# Presentation: 079158

**MDUFMA Reauthorization: **
**Key Issues For Venture Capital**
***Jack Lasersohn***
***NVCA, Medical Industry Group***
***Vertical Group***

## Venture Capital Improves the Lives of Millions

**OVER 100 MILLION AMERICANS**
- have been positively affected by venture-backed medical innovations developed during the past 20 years
**OVER 25 MILLION AMERICANS**
- have their life extended or quality of life improved        every year, through the use of venture-backed diagnostic and therapeutic products

## Venture Capital Combats America’s Leading Causes of Death

| Cause of
Death | Deaths
(2001) | Venture Capital
Support 
(20 years) | Venture Backed
Innovative Treatments |
| --- | --- | --- | --- |
| Heart
Disease | 700,142 | $22 billion | angioplasty, minimally invasive
coronary bypass, electroablation,
implantable cardioverter-defibrillators (ICD’s),
automated external defibrillators (AED’s)
Integrilin, ReoPro
 |
| Cancer | 553,768 | $9 billion | doppler ultrasound, minimally
invasive biopsy, PSA, MRI,
Avastin, Erbitux, Velcade,
Gliadel, Herceptin, Rituxan |
| Stroke | 163,538 | $1.3 billion | MRI, TPA
 |
| Respiratory
Disease | 123,013 | $19.5 billion | FluMist, mechanical ventilators, pulse oximetry
 |

## Life Sciences and Healthcare Services YTD Make Up Almost 1/3 of Total Venture Investment

**Life Sciences and Healthcare Services YTD Make Up Almost 1/3 of ****Total Venture Investment**

***Source: PricewaterhouseCoopers/Venture Economics/National Venture Capital Association MoneyTreeTM - 9m2004***

## Number of First Financings forLS Companies Remains Strong

**Number of First Financings for****LS Companies Remains Strong**

***Source: PricewaterhouseCoopers/Venture Economics/National Venture Capital Association MoneyTreeTM***

## Medical Device Startups

***Source: PricewaterhouseCoopers/Venture Economics/National Venture Capital Association MoneyTreeTM***

***First Financings for Medical Device Companies***

**Millions**

## Total Life Science Investment Is Much Greater than Pre-2000 Levels

***Source: PricewaterhouseCoopers/Venture Economics/National Venture Capital Association MoneyTreeTM***

## Number of LS CompaniesReceiving Funding Is Steady

***Source: PricewaterhouseCoopers/Venture Economics/National Venture Capital Association MoneyTreeTM***

## Medical Device Industry Remains Strong 

***Source: PricewaterhouseCoopers/Venture Economics/National Venture Capital Association MoneyTreeTM***

***VC Funding for Medical Device Companies***

**Millions**

## Funding Requirements for Medical Devices

**Funding Requirements ****for Medical Devices**

_**1980**_
**$3 million**
**Balloon Angioplasty**

_**2000**_
**$75 million**
**Balloon Kyphoplasty**

***Start up to Profitability***

## VC Funded Medical Devices

**Angioplasty**
**MRI**
**Pulse oximetry**
**Cardiac ablation**
**ICDs**

**AAA Stent Grafts**
**Beating Heart Surgery**
**Disc Arthroplasty**
**Kyphoplasty**
**Neurostimulation**

_***Revolutionary***_***, Not Evolutionary***

## Novelty CreatesUnique Regulatory Challenges

**Education of CDRH Staff**
**Lack of Precedents and Pathways**
**Clinical Trial Design and Interpretation **

## What VCs Are Doing in 2005

_**Project**_
**Spinal Nucleus replacements**
**Dynamic spine stabilization**
**Intervention Treatment of Peripheral Atherosclerosis**
**Biodegradeable or Novel Stents**
**Neurostimulation**
**    ****(Pain, Epilepsy, Depression)**
**PFO Closure**
**Vulnerable Plaque**

_**Companies**_
**	****	****>15**
**	****	****>15**
**	****	****>15**
**	****	****>15**
**	****	****>10**
**	****	****>10**
**	****	****>10**

## What VCs Are Doing in 2005

_**Project**_
**Cartilage Repair**
**Stabilization of Heart Failure**
**Interventions for AMI**
**Interventions for Acute Stroke**
**3rd Generation AAA Gaffs**
**Artificial Retinas**
**3rd Generation LVADs**
**Acute Interventions for DVTs**

_**Companies**_
**	****	****>10**
**	****	****>10**
**	****	****>10**
**	****	****>5**
**	****	****>5**
**	****	****>5**
**	****	****>5**
**	****	****>5**

## Performance Goals and Funding

- Substantial additional improvements  possible in the** ****timeliness, transparency and consistency**** **of product reviews  without a substantial increase in fees
- Assuring** ****greater consistency, predictability and quality**** **in the review of expedited PMAs and modular PMAs to be of the greatest importance

## Performance Goals and Funding

- FDA and sponsors should consider the potential for** ****modest, non-application fees****  **reflecting appropriate small business exemptions to** ****increase the stability**** **of MDUFMA funding and** ****decrease existing application fees**, as well as to better assure** ****greater consistency, predictability and quality**** **of premarket reviews

## Achieving First Cycle Approvals and Clearances 

- FDA must recommit to consistency and clarity in its **application of “least burdensome” principles** to reduce unnecessary regulatory burden
- Congress should consider the necessity of **additional statutory guidance** to advance timely patient access to innovative treatments while ensuring CDRH seeks and receives from sponsors the appropriate assurance of device safety and effectiveness

## Achieving First Cycle Approvals and Clearances 

**Meeting management performance goals** with specific timeframes for determination and agreement meetings, informal pre-IDE meetings, pre-PMA meetings, pre-PMA filing meetings should be a critical feature of any future MDUFMA agreement
- Targeted strategies like **special protocol assessments (SPA)**, and **continuous marketing application (CMA)** demonstrations should be considered

## COA and Postmarket Surveillance Studies 

**Sponsor compliance** with postmarket surveillance studies should be reported accurately.  CDRH **reporting and oversight should be improved**
- FDA should **issue guidance and outreach** to IRBs to expedite approval and conduct of COA studies

## HDEs and Pediatric Devices 

- Reconsider restrictive HDE/HUD guidance requiring sponsors to demonstrate that devices cannot be used for any other **“medically plausible” subpopulations** other than the population intended to be treated under a HDE designation
- Statutory and regulatory changes needed to raise the **patient population cap** and to remove the **profit restriction** on HDE/HUDs
**Reduce unnecessary burdens** and clarify **diversity of study designs** permissible to establish safety and effectiveness, as well as the different forms that “valid scientific evidence” may take, for  pediatric populations

## Critical Path Initiative

- As medical technology advances at an accelerating rate, the **Critical Path Initiative** is an important opportunity to advance the scientific basis for CDRH operations
**NVCA is committed to collaborating with FDA  **to identify new scientific and regulatory opportunities to get safe and effective medical devices and radiological products to market more quickly

## Thank You