Lessons Learned

Genzyme's Larry Blankstein looks at the challenges of completing global clinical trials on time and effectively.

Optimizing Genzyme's resources

Genzyme’s clinical research programs are focused on inherited lysosomal storage diseases, renal disease, orthopedics, cancer, transplant and immune diseases, diagnostic and genetic testing. As Senior Director of Clinical Research, Larry Blankstein has clinical operations responsibility for the endocrinology business unit. He has been actively engaged in a number of drug-drug interaction phase I studies, as well as thorough QTc studies in Genzyme’s other business areas.

For Blankstein, ensuring the company’s resources are being optimized across studies is an important focus. “As certain studies slow and begin to wind down, while others are entering phase III, we have to make sure our resources are utilized efficiently across studies,” he stresses. “The challenge is to make sure that we’re maximizing both our internal resources as well as our external resources in terms of their utilization and effectiveness.”

One of the most interesting developments happening has been in ultra-orphan diseases. The initial technologies deployed were enzyme replacement therapies where patients would receive IV infusions every other week. These were very successful and worked well. However, new small molecule technologies are beginning to make an impact, which means that subjects can take a pill and may not have to come into a clinic, infusion unit or a hospital every two weeks or so.

“This is one of the advances we’re seeing in this area,” highlights Blankstein. “We are expanding our platforms into other areas. For example, we have a drug used in bone marrow transplants and for multiple myeloma. We’re also looking at using this in other indications for chemosensitization assays and chemosensitization treatments. We’re using drugs that we once used to treat cancer to treat multiple sclerosis. At Genzyme we are focused on expanding our science and technology platforms to treat orphan indications or larger patient population with unmet medical needs.”

An important lesson

Blankstein describes how applying a ‘lessons learned’ process early on and throughout a development program will help the study team perform at a much higher level and be much more effective and successful at completing a trial. The key is trying to understand by using lessons learned what the challenges are for a project team as they work through a large global study. “It is important to use a lessons learned process throughout the study so the team is regularly understanding and managing team issues,” he advises. “In many situations, lesson learned are performed at the end of a project, which is often too late. By then, everything has happened.

“It’s important to identify issues that are affecting team performance early in the project so they can be dealt with immediately, so as the team progresses through a global trial they have a greater likelihood of meeting the study’s challenges and achieving success. With global trials, the goal is to complete them on time and on budget with a high level of quality to increase the likelihood of approval. Having a project team not proactively manage issues as they develop on an ongoing basis through the study can lead to significant delays and poor quality. A timely, effective lessons learned process can help avoid many of these issues.”

Future goals

Completing its clinical trials on time and efficiently is another focus for the company. Genzyme has four key clinical programs, and Blankstein believes the company has done well to organize itself around these programs and adjusting resource allocation to focus on completing them on time.

“If the company is successful doing this, then it will continue to contribute to the growth of Genzyme,” he explains. “Over the next 12-18 months, we will emphasize these key programs, keep our focus on them, but not forget other projects that are in pre-clinical, phase I or phase II that are also important for our long term growth. We have a level of resource and funding for these programs as well, so when we complete the key projects others will move up in priority. We also have a very active business development focus that looks for acquisitions, partnerships and mergers, to add to our pipeline so Genzyme can continue to provide important treatments for patients with unmet medical needs.”

In the clinical research area, Blankstein sees a number of developments ahead. He believes there will be a move towards more outsourcing models. “The traditional concept of companies like Genzyme having a large clinical staff that can manage and monitor all of their trials is changing. Companies are moving more towards outsourcing certain operations so they can focus on their core competencies with a staff that is much more experienced in how to outsource and manage service providers successfully. By doing so the relationship between the sponsor and the provider can be maximized to bring the greatest potential.”

Larry Blankstein is Senior Director of Clinical Research at Genzyme. Blankstein has more than 20 years of experience in pharmaceutical and biotechnology drug development. Prior to joining Genzyme, he was Executive Director of Program Management at Quintiles.

---EXTRA INFO---

Genzyme’s research areas

Genetic disease

Genzyme is recognized as a global leader in research, product development, and outreach to the medical and patient communities for rare genetic diseases known as lysosomal storage disorders (LSDs). In 1991, the company introduced the first product ever approved to treat a lysosomal storage disorder, Ceredase (alglucerase injection).

Since then, Genzyme has developed a second-generation Gaucher disease product, Cerezyme (imiglucerase for injection), and introduced Fabrazyme (agalsidase beta) for Fabry disease, Aldurazyme (laronidase) for Mucopolysaccharidosis I (MPS I) and Myozyme (alglucosidase alfa) for Pompe disease.

Renal disease

Genzyme is enhancing the treatment of chronic kidney disease with its phosphate binder, Renagel (sevelamer hydrochloride). Nearly all patients on hemodialysis take a phosphate binder which, before Renagel was introduced, was typically aluminum or calcium-based. Renagel is the only calcium-free, metal-free non-absorbed phosphate binder on the market.

Oncology

Genzyme’s oncology program is building a foundation in cancer treatment with a strong focus on antibody and small molecule therapies. Genzyme currently has two marketed leukemia products, Campath (alemtuzumab for injection) and Clolar (clofarabine) for intravenous infusion. The company is also focused on new treatments for cancer patients through both internal research and external collaboration.

Transplant/immune disease

The field of transplantation medicine has evolved rapidly, particularly where management of acute organ rejection is concerned. While in the past, organ loss often occurred one to two years after transplantation, the introduction of drugs such as Thymoglobulin (anti-thymocyte globulin, rabbit) has significantly improved the success rate of these surgeries.

Orthopaedics

Genzyme is a leader in the field of orthopaedics, with a promising group of products on the market and in the development. The leading product in this area is Synvisc (hylan G-F 20), a viscosupplementation treatment for relieving knee pain associated with osteoarthritis.

Adhesion prevention

The company has developed a suite of biomaterials used to help improve the outcome of certain types of surgeries. Its Sepra line of hyaluronic acid-based products has been clinically shown to reduce the incidence of adhesions following general abdominal and gynecologic surgical procedures.

Cardiovascular disease

Genzyme is a pioneer in exploring both gene therapies and cellular therapies as potential treatment modalities for serious cardiovascular diseases. With clinical programs employing both therapeutic methods, Genzyme is focused on treating ischemic diseases characterized by inadequate blood flow and poor cardiac function.

Diagnostic products and services

Genzyme Diagnostics, through its partners and distributors, offers a novel line of products for the diagnostics industry and the clinical laboratory. The company continues to develop new tests for diagnosing a variety of indications including heart disease, diabetes, pancreatitis, infectious disease, emergency medicine and women’s health.

Genzyme Genetics provides reproductive and oncology diagnostic testing services. In the area of reproductive testing, the company focuses on technology that will allow information to be provided on a range of diseases from a single patient sample.