Good morning, my name is Jayson Slotnik and I am here on behalf of the Biotechnology Industry Organization (BIO), the trade association for biotech companies, academic institutions and scientific entrepreneurs. Our over 1,000 members brought you over 200 new biotech drugs and vaccines that have helped over 325 million people worldwide. Over 300 biotech drugs are also in clinical development addressing cancer, heart disease, Alzheimer's, obesity, Parkinson's and other intractable diseases.
After careful consideration and review of these draft guidelines, BIO is concerned that these therapeutic categories and pharmacological classes will NOT ensure that the entire Medicare population has access to needed lifesaving medications. Approximately one in eight Medicare beneficiaries is disabled including: 74,000 beneficiaries with AIDS, 100,000 beneficiaries with multiple sclerosis, and 150,000 beneficiaries with lupus. In addition to the disabled Medicare beneficiaries, BIO's products treat millions of Medicare beneficiaries who suffer from rare diseases including rare forms of cancer, blood diseases and immune deficiencies. Our treatments are often the last line of therapy used to treat these life threatening ailments.
Congress entrusted USP with tremendous responsibility in developing these draft guidelines because of the safe harbor available to private plans who adopt the USP guidelines. BIO believes that these draft guidelines do not have enough protections in place to guarantee sufficient access for the entire Medicare patient population to their needed medicines. BIO urges USP to address the following three (3) issues and adopt the following four (4) recommendations as it completes its model guidelines.
Orphan Drugs- BIO disagrees with the way orphan drugs are classified. By definition, an orphan drug treats a rare disease and in many instances is the only product available for that particular disease. More than 50 percent of biologicals on the market are orphan drugs and as in real estate, the three most important aspects of formulary placement are location, location, location. We believe that these guidelines ignore each of these important aspects.
First, the draft guidelines place many of the orphan products into categories with a large number of drugs, or even in the same category creating the very real possibility that these drugs will not be covered. Second, no appropriate category exists for many of our drugs. For example, there is no category for drugs that treat either neurological or neuromuscular diseases.
Therefore, BIO recommends that USP and CMS work together to develop one comprehensive therapeutic category that includes all FDA approved orphan drugs AND to require all private plans to cover each drug in this category in order to receive CMS approval. This is the only way to apply the required sensitivity to patients who suffer from rare diseases.
Uniquely Classified Products- Similar to orphan products, many of our treatments for blood disorders, autoimmune diseases and organ failure are the only available treatment in their category or class. BIO is concerned that the USP has not adequately addressed these drugs because each drug is either placed in the wrong category, not clearly identified as to which category it will be in, or it is completely excluded. These uniquely classified products require special attention, in the form of their own category or class in order to ensure that the entire Medicare population has access to these lifesaving treatments. BIO urges the USP to further refine the system to take these products into account.
Vaccines- Vaccine products cover a broad range of therapeutic categories similar to anti-infectives and therefore there should not be one broad class as you have created called Immunizing Agent (Active). Because vaccines have been developed to treat or prevent bacterial, viral, fungal and other types of infections, we recommend that vaccines be assigned a separate therapeutic category with pharmacological classes appropriate for the therapeutic area such as Bacteria, Virus, and Fungi. Specifically, we urge the USP to create separate sub-classifications for each antigen; for example, Hepatitis B and Hepatitis A.
Under the current therapeutic category for hormones the committee created various pharmacologic classes for Adrenal, Parathyroid and other regulation agents. However, they omitted a category for Phosphate Binding agents and we urge the USP to create a new pharmacologic class for Phosphorus Regulating Agents with a subdivision for Calcium Acetates.
With an eye towards the future, recent data from the biotech and the scientific community suggest that vaccines may well become useful preventative or therapeutic modalities against neoplastic diseases and immunologic disorders such as Alzheimer's or Parkinson's disease, and we urge USP to take this data into consideration as it modifies these guidelines.
Include Recommended Column as Separate Pharmacological Classes- BIO recommends that the USP adopt its recommended subdivisions as additional pharmacological classes. The expert committee clearly stated in their "Companion Document" to the draft guidelines that the recommended subdivisions column was included (and I quote) "as an illustration of subclasses that would ensure beneficiary access." The goal of these draft guidelines is just that, to ensure beneficiary access. Therefore, the recommended subdivisions should be included as separate pharmacological classes to accomplish this goal.
How Will the Model Guidelines Adopt to New Therapeutic Categories and Pharmacological Classes?
BIO members often create and invent products requiring a new therapeutic category and urge USP for some clarity and certainty beyond the statutorily stated "from time to time" on how new therapeutic categories will be added. BIO believes that the model guidelines must include criteria and a process to accommodate new categories, classes, products and new indications of products. This process must be transparent, science-based and open for public input.
Definition of Therapeutic Categories-As the USP proceeds, BIO requests that you publish definitions of each of the therapeutic categories and pharmacological classes so our members can have a better understanding of how their drugs and biologicals will be treated. This understanding will help the members adequately prepare their written comments.
Population of Categories and Classes Should be a Public Process- According to the Cooperative Agreement between CMS and USP, once the categories and classes are finalized the USP will populate each category and class. BIO urges the USP to conduct a similar public process to seek stakeholder input regarding the population process.
In conclusion, BIO appreciates the opportunity to testify today and looks forward to working with USP, CMS and our fellow members of the Manufacturers Advisory Forum to develop Model Guidelines that allow for clinically appropriate combination therapies, provide Medicare beneficiaries with adequate and real choices and support clinically appropriate off-label use. Thank you very much.