A Big Year for Novel Drugs Approvals

What was your top Biopharma highlight for 2012? If it wasn’t the stellar move in the AMEX Biotech Index (up 42%), or the reauthorization of PDUFA, then perhaps it was the decade high for innovative drugs approvals. We would need to go back to the 1990s to find a year with as many FDA approvals for novel drugs as we had in 2012. Adding to the positive trend in numbers, up 29% from the robust 31 approvals in 2011, a number of last year’s approvals came earlier than expected. Vertex’s Kalydeco and Ariad’s Iclusig, for example, both came three months ahead of their respective PDUFA dates. Over 50% of the approvals had FDA Priority review or Orphan designations.



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Pharma & Biotech Companies Donate Hundreds of Millions of Dollars to Copayment Foundations. Here’s Why.

from BIOtechNow by biotechnow@bio.org (Biotechnology Industry Organization)

 

In addition to using their own Patient Assistance Programs, many pharma and biotech companies are supporting independent 501(c)3 non-profit copayment foundations to increase treatment compliance, access to care for insured Americans (including Medicare patients), and to promote proper disease management.

Mary P. Sundeen, President, HealthWell Foundation

Copayment foundations have helped hundreds of thousands of insured Americans afford their coverage and their treatments. The HealthWell Foundation, one of the nation’s largest non-profits, recently issued a white paper on copayment assistance as a primer for pharmaceutical and biotechnology personnel. Our goal is to assist with reducing a manufacturer’s exposure to legal pitfalls and to maximize their charitable donation.

for complete article:

Pharma & Biotech Companies Donate Hundreds of Millions of Dollars to Copayment Foundations. Here’s Why.

BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

from BIOtechNow by biotechnow@bio.org (Biotechnology Industry Organization)

 

The signing of FDASIA by President Obama in July reinforces the country’s commitment to fostering innovation for rare diseases. With a record 460 medicines in late-stage clinical trials, biopharmaceutical companies have embraced rare disease medicine and are rapidly developing the pipeline. In anticipation of an even more compelling opportunity, many companies are taking a step beyond and focusing on ultra rare diseases.

Last week’s 11th Annual BIO Investor Forum in San Francisco hosted a panel, Raring to Go: The Race to Treat Ultra-Rare Diseases, which provided an in-depth analysis of this thriving market opportunity by featuring companies specializing in the ultra rare disease market alongside clinical and policy / regulatory experts in the field.

Moderated by Ellen Licking, Senior Writer & Analyst, Real Endpoints, the panel opened with a focus on the legislation and the regulatory environment. Licking pointed out that there has been a recent uptick in interest on rare diseases which has led to an increase in deal-making. With the recent passage of FDASIA, there have been new opportunities identified for expediting review and approval of rare disease drugs, including accelerated approval, expanded reviewer training and priority review voucher programs to include pediatric rare diseases.

for complete article: BIO Investor Forum – Raring to Go: The Race to Treat Ultra-Rare Diseases

BIO Travels to Brazil to Support the Ecosystem of Innovation

from BIOtechNow by biotechnow@bio.org (Biotechnology Industry Organization)

 

Government policies that support innovation are critical as the biotechnology industry seeks to develop innovative healthcare, agricultural, industrial and environmental biotechnology products and provide them to people all around the world. Innovation requires not only scientific research and commercial expertise, but also supportive and dynamic governments that help facilitate long-term growth of the industry.

Brazil has recognized the value of innovation in maintaining a robust, diversified economy that can compete in the 21st century, and declared biotechnology a national priority. Boosting biotech within Brazil has the potential to create high paying jobs and develop a healthier, more productive workforce.

This week BIO will be in Brazil for a roundtable meeting with representatives from Brazil’s National Committee on Biotechnology (or Comité Nacional de Biotecnologia (CNB)), representing close to a dozen relevant government agencies and ministries including the Ministry of Trade’s Innovation Secretariat, Ministry of Agriculture, Ministry of Health and Ministry of Science, Technology & Innovation.

for complete article: BIO Travels to Brazil to Support the Ecosystem of Innovation

FDA: Navigating the changes with professional development

The Food and Drug Administration Safety and Innovation Act (FDASIA) is Law. Now What?  Join DIA, Food and Drug Administration (FDA), and The Food and Drug Law Institute (FDLI) as they convene the foremost experts in separate conferences to explain the nuances and forecast the implications of the User Fee Acts—PDUFA V, MDUFA III, GDUFA, and BsUFA. DIA/FDA Biosimilars Conference: Guidances, Science, and BsUFA September 12-13 | Washington, DC Co-sponsored by In 2009, the Biologics Price Competition and Innovation Act (BPCI Act) was enacted as part of the Affordable Care Act. This conference will deal with the scientific and regulatory issues related to the demonstration of biosimilarity, focusing both on the FDA’s current thinking on approaches to biosimilar development as well as perspectives of other regulators. Hotel Early-Bird Rates End August 27! In collaboration with Keynote Speaker: Janet Woodcock, MD Director, Center for Drug Evaluation and Research (CDER) Food and Drug Administration (FDA) View Program Online | Register Today Unwrapping FDA's 2012 UFA Package: What's Inside the Statute, What's Next? September 20 | Washington, DC Co-sponsored by The content of the UFA packages will shape the timeliness of reviews of life-saving medicines and medical devices, the requirements of all phases of medical product development, and the industry’s ability to innovate and develop needed medical products. This one-day conference will provide a first look at the final packages enacted for PDUFA V, MDUFA III, GDUFA and BsUFA. FDA will discuss the goals and commitments for their Centers, the changes of emphasis, and the potential impact on future programs. Reactor panels will provide the exchange of views on what the UFA provisions will mean for innovation, how they will improve patient access to needed medical products over the next five years, and next steps in preparing for 2017. Keynote Speakers: Jeanne Ireland Assistant Commissioner for Legislation Food and Drug Administration (FDA) Jeffrey E. Shuren, MD, JD Director, Center for Devices and Radiological Health (CDRH) Food and Drug Administration (FDA) View Program Online | Register Today DIA/FDA Industry PDUFA V Conference October 18-19 | Arlington, VA Co-sponsored by With the recent authorization of PDUFA V, this two-day conference sets the stage for FDA, industry, patient, consumer, and healthcare professional group representatives, and technology vendors to discuss the key PDUFA V commitments, current plans for their implementation, and potential impact on all stakeholders. Keynote Speakers: Theresa M. Mullin, PhD Associate Director, Office of Planning and Informatics Center for Drug Evaluation and Research (CDER) Food and Drug Administration (FDA) David Wheadon, MD Senior Vice President, Scientific and Regulatory Affairs PhRMA   View Program Online | Register Today My DIA    |    Forward to a Friend    |

FDA and You - DIA has you covered this fall (FDASIA, UFA and PDUFA)

The Food and Drug Administration Safety and Innovation Act (FDASIA) is Law. Now What? Join DIA, Food and Drug Administration (FDA), and The Food and Drug Law Institute (FDLI) as they convene the foremost experts in separate meetings to explain the nuances and forecast the implications of the User Fee Acts—PDUFA V, MDUFA III, GDUFA and BsUFA. DIA/FDA Biosimilars Conference: Guidances, Science, and BsUFA September 12-13 | Washington, DC Co-sponsored by In 2009, the Biologics Price Competition and Innovation Act (BPCI Act) was enacted as part of the Affordable Care Act. This conference will deal with the scientific and regulatory issues related to the demonstration of biosimilarity, focusing both on the FDA’s current thinking on approaches to biosimilar development as well as perspectives of other regulators. In collaboration with Keynote Speaker: Janet Woodcock, MD Director, Center for Drug Evaluation and Research (CDER) Food and Drug Administration (FDA) View Program Details | Enroll Today Unwrapping FDA's 2012 UFA Package: What's Inside the Statute, What's Next? September 20 | Washington, DC Co-sponsored by The content of the UFA packages will shape the timeliness of reviews of life-saving medicines and medical devices, the requirements of all phases of medical product development, and the industry’s ability to innovate and develop needed medical products. This one-day conference will provide a first look at the final packages enacted for PDUFA V, MDUFA III, GDUFA and BsUFA. FDA will discuss the goals and commitments for their Centers, the changes of emphasis, and the potential impact on future programs. Reactor panels will provide the exchange of views on what the UFA provisions will mean for innovation, how they will improve patient access to needed medical products over the next five years, and next steps in preparing for 2017. Keynote Speakers: Jeanne Ireland Assistant Commissioner for Legislation Food and Drug Administration (FDA) Jeffrey E. Shuren, MD, JD Director, Center for Devices and Radiological Health (CDRH) Food and Drug Administration (FDA) View Program Details | Enroll Today DIA/FDA Industry PDUFA V Conference October 18-19 | Arlington, VA Co-sponsored by This two-day conference sets the stage for professionals in the FDA, Industry, patient, consumer, and health care groups to give new perspectives to current plans and potential impact on all stakeholders on the Prescription Drug User Fee Act (PDUFA). When PDUFA was enacted in 1992, it created amazing forward thinking in public health. Patients now have quicker access to over 1,500 new biologics and drugs, focusing on cancer treatments, infectious diseases, neurological and psychiatric disorders, and cardiovascular diseases all the while maintaining FDA’s meticulous safety and drug efficacy standards. With the recent authorization of PDUFA V, this conference will explore the next steps. View Program Details | Enroll Today

FDA’s draft guidance documents on Biosimilars - from PharmTech Talk

From PharmTech Talk

Published by Amy Ritter on April 25, 2012 02:00 pm under Regulation

Apr. 16, 2012 marked the closing date for submitting comments on FDA’s draft guidance documents clarifying the approval process for biosimilars. More than 35 pharmaceutical companies, trade associations, and patient advocacy groups submitted public comments on the guidance documents, which are available here.

A careful slog through the comments, revealed a few common issues, but also some topics that reflected the commentator’s industry outlook. Both BIO and PhRMA, industry trade groups, commented on the importance of protecting the innovator’s data until the period of exclusivity had expired, and also requested that the criteria for granting exclusivity be further clarified.

More than a few of the commentators asked for clarification around interchangeability. The guidance document has little detail on the topic, other than defining it as an approval designation permitting free substitution with the innovator product. Companies including Elan, Bayer, and Novo Nordisk asked that the agency provide more detail around the requirements for interchangeability, both in terms of product characterization and the sequence of steps to follow to obtain that designation.

for more of Amy's analysis, see http://blog.pharmtech.com/2012/04/25/a-few-comments-on-the-comments/

BIO Testifies on Importance of Timely Passage of BsUFA and PDUFA

BIO Testifies on Importance of Timely Passage of BsUFA and PDUFA

Washington, D.C. (March 29, 2012) – Sara Radcliffe, Executive Vice President of Health at the Biotechnology Industry Organization (BIO), testified today on biosimilar user fees (BsUFA) and the Prescription Drug User Fee Act (PDUFA) before the Senate Health, Education, Labor and Pensions Committee in a hearing entitled, “FDA User Fee Agreements: Strengthening FDA and the Medical Products Industry for the Benefit of Patients.”

In her testimony, Radcliffe expressed support for the establishment of a biosimilars user fee program as part of the Food and Drug Administration’s (FDA) implementation of a well-constructed, science-based pathway for the approval of biosimilar products that protects patient safety and preserves incentives for innovative new therapies.

“A transparent, predictable, and balanced regulatory framework for the review and approval of biosimilars, accompanied by reasonable performance goals and a dedicated, independent funding stream, will ensure that FDA can facilitate the development and evaluation of biosimilars products,” Radcliffe testified.

click link for full article: BIO Testifies on Importance of Timely Passage of BsUFA and PDUFA