was, "We do not need any enemies regarding better access to developmental life saving drugs. We need the help of the multi-billion dollar pharmaceutical industry, the U.S. Government, and others to help solve this problem."

One of the early accomplishments of the Abigail Alliance was our successful effort in negotiating an expanded access program for Erbitux with Imclone Systems and Bristol-Myers Squibb. Although there is significantly more to do, the Abigail Alliance does have an impressive list of accomplishments that were achieved in a fairly short time and on a very slim budget.

The Abigail Alliance Mission

The main mission of the Abigail Alliance is to help cancer patients and others with life-threatening illnesses. In this context we attempt to:

	Educate the public on the need to have more expanded access and compassionate use programs for developmental cancer drugs and other developmental drugs for life-threatening illnesses for patients  that have run out of conventional treatment options.
	Develop creative ways to help pharmaceutical companies do more expanded access and compassionate use without putting the burden on the companies. This could save thousands of lives a year.
	Research current government policies and regulations to help insure that there are no Federal Government issues that are blocking wider access to developmental drugs.
	Research and make sure current regulations are being followed so that cancer patients and other patients have a complete listing of current available clinical trials.
	Negotiate with pharmaceutical companies to add or increase expanded access and compassionate use of developmental potentially lifesaving drugs to help cancer patients and others who have exhausted conventional treatments.
	Help cancer patients and others who have exhausted conventional treatments for their life threatening illness find clinical trials, expanded access programs and find compassionate use programs.
	Help develop specific legislation that could help increase expanded access and compassionate use of developmental cancer drugs and other potentially lifesaving drugs, and legislation that could help companies develop new drugs faster.
	Work to develop ways to make sure more oncologists and other doctors refer patients to information about clinical trials, especially at NCI’s web sites clinicaltrials.gov and Cancer.gov.

Our long range goal and focus is to raise money to help poor cancer patients and others with life-threaten illnesses with travel costs to get to treatment in clinical trials or expanded access programs.  Another long term goal and focal point is to continue to work with individual patients in guiding them to information that many help them in there battle to live.

The Abigail Alliance has two very recent efforts to advance changes that could save and extend tens of thousands of lives every year. The first is reflected in the current input from the Abigail Alliance at the website NCI Listens.

The second very recent effort is the Abigail Alliance’s Considering the Needs of Patients on the Edge. This is an initiative to bring about a portion of much needed change this year in the U.S. Congress, the FDA, and the Department of Health and Human Services.

Our Ongoing Efforts

	Developed the ‘Tier 1 Initial Approval’ (‘Tier 1’) initiative that would greatly increase access to developmental drugs to tens of thousands of cancer patients and others with life threatening illnesses.
	Filed a lawsuit in U.S. District Court in Washington D.C. in support of better access to new life saving therapies with the help of the Washington Legal Foundation.
	Filed a Citizens Petition with the FDA in support of ‘Tier 1’, again with the help of our close friends, the Washington Legal Foundation. We have learned that the FDA is working on proposed policy changes as a result of the efforts of the Abigail Alliance.
	Continue to keep up our media overage of Abigail Alliance issues.
	Continue to elevate the issue of helping tens of thousands of precious people with our work in the media, at the FDA, on Capitol Hill, in the advocacy community, and elsewhere.
	Helped get two expanded access programs launched including one for the colon cancer drug Erbitux one year before FDA approval.
	Continue to provide patients with information and emotional support.
	Helped get several people back on to developmental drugs, after they had to leave clinical trials on minor issues, but were responding well to their therapies.
	Have helped some patients get compassionate use of promising life saving drugs.
	Continue to promote ways to better inform the public about clinical trials.
	Influencing the move toward faster review and approval of new life saving drugs.
	Continue to build support on Capitol Hill.
	Continue to gain support from prestigious individuals and organizations.

The Abigail Alliance is moving forward. We have been speaking at major meetings, promoting creative ideas at the FDA and on Capitol Hill, getting information out to the public, and directing cancer patients to reliable sources for further information.

Abigail Alliance Input to the NCI’s Challenge Goal to the Nation is to Eliminate the Suffering and Death due to Cancer

1. 1. A very important way to move forward has been and continues to be very vigorously pushed forward by the Abigail Alliance and many others; the Abigail Alliance ‘Tier 1 Initial Approval’ initiative ‘Tier 1 Initial Approval’ would bring promising cancer drugs and drugs for other life-threatening illnesses to people fighting for their lives years earlier. It is incredible that the FDA has been sitting on the Abigail Alliance and Washington Legal Foundation Citizens Petition regarding ‘Tier 1 Initial Approval’ for almost two years! They are supposed to respond within six months!

2. 2. A second very important way to move forward regarding this vital issue is the FDA Critical Path Initiative. However, the Critical Path Initiative needs more attention from the FDA and needs to move forward with great vigor! We all need to push the FDA forward as regards Critical Path.

3. 3. Another important way to move forward has been sadly sidelined by the Vioxx mess and that is the FDA vitally needs to finish its work on their proposed treatment and policy changes.

4. 4. Lastly, in order to better help so many people fighting for their lives, we need a forward thinking creative person to head the FDA Oncology Division.

Message to the FDA, Health and Human Services and The U.S. Congress—March 4, 2005

Recent events with high profile prescription drugs have caused many to question the effectiveness of our nation’s drug development and approval process. In response to these events FDA and HHS have proposed immediate reforms and requested a comprehensive review of existing performance and policies by the Institute of Medicine.  Congress has conducted hearings on our drug development and approval process and is considering legislation to expand on the reforms already proposed. In this atmosphere of intense scrutiny and impending change, it is critical that the needs of patients with serious, life-threatening and terminal medical conditions be fully considered and addressed.

We strongly urge the FDA, Health and Human Services, and the U.S. Congress to consider and act on the following issues:

1. Designate the FDA’s Critical Path Initiative as a top priority for FDA, Health and Human Services and the nation.  Provide staffing and grant funding resources for the Critical Path effort, set goals and reporting dates for tracking progress, and establish a mission statement for Critical Path calling for the timely transformation of scientific progress into regulatory and medical practice.  Require Health and Human Services to report to Congress on the accomplishments of the Critical Path Initiative semi-annually, with the first report detailing the structure, objectives and priorities of the initiative to be submitted to the Senate HELP Committee and the House Energy and Commerce Committee on or before May 1, 2005.

2. Complete and issue in a timely manner the draft Treatment Use regulation clarifying and modifying FDA policies regarding patient access to investigational drugs.  Incorporate into the draft regulation reforms that address in a meaningful way the needs of patients who have exhausted approved options and cannot gain entry into appropriate clinical trials.  Require the FDA and Health and Human Services to issue the draft regulation for comment no later than April 1, 2005.  (This was in process, but sidelined this past fall by the drug safety issue.)

3. Include written and live testimony from at least one panel of patient advocates, including at least one patient advocate representing a spectrum of patients with life-threatening and terminal diseases, in any and all Congressional hearings addressing drug safety, drug development, and drug approval issues. 

4. Amend the FDA’s mission statement to include pursuit of continual improvement in its regulatory practices and performance for all segments of the patient population.  Specify that such continual improvement should be based on delivery of progress to the universe of patient populations that fully recognizes the widely divergent risk/benefit calculus that applies to those populations.  An example is the difference between a teen with acne and an advanced pancreatic cancer patient.  Acne is not a life-threatening disease – advanced pancreatic cancer is a terminal disease.  Current drug approval and access policies do not adequately address the needs of the advanced pancreatic cancer patient. Hold joint Congressional hearings on the establishment and pursuit of the new mission statement by April 1, 2005 or as soon as practicable, and direct FDA and HHS to produce a plan for pursuing continual improvement to the Senate HELP and House Energy and Commerce Committees by June 1, 2005.

5. Select forward-thinking, reform-minded leadership for the FDA’s new Office of Oncology Drugs.  The need for rapid reform and new regulatory concepts for development and approval of science-based oncology treatments should exclude consideration of candidates that support the status quo, or who are likely to serve as defenders of current practices and policies. 

6. Any and all reforms proposed to improve the detection of drug safety issues and support regulatory determinations must be balanced with similar reforms and improvements in the drug development process and the tools used to identify and measure the benefits of new and existing treatments.  We strongly counsel the FDA, HHS and Congress to avoid implementing unbalanced reforms.

7. Congress should direct and fund HHS with new legislation to implement a national medical progress awareness program.  The program should focus on an explanation of the process, the important roles of stakeholders, and the critical need to continue the pursuit and delivery of medical progress to the public.

V2N2 ESUN Copyright © 2005 Liddy Shriver Sarcoma Initiative