Thirty years ago, patient recruitment was a challenge for pharma companies. Today the patient participation rate in trials still hovers around five percent and is not expected to improve anytime soon. Patient recruitment remains a challenge that pharma must work to overcome.
In addition to my career as a pharma industry consultant, I’m a writer. While I don’t claim to be the next Ernest Hemingway, I do consider myself a decent writer. I’ve put in the 10,000 hours of writing Malcolm Gladwell told us it takes to be an expert in his 2008 book, Outliers: The Story of Success. I’ve always had a passion for writing. At 24, I made a serious commitment to become a real “writer.”
Good recruitment and retention is critical to the success of clinical trials. Get it right, and a trial will likely achieve its primary objective; get it wrong, and the time, effort, expense, and any patient participation is likely wasted. This article discusses how a new national approach to involving patients and the public in the U.K. is helping life sciences companies get it right the first time.
Developing new, innovative pharmaceutical agents to address global health needs is becoming more complex, challenging, and costly. As the industry attempts to adapt and gain value for patients, healthcare providers, and other stakeholders, technologies, digitization, real-world data, immediate access to clinical trial data, and the adoption of wearable devices provide both opportunities and challenges. Disparate attempts to incorporate these capabilities across pharma, biotech, contract research organizations (CROs), regulators, and investigative sites create confusion and frustration while frequently adding unintended costs and quality issues.
When it comes to clinical supplies, the journey is every bit as important as the destination and the price of failure is high. This paper discusses how supply logistics are changing and contains examples of how Fisher Clinical Services is deploying flexible solutions to ensure secure, efficient and cost-effective passage of clinical supplies.
For biopharmaceutical sponsors, clinical trial data are both the greatest organizational asset and the greatest challenge. This paper discusses the principles of data governance and how they are used to build a business intelligence framework that advances data quality, acquisition, and integration to deliver actionable information for use across the drug development enterprise.
How can researchers identify participants’ overall level of suggestibility and then focus on the minimization of this characteristic to solve the issue of suggestibility in clinical trial?
Genetic testing is making it easier to identify patients for clinical trials. However, genetic testing in the context of clinical trials raises raise important ethical issues, including ones related to informed consent and disclosure of results. Genetic counselors can play a crucial role in helping sponsors address these operational and ethical issues, making trials more efficient, more patient-centered and, ultimately, more successful.
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Good recruitment and retention is critical to the success of clinical trials. Get it right, and a trial will likely achieve its primary objective; get it wrong, and the time, effort, expense, and any patient participation is likely wasted. This article discusses how a new national approach to involving patients and the public in the U.K. is helping life sciences companies get it right the first time.
There has been an increasing and requisite focus on the needs of individuals affected by chronic or life-limiting disease, which has in turn resulted in a focus on the development of possible interventions and how they are administered. The success of participation and retention of these patients in clinical research, advancement of the drug development process, and adherence post-commercialization require the involvement of another critical expert — the caregiver.
Every biopharma company talks about putting the patient at the center of everything they do, incorporating the voice of the patient and being patient-centric. However, very few companies are actually doing it. Why is it so hard to conduct clinical development in a patient-centric manner? What actions can we take in 2019?
The ever-increasing demand for healthcare advances has prompted legislators to consider means of facilitating the development of potentially life-saving new therapies. In this guest column, Alison Wilson will review the definition of a regenerative medicine advanced therapy (RMAT) and how this designation can facilitate speed to market for these innovative therapies. Alison is an independent regulatory affairs consultant specializing in human cell and tissue-based advanced therapies.
In December 2016 the US Congress enacted the 21st Century Cures Act1, which provides for >US$6 billion in funding intended to facilitate, among other things, action against the misuse of opioids and promotion of measures to increase health insurance protection for mental health issues. Of specific interest to the advanced therapy industry, the Act required FDA to facilitate an efficient development program for, and expedited review of, new medicines meeting the definition of a regenerative advanced therapy (RAT) (Section 3033). The RAT designation in the legislation is now referred to as the Regenerative Medicine Advanced Therapy (RMAT) designation. Such therapies are defined as a:
cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products, which are intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition
Cell and tissue-based products regulated solely under Section 361 of the Public Health Service Act and 21 CFR part 12712 are excluded from the definition: broadly these are minimally manipulated human cell/tissue products for which claims are restricted to homologous functions, such as decellularized skin or demineralized bone matrix.
Details of the RMAT designation are set out on the relevant CBER webpage3. Sponsors can find help on interpretation of the terms “serious disease or condition” and “unmet medical need” in FDA’s guidance document on fast-track expedited programs4.
Requesting RMAT Designation
Sponsors of drug products meeting the above criteria can request RMAT designation from the Office of Tissues and Advanced Therapies (OTAT) when submitting an Investigational New Drug application (IND), or after one has been submitted. This implies that the Agency will accept early non-US clinical data, since the designation requires preliminary clinical evidence that the new therapy may address an unmet medical need. However the IND must be active, and not on hold; if the IND part of a combined submission is placed on hold the RMAT designation will not be issued.
RMAT designation requests do not require submission of full clinical data; instead sponsors must describe the preliminary clinical evidence on which the request is based. This should include a brief description of any available therapies for the disease or condition, the study design including population studied and endpoint(s) used. A description of the study results and statistical analyses should be provided. The Agency has 60 days to review the request for RMAT designation; if it is denied OTAT will provide a justification for not assigning the designation.
Benefits of RMAT designation
The RMAT designation gives the sponsor of a new drug access to increased meeting opportunities with FDA, in a manner comparable to those offered to sponsors of breakthrough-designated therapies. In fact the RMAT may be considered as analogous to the breakthrough designation for regenerative medicine drugs. Because the designated products meet the criteria for unmet medical need in the treatment of a serious condition, they may be eligible for priority review, in which the initial assessment of the BLA is reduced from 10 months to 6 months, and accelerated approval, which bases approval on an effect on a predictive surrogate endpoint or an intermediate clinical endpoint. RMATs qualifying for accelerated approval may be able to satisfy licensing requirements through commitment to post-approval clinical studies as well as real-world data such as patient registries and health record analysis: as a consequence of the 21st Century Cures Act, FDA are now required to take account of clinical evidence beyond controlled clinical trials. The eligibility of the RMAT-designated product for these expedited programs can be discussed with FDA at specific development meetings. The increased access to FDA during early development is a significant benefit for sponsors, because the typical Type B development meetings are normally restricted to one each at pre-IND, end of Phase II/pre-Phase III and pre-BLA submission. In addition, the option to qualify for fast-track approval, also based on the potential to serve an unmet medical need in the treatment of a serious condition, allows for a so-called “rolling review” of parts of the BLA, which can be submitted for assessment following agreement of a review timetable with CBER.
Recent Approvals
Several products have already achieved RMAT designation since the program’s inception, including:
Outlook
It appears that the RMAT designation has caught the attention of advanced therapies. The opportunity for early and more frequent interactions with FDA during critical development stages is understandably very attractive to the companies involved in advanced therapy development, many of whom are small, clinician-led enterprises with little experience of conventional drug development pathways. The extent to which we can shift the balance of evidence from licensing to the post-marketing phase is something that regulators and sponsors are exploring in many expedited development programs, and this takes on an urgent appeal when considering the life-threatening nature of many of the conditions these regenerative medicines are intended to address. The flexibility granted to FDA in considering forms of clinical evidence other than the usual randomized controlled clinical trials could facilitate pragmatic decision-making and the possibility of bringing key new therapies to market more quickly, or represent a weakening of the requirements and reduction in patient protection depending on your viewpoint. More RMAT designations are anticipated; it will be several years before we see whether this initiative is achieving its goal of facilitating faster development whilst maintaining rigorous standards of safety and efficacy.
The current limitations on the use of immunotherapy drugs could be swept away thanks to the development of immune checkpoint inhibitors. They block disruptive proteins that limit the body’s natural immune response and stop T-Cells from destroying cancer cells. Some of the biggest factors stopping the more widespread use of these drugs are the limitations of current standards in effectively measuring how well they work. Read how improvements in the methods used for immuno-oncology drug trials could benefit both researchers and patients.
Deep-learning algorithms have shown significant promise as applications in natural language understanding, decision making, and speech and image recognition. These algorithms are now being applied in bioinformatics applications within the biopharma industry to manage the increasing amounts of data from high-throughput techniques. Read more about recent applications of these algorithms to predict a variety of biological processes and interactions, particularly with respect to proteins.
Determining appropriate stratifications and relevant clinical endpoints for specific sub-populations can be challenging. Therefore, it is necessary for development strategies to incorporate explorations and determinations of suitable biomarkers early in the development of a new therapy.
In this blog, Jonathan Pritchard, Director Business Development at Cytel, draws on his experience in commercial, clinical and technology roles within the biopharmaceutical industry and shares his insights on the primary considerations for sponsors when implementing an ePRO solution.
