Parkinson Pipeline Project-treatment development database, research, FDA drug approval, PD clinical trials and sponsors, patient involvement

Recruitment and Retention

Clinical Trial Facts:

In 2004, between five and six million people participated in some 80,000 clinical research studies in the U.S., and more will likely be needed in coming years to fulfill safety and efficacy requirements.
The top 20 drug companies spend $30 billion on research and development, about 40 percent of which goes to fund clinical trials.
Eight of the top 15 drug companies did not get the go-ahead for a single drug last year.
In 1980, drug companies spent some $2 billion on R&D, and 34 new drugs were approved. In 2000, they spent close to $30 billion, but only 24 drugs were approved.
Seven of 10 of drugs approved by the FDA never make enough money to justify their development costs.
Completing all the phases of clinical trials required for approval of a new drug can cost anywhere from $300 to $800 million.
Although the NIH budget has doubled in the past five years — with the implied purpose of encouraging the development of new drugs – the FDA's budget remains inadequate to review these drugs for qualification.
Drug costs are increasing by about 18 percent a year, but only 4 percent is due to price increases. The rest is the result of replacing older, more invasive, expensive, and less effective medical treatments.

Recruitment and Retention of Clinical Trial Participants

Human clinical trials are critical to improving medical treatments and finding cures for chronic, debilitating diseases, yet less than one percent of those with a given disease participate.^[1] The biggest obstacle to patient participation is lack of awareness about clinical research studies and the role they play in the drug development process.

Currently, there is no single national listing of every current Parkinson’s related clinical trial that includes procedures for enrolling and eligibility criteria for specific trials. Finding this information may be a hit-or-miss process, dependent on a patient's doctor, location, and access to informational resources rather than on appropriateness/eligibility criteria. The Advancing Parkinson’s Therapy (APT) campaign, a collaborative effort of advocacy groups, including the Parkinson’s Pipeline Project, is addressing this issue.

We are building public awareness of the critical need for patient participation in clinical trials and making information about trials available on-line at Pdtrials.org.

Difficulties in patient enrollment delay 81 percent of all clinical trials at least one to six months, with another 5 percent postponed six months or more, according to CenterWatch. “Drug companies stand to lose between $600,000 and $8 million each day clinical trials delay a drug’s development and launch. According to McKinsey & Co., the number of patients required to receive FDA approval has nearly doubled since the early 1990s, and more trials are needed per compound. These factors lead to longer trial timelines, which exacerbate an already burdensome issue: rising clinical costs.”^[2]

The average per-patient cost is about $5,500 for a Phase I trial; $6,500 for a Phase II trial; and more than $7,600 for a Phase III trial. Recruiting clinical trial participants “costs more and consumes more time” than any other aspect of the drug development process.

“With so many parties involved in clinical development — study sponsors, clinical research offices (CROs), site management organizations (SMOs), patient recruiters, investigators, and patients — it is no surprise that on average, trials last 30%-42% longer than expected. Improving communication and coordination among the many stakeholders can shorten trials, potentially saving lives and generating significant additional revenue to reinvest in the research and discovery of new therapies.” ^[2]

More effective recruitment can stem rising costs

Effective recruitment offers one of the greatest opportunities to stem rising clinical costs and accelerate the trial process. Creating more efficient marketing campaigns and new ways of attracting patient populations will provide an edge as competition for patients increases. To further improve patient recruitment and retention, trial sponsors are:

Employing professional patient recruitment firms and site management organizations.
Reducing the number of participants in any given trial.
Outsourcing clinical trials to developing countries where costs are less. One-third of the participants in any given trial, however, must come from the U.S. and/or Europe to avoid skewing results because of cultural, nutritional, or economic differences of foreign trial participants from the average American trial participant.
Shortening trial duration by securing FDA "fast track" designation for certain drugs or treatments (primarily cancer). Approval is granted before all the clinical trials are conducted, with the sponsor promising to complete the clinical trials after approval is obtained. The risk is that these "catch up" trials do not always take place.
Obtaining insurance against the risk of failure to obtain FDA approval.^[3]

The FDA encourages sponsors to determine why participants withdraw from studies because it could be indicative of an important safety problem. It is not helpful to record vague explanations such as "withdrew consent," "failed to return," or "lost to follow-up." Participants who leave a study because of significant safety issues should be followed closely until they are fully and permanently resolved. Pipeline Project Research Partners could play a vital role in this area.

Advocates positively impact clinical trials

Once enrollment is complete, the challenge is keeping patients interested, participating, and committed throughout the course of the clinical trial. Attrition rates are near 25 percent, and higher for long-term trials. The Pipeline Project’s trained patient Research Partners, Company Advisors, and FDA Consultants can promote increased retention by:

Encouraging participation in clinical trials through education and outreach, innovative recruitment techniques, and expanded reimbursement of clinical trial expenses.
Ensuring that patients fully understand their role in the research process, how their contributions can make a difference, and what rights and protections they have.
Intervening on behalf of trial participants to resolve problems that may otherwise lead them to drop out of the study.
Advocating for greater sharing of databases and clinical trial data to accelerate medical research.

A five-year study involving the University of California-San Francisco found that integrating advocates into the administration and planning process in the clinical and research program positively impacted outcomes in research, clinical trials, patient services and philanthropy. Improvements were noted in all assessed areas, most significantly in research grants (286%) and philanthropy (1,796%)^.[4]

The 100 or so advocates who are part of UCSF Prostate Cancer Specialized Program of Research Excellence (SPORE) include cancer survivors and family members dedicated to advancing and supporting activities that will lead to improved awareness, diagnosis, and treatment of prostate cancer. “Bringing patient viewpoints into medical research,” they participate in biweekly research discussions and informal multidisciplinary discussion sessions; help with trial design, patient enrollment, Institutional Review Board approvals, and fundraising; and review eligibility criteria, informed consent documents, and the necessity of frequent invasive tests to eliminate built-in barriers to participation.^[3]

Advocates brought about at least 25 new initiatives to improve patient services in the areas of access to care, patient education, psychosocial support, outreach, etc. Since the initiation of a prostate cancer advocacy effort at UCSF, substantial and quantitative improvements in several key metrics have been identified. Although an exact cause and effect cannot be stated with certainty, it seems likely.

Involving participants improves retention

Improved retention of clinical trial participants requires:

Making participants feel comfortable, informed, and valued at each contact.
Building trust.
Promoting a sense of being a member of the study team.
Involving patients in designing the research and preparing study materials to be sure they meet their needs and expectations.
Creating a participant advisory board to give feedback on all aspects of the trial.
Having the participant see the same staff member at each visit.
Maintaining extended, flexible clinic hours and providing at-home follow-up.
Choosing a comfortable, secure study site, with free parking.
Reminding participants of upcoming appointments.
Recruiting patient volunteers to assist with daily trial tasks.
Regularly informing participants about the study protocol, treatment, trial outcomes, and implications through meetings, newsletters, or other updates.

Sources:

[1] http://www.patientshelpingdoctors.org/phd/patients_challenges Faster Cures: Patients Helping Doctors The PHD Program

[2] www.AcceleratedClinicalTrials.com Cutting Edge Information White Paper- Accelerating Clinical Trials: Budgets, Patient Recruitment and Productivity

[3] Journal of the National Cancer Institute Cancer Spectrum Vol. 90, No. 16
Vanchieri, pp. 1193-1195 Patient Advocates Help Researchers Avoid "Bumps in the Road"

[4] J Urol. 2004 Nov;172(5 Pt 2):S58-61; discussion S61-2. The impact of patient advocacy
PMID: 15535445 [PubMed - indexed for MEDLINE] www.fastercures.org; Faster Cures The Center for Accelerating Medical Solutions, Clinical Trials Delayed 30-42% Due to Patient Recruitment Difficulties
PR Newswire via NewsEdge Corporation March 22 /PRNewswire
http://www.market-research-report.com/datamonitor/DMHC1819.htm; Faster Cures Acceleration Agenda www.fastercures.org/sec/clinical sidebar